Tutkimus emitsitsumabin turvallisuuden, tehon, farmakokinetiikkaa ja farmakodynamiikasta arvioimiseksi potilailla, joilla on lievä tai keskivaikea hemofilia A ilman FVIII-estäjiä (HAVEN 6)
keskiviikko 5. elokuuta 2026 päivittänyt: Hoffmann-La Roche
Monikeskustutkimus, jossa arvioidaan emitsitsumabin turvallisuutta, tehokkuutta, farmakokinetiikkaa ja farmakodynamiikkaa potilailla, joilla on lievä tai keskivaikea hemofilia A ilman FVIII-estäjiä
Tämä on monikeskus, avoin, yksihaarainen tutkimus, jonka tarkoituksena on arvioida emitsitsumabin turvallisuutta, tehoa, farmakokinetiikkaa ja farmakodynamiikkaa potilailla, joilla on lievä tai keskivaikea hemofilia A ilman tekijä VIII:n (FVIII) estäjiä.
Tutkimuksen yleiskatsaus
Tila
Valmis
Ehdot
Interventio / Hoito
Opintotyyppi
Interventio
Ilmoittautuminen (Todellinen)
73
Vaihe
- Vaihe 3
Yhteystiedot ja paikat
Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.
Opiskelupaikat
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Amsterdam, Alankomaat, 1105 AZ
- Amsterdam UMC Location AMC
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Brussels, Belgia, 1200
- Cliniques Universitaires St-Luc
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Leuven, Belgia, 3000
- UZ Leuven Gasthuisberg
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Madrid, Espanja, 28046
- Hospital Universitario La Paz
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Seville, Espanja, 41013
- Hospital Universitario Virgen del Rocío
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Johannesburg, Etelä-Afrikka, 2193
- Charlotte Maxeke Johannesburg Hospital
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Alberta
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Edmonton, Alberta, Kanada, T6G 1Z1
- Kaye Edmonton Clinic
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Newfoundland and Labrador
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St. John's, Newfoundland and Labrador, Kanada, A1B 3V6
- Eastern Health - General Hospital
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Warsaw, Puola, 02-776
- Instytut Hematologii i Transfuzjologii
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Bron, Ranska, 69677
- Hopital Cardio-vasculaire Louis Pradel
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Le Kremlin-Bicêtre, Ranska, 94275
- CH de Bicêtre
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Paris, Ranska, 75015
- Groupe Hospitalier Necker Enfants Malades
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Bonn, Saksa, 53127
- Universitatsklinikum Bonn
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München, Saksa, 80336
- Klinikum der Universität München, Campus Innenstadt
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Cardiff, Yhdistynyt kuningaskunta, CF14 4XW
- Cardiff and Vale NHS Trust
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London, Yhdistynyt kuningaskunta, NW3 2QG
- Royal Free Hospital
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London, Yhdistynyt kuningaskunta, WC1N 3HR
- Great Ormond Street Hospital for Children NHS Foundation Trust
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California
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Los Angeles, California, Yhdysvallat, 90027
- Childrens Hospital LA
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Georgia
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Atlanta, Georgia, Yhdysvallat, 30308
- Hemophilia of Georgia Center for Bleeding & Clotting Disorders
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Indiana
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Indianapolis, Indiana, Yhdysvallat, 46260
- Indiana Hemophilia & Thrombosis center
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Michigan
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Ann Arbor, Michigan, Yhdysvallat, 48109
- University of Michigan, C.S. Mott Children's Hospital
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Washington
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Seattle, Washington, Yhdysvallat, 98101
- Washington Institute for Coagulation
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Osallistumiskriteerit
Tutkijat etsivät ihmisiä, jotka sopivat tiettyyn kuvaukseen, jota kutsutaan kelpoisuuskriteereiksi. Joitakin esimerkkejä näistä kriteereistä ovat henkilön yleinen terveydentila tai aiemmat hoidot.
Kelpoisuusvaatimukset
Opintokelpoiset iät
- Lapsi
- Aikuinen
- Vanhempi Aikuinen
Hyväksyy terveitä vapaaehtoisia
Ei
Kuvaus
Sisällyttämiskriteerit:
- Diagnoosi lievä (FVIII-taso välillä >5 % ja
- Paino ≥3 kilogrammaa (kg)
- Ennaltaehkäisyn tarve tutkijan arvion perusteella
- Negatiivinen testi inhibiittorille (esim.
- Ei dokumentoitua inhibiittoria (ts.
- Asiakirjat profylaktisen tai episodisen FVIII-hoidon yksityiskohdista ja vuotokohtausten lukumäärästä vähintään 24 viimeisten viikkojen aikana ennen ilmoittautumista
- Riittävä hematologinen maksan ja munuaisten toiminta
- Hedelmällisessä iässä olevat naiset: suostumus pysymään pidättyväisenä tai käyttämään ehkäisyä (kuten protokollassa on määritelty) hoitojakson aikana ja vähintään 24 viikkoa tutkimuslääkkeen viimeisen annoksen jälkeen
Poissulkemiskriteerit:
- Muu perinnöllinen tai hankittu verenvuotohäiriö kuin lievä tai keskivaikea synnynnäinen hemofilia A
- Anamneesi laittomien huumeiden tai alkoholin väärinkäyttöä 48 viikon aikana ennen seulontaa, tutkijan arvion mukaan
- Aiempi (viimeisten 12 kuukauden aikana) tai nykyinen hoito tromboemboliseen sairauteen tai tromboembolisen sairauden oireisiin
- Muut sairaudet, jotka voivat tällä hetkellä lisätä verenvuodon tai tromboosin riskiä
- Aiemmin kliinisesti merkittävä yliherkkyys, joka liittyy monoklonaalisiin vasta-ainehoitoihin tai emitistsumabi-injektion komponentteihin
- Suunniteltu leikkaus emitistsumabin kyllästysannosvaiheen aikana (emitistsumabia saaneiden osallistujien leikkaukset viikosta 5 alkaen ovat sallittuja)
- Tunnettu HIV-infektio ja CD4-määrät
- Samanaikainen sairaus, tila, merkittävä poikkeavuus seulontaarvioinnissa tai laboratoriokokeissa tai hoito, joka voisi häiritä tutkimuksen suorittamista tai joka tutkijan mielestä aiheuttaisi ylimääräisen ei-hyväksyttävän riskin tutkimuslääkkeen antamisessa osallistujalle
- Kuitti jostakin seuraavista: Tutkimuslääke hemofiilisen verenvuodon hoitoon tai riskin vähentämiseen 5 puoliintumisajan sisällä viimeisestä lääkkeen annosta, lukuun ottamatta aikaisempaa emitsitsumabin estohoitoa; Ei-hemofiliaan liittyvä tutkimuslääke viimeisten 30 päivän tai 5 puoliintumisajan sisällä sen mukaan, kumpi on lyhyempi; tai mikä tahansa muu tutkimuslääke, jota parhaillaan annetaan tai jota suunnitellaan annettavaksi
- Kyvyttömyys noudattaa tutkimuspöytäkirjaa tutkijan mielestä
- Raskaana tai imetyksen aikana tai aikovat tulla raskaaksi tutkimuksen aikana (hedelmällisessä iässä olevilla naisilla on oltava negatiivinen seerumin raskaustesti 7 päivän kuluessa ennen tutkimuslääkkeen aloittamista)
Opintosuunnitelma
Tässä osiossa on tietoja tutkimussuunnitelmasta, mukaan lukien kuinka tutkimus on suunniteltu ja mitä tutkimuksella mitataan.
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
- Ensisijainen käyttötarkoitus: Hoito
- Jako: Ei käytössä
- Inventiomalli: Yksittäinen ryhmätehtävä
- Naamiointi: Ei mitään (avoin tarra)
Aseet ja interventiot
Osallistujaryhmä / Arm |
Interventio / Hoito |
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Kokeellinen: Emisitsumabi
Osallistujat, joilla on lievä tai keskivaikea hemofilia A ilman tekijä VIII (FVIII) estäjiä, otetaan mukaan emitistsumabin kyllästysannosohjelmaan, jonka jälkeen osallistuja valitsee yhden kolmesta ylläpitoannostusohjelmasta.
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Neljä emitistsumabin kyllästysannosta 3 milligrammaa painokiloa kohden (mg/kg) annetaan ihonalaisesti (SC) kerran viikossa (QW) 4 viikon ajan, minkä jälkeen osallistuja valitsee jonkin seuraavista kolmesta SC ylläpitoannostusohjelmasta: 1,5 mg /kg QW, 3 mg/kg kerran 2 viikossa (Q2W) tai 6 mg/kg kerran 4 viikossa (Q4W).
Muut nimet:
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Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Mallipohjainen vuotuinen vuotoaste hoidetuille verenvuodoille
Aikaikkuna: Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Hoidettujen vuotojen määrä tehokkuusjakson aikana arvioitiin vuositasolla (ABR) käyttämällä negatiivista binomiaalista regressiomallia, joka vastaa eri seuranta-aikoja.
Käsitelty verenvuoto määriteltiin verenvuodoksi, jota seurasi suoraan hemofilialääkitys, jonka ilmoitettiin olevan "verenvuodon hoito".
72 tunnin sääntö otettiin käyttöön: kaksi samantyyppistä ja samasta anatomisesta sijainnista saatua verenvuotoa laskettiin yhdeksi verenvuodoksi, jos toinen verenvuoto tapahtui 72 tunnin sisällä ensimmäisen verenvuodon viimeisestä hoidosta.
Leikkauksesta/toimenpiteestä johtuvat verenvuodot jätettiin pois.
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Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Mean Calculated ABR for Treated Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Keskimääräinen laskettu vuotuinen vuotonopeus hoidetuille spontaaneille verenvuodoille
Aikaikkuna: Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Hoidettujen spontaanien vuotojen määrä tehokkuusjakson aikana esitetään tässä laskennallisena vuotuisena vuotomääränä (ABR), joka vuositasoitettiin kullekin osallistujalle seuraavalla kaavalla: ABR = (vuotojen määrä / päivien lukumäärä tehokkuusjakson aikana) x 365,25 .
Käsitelty spontaani verenvuoto määriteltiin hoidetuksi verenvuodoksi (verenvuoto, jota seurasi välittömästi hemofilialääkitys, jonka kerrottiin olevan "hoito verenvuodon hoitoon") ilman muita tunnettuja vaikuttavia tekijöitä, kuten trauma tai toimenpide/leikkaus.
72 tunnin sääntö otettiin käyttöön: kaksi samantyyppistä ja samasta anatomisesta sijainnista saatua verenvuotoa laskettiin yhdeksi verenvuodoksi, jos toinen verenvuoto tapahtui 72 tunnin sisällä ensimmäisen verenvuodon viimeisestä hoidosta.
Leikkauksesta/toimenpiteestä johtuvat verenvuodot jätettiin pois.
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Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Keskimääräinen laskettu vuotuinen verenvuotoprosentti hoidetuille spontaaneille verenvuodoille
Aikaikkuna: Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Hoidettujen spontaanien vuotojen määrä tehokkuusjakson aikana esitetään tässä laskennallisena vuotuisena vuotomääränä (ABR), joka vuositasoitettiin kullekin osallistujalle seuraavalla kaavalla: ABR = (vuotojen määrä / päivien lukumäärä tehokkuusjakson aikana) x 365,25 .
Käsitelty spontaani verenvuoto määriteltiin hoidetuksi verenvuodoksi (verenvuoto, jota seurasi välittömästi hemofilialääkitys, jonka kerrottiin olevan "hoito verenvuodon hoitoon") ilman muita tunnettuja vaikuttavia tekijöitä, kuten trauma tai toimenpide/leikkaus.
72 tunnin sääntö otettiin käyttöön: kaksi samantyyppistä ja samasta anatomisesta sijainnista saatua verenvuotoa laskettiin yhdeksi verenvuodoksi, jos toinen verenvuoto tapahtui 72 tunnin sisällä ensimmäisen verenvuodon viimeisestä hoidosta.
Leikkauksesta/toimenpiteestä johtuvat verenvuodot jätettiin pois.
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Ensimmäisen emitistsumabiannoksen päivästä vähintään 52 viikon emitistsumabihoitoon (mediaani [alue, min-max] tehon ajanjakso: 55,64 [8,6-89,9] viikkoa)
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Niiden osallistujien prosenttiosuus, jotka suosivat emitistsumabin SC-hoitoa, heidän aiempaa hemofilia IV -hoitoaan tai eivät saa etusijaa, arvioituna käyttämällä Emitsitsumabin mieltymystutkimusta viikolla 17
Aikaikkuna: Viikko 17
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Emicitsumab Preference Survey on toimeksiantajan kehittämä tarkoitukseen sopiva kyselylomake, jolla kirjataan osallistujan mieltymys ihonalaiseen (SC) emitistsumabihoitoon, suonensisäiseen (IV) tekijä VIIII:ään (FVIII) tai ei.
95 %:n luottamusvälit laskettiin Pearson-Clopper-menetelmällä.
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Viikko 17
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Niiden omaishoitajien prosenttiosuus, jotka suosivat emitistsumabin SC-hoitoa, heidän lapsensa aiempaa hemofilia IV -hoitoa tai heillä ei ole etusijaa, arvioituna käyttämällä Emitsitsumabin mieltymystutkimusta viikolla 17
Aikaikkuna: Viikko 17
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Emicitsumab Preference Survey on toimeksiantajan kehittämä tarkoitukseen sopiva kyselylomake tallentaakseen hoitajan mieltymyksen lapsensa ihonalaiseen (SC) emitistsumabihoitoon, suonensisäiseen (IV) tekijä VIIII:ään (FVIII) tai ei.
95 %:n luottamusvälit laskettiin Pearson-Clopper-menetelmällä.
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Viikko 17
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Muutos lähtötasosta päivittäisen aktiivisuuden keskimääräisessä huippukestossa ajan myötä
Aikaikkuna: Lähtötilanne (viikot 1–2) ja viikot 13 (jaksot 12–13), 25 (jaksot 24–25), 37 (jaksot 36–37) ja 49 (jaksot 48–49)
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Fyysisen aktiivisuuden arviointia varten 5-vuotiaita osallistujia kehotettiin pitämään tutkimuksen kiihtyvyysmittaria ranteessa jatkuvasti (24 tuntia/vrk) joka päivä määrättyjen 2 viikon ajanjaksojen ajan tutkimuksen aikana.
Osallistujan katsottiin noudattavan fyysisen aktiivisuuden arviointia, jos hän käytti tutkimuslaitetta jatkuvasti (≥ 8 tuntia/päivä) joka päivä vähintään 8 päivän ajan jokaisesta määrätystä 2 viikon jaksosta tutkimuksen aikana.
Jos tätä vaatimustenmukaisuuskriteeriä ei saavutettu tiettynä ajankohtana, osallistujaa ei otettu mukaan niiden 2 viikon jaksojen analyysiin, joissa vaatimustenmukaisuutta ei saavutettu.
Päivittäisistä mittauksista laskettiin keskiarvo 14 päivän aikapisteeltä.
Aktiivisuusmäärä oli laitteen mittaaman kiihtyvyyden mitta.
Päivittäinen huippuaktiivisuuden kesto määriteltiin kohtalaisen voimakkaan aktiivisuuden summana päivässä (minuutteina).
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Lähtötilanne (viikot 1–2) ja viikot 13 (jaksot 12–13), 25 (jaksot 24–25), 37 (jaksot 36–37) ja 49 (jaksot 48–49)
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Muutos lähtötasosta keskimääräisessä päivittäisessä askelmäärässä ajan kuluessa
Aikaikkuna: Lähtötilanne (viikot 1–2) ja viikot 13 (viikot 12–13), 25 (viikot 24–25), 37 (viikot 36–37) ja 49 (viikot 48–49)
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Fyysisen aktiivisuuden arviointia varten 5-vuotiaita osallistujia kehotettiin pitämään tutkimuksen kiihtyvyysmittaria ranteessa jatkuvasti (24 tuntia/vrk) joka päivä määrättyjen 2 viikon ajanjaksojen ajan tutkimuksen aikana.
Osallistujan katsottiin noudattavan fyysisen aktiivisuuden arviointia, jos hän käytti tutkimuslaitetta jatkuvasti (≥ 8 tuntia/päivä) joka päivä vähintään 8 päivän ajan jokaisesta määrätystä 2 viikon jaksosta tutkimuksen aikana.
Jos tätä vaatimustenmukaisuuskriteeriä ei saavutettu tiettynä ajankohtana, osallistujaa ei otettu mukaan niiden 2 viikon jaksojen analyysiin, joissa vaatimustenmukaisuutta ei saavutettu.
Päivittäisistä mittauksista laskettiin keskiarvo 14 päivän aikapisteeltä.
Aktiivisuusmäärä oli laitteen mittaaman kiihtyvyyden mitta.
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Lähtötilanne (viikot 1–2) ja viikot 13 (viikot 12–13), 25 (viikot 24–25), 37 (viikot 36–37) ja 49 (viikot 48–49)
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Model-Based ABR for All Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Mean Calculated ABR for All Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for All Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Model-Based ABR for Treated Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Mean Calculated ABR for Treated Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
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From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
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Median Calculated ABR for Treated Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Target Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Mean Calculated ABR for Treated Target Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Median Calculated ABR for Treated Target Joint Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Model-Based ABR for Treated Spontaneous Bleeds
Aikaikkuna: From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
From the day of first emicizumab dose to at least 52 weeks of emicizumab treatment (median [range, min-max] efficacy period: 55.64 [8.6-89.9] weeks)
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Bleeds
Aikaikkuna: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Bleeds
Aikaikkuna: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Bleeds
Aikaikkuna: Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated bleed was defined as a bleed that was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
Median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for All Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for All Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy: Median Calculated ABR for All Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of all bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
In this outcome measure, all bleeds are included, irrespective of treatment with coagulation factors, with the following exception: bleeds due to surgery/procedure are excluded.
As "all bleeds" comprises both treated and non-treated bleeds, the 72-hour rule was implemented separately for treated and non-treated bleeds.
For treated bleeds, the 72-hour rule meant that two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
For non-treated bleeds, the 72-hour rule was implemented by calculating a treatment-free period of 72 hours from the bleed itself.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated joint bleed was defined as a bleed with type reported as "joint" based on at least one of the following symptoms: increasing swelling or warmth of the skin over the joint and/or increasing pain, decreased range of motion, or difficulty using the joint compared with baseline, and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Target Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Target Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Target Joint Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated target joint bleeds over the efficacy period is presented here as a calculated ABR that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated target joint bleed was defined as a joint bleed in a target joint, which is a joint location where at least 3 bleeds have occurred over the last 24 weeks prior to study entry or an unresolved target joint (target joint that does not fulfil ≤2 bleeds into this joint within a consecutive 12-month period), and the bleed was directly followed by a hemophilia medication reported to be a "treatment for bleed".
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Model-Based ABR for Treated Spontaneous Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period was estimated as an ABR using a negative binomial regression model, which accounts for different follow-up times.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Mean Calculated ABR for Treated Spontaneous Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
Long-term Efficacy of Emicizumab: Median Calculated ABR for Treated Spontaneous Bleeds
Aikaikkuna: median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
The number of treated spontaneous bleeds over the efficacy period is presented here as a calculated annualized bleed rate (ABR) that was annualized for each participant using the following formula: ABR = (number of bleeds/number of days during the efficacy period) x 365.25.
A treated spontaneous bleed was defined as a treated bleed (bleed directly followed by a hemophilia medication reported to be a "treatment for bleed") with no other known contributing factor such as trauma or procedure/surgery.
The 72-hour rule was implemented: two bleeds of the same type and at the same anatomical location were counted as one bleed if the second bleed occurred within 72 hours from the last treatment for the first bleed.
Bleeds due to surgery/procedure were excluded.
|
median [range, min-max] efficacy period: 156.07 [24.1-274] weeks
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Daily Activity Risk Perception (RP) and Impact Domain Scores Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the daily activity RP and impact domain has 48 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and Study completion (SC)/Early discontinuation (ED) (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Social Activity RP and Impact Domain Scores Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the social activity RP and impact domain has 22 items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Scores Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the recreational activity RP and impact domain has 32 items and a larger bank of additional items with each item scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale for both the RP and Impact.
Higher scores indicating higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Work Impact (WI) and College Impact (CI) Domains Score Over Time
Aikaikkuna: WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥ 18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, work and college impact has 10 items and the items were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated impact of hemophilia on work and college activities.
|
WI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks); CI: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 10 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 8 items which were scored on 4 or 5 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain Associated With a Bleed Over Time
Aikaikkuna: Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consisting of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question: "Did you get a bleed in the past 7 days?"
If the response was yes, participants were asked to rate their pain by selecting the number that best described the pain associated with the bleed.
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 25, 49, 61, 85, 121, 133, 157 and 169 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain in Target Joints Over Time
Aikaikkuna: Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Do you have target joints?"
(If yes, please rate your pain by picking the number that best describes your pain in your target joint(s) over the past 7 days).
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Worst Over Time
Aikaikkuna: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its WORST over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
|
CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least Over Time
Aikaikkuna: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its LEAST over the past 7 days."
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Adult Participants: Number of Participants by Responses to Their Level of Pain at Its Least on the Average Over Time
Aikaikkuna: Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on adult participants (aged ≥18 years).
The adult version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, work impact, preoccupation, treatment burden, and pain.
Of this, the pain domain consists of five items, assessed using an 11-point numeric rating scale where 0 = no pain and 10 = pain as bad as you can imagine.
The data reported here are from participants who answered the following question "Please rate your pain by picking the number that best describes your pain at its average over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 1, 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, daily activity RP and impact has 38 items with each item scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicating higher perceived risk of having a bleed while doing daily activities and higher impact of hemophilia on daily activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, social activity RP and impact has 16 items which were scored on 3 or 4 points ordinal scale.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing social activities and higher impact of hemophilia on social activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, recreational activity RP & impact has 34 items + larger bank of additional items.
The items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher perceived risk of having a bleed while doing recreational activities and higher impact of hemophilia on recreational activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, school impact has 11 items and the items were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated higher impact of hemophilia on school activities.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, preoccupation has 3 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, treatment burden has 7 items which were scored on 3 or 4 points ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept & applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated lower perceived burden of the hemophilia treatment.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
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CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain).
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "Did you get a bleed in the past 7 days?
( If yes, how much did the bleed hurt?)."
Data is reported only for categories/timepoints with non-zero values.
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Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
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CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on pediatric participants (aged 8-17 years).
The pediatric version has 7 domains (daily activity RP and impact, social activity RP and impact, recreational activity RP and impact, school impact, preoccupation, treatment burden, and pain.
Of this, pain domain (Hurt) consists of two items, assessed using an 11-point numeric rating scale where 0 = no hurt and 10 = a lot of hurt.
The data reported here are from participants who answered the following question "What is the WORST that your body has hurt over the past 7 days".
Data is reported only for categories/timepoints with non-zero values.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145,157, 169 and SC/ED (up to approximately 274 weeks)
|
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CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, preoccupation has 13 items with each of the items were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater preoccupation related to hemophilia.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
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CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
CATCH was used to measure the effect of hemophilia and its treatment on caregivers of pediatric participants (aged <8 years).
The caregiver version has 2 domains (preoccupation and treatment burden).
Of this, treatment burden has 8 items which were scored on 4 or 5 ordinal scales.
Raw domain scores were obtained by calculating the mean of the item scores for all items within the corresponding concept and applying a linear transformation.
The transformed scores range from 0-100 scale.
Higher scores indicated greater perceived burden of the hemophilia treatment.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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Change From Baseline in Hemophilia Joint Health Scores Over Time
Aikaikkuna: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
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The HJHS measures joint health of the joints (knees, ankles, and elbows) most commonly affected by bleeding in hemophilia.
The HJHS 2.1 provides joint specific total scores which are added to obtain the sum of joints totals and also a global gait score.
These two scores are then added to obtain HJHS total score.
The minimum score per joint is 0, the maximum score is 20.
In addition, Gait is scored on a scale from 0 to 4 based on the number of skills that are not within the normal limits.
The total score is the sum of scores across all six joints plus the gait score (range from 0 to 124, with 0 being normal and 124 being the most severe disease).
A higher score indicates worse joint health.
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Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
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Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
Aikaikkuna: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The MBQ score ranges from 0 to 75.
Higher scores indicate a worse quality of life and more severe symptoms.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
Aikaikkuna: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
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The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for heaviness subscale ranges from 0 to 29.
Higher score indicates more heavy bleeding.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137 (up to approximately 274 weeks)
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
Aikaikkuna: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for QoL subscale ranges from 0 to 37. Higher scores indicate a worse QoL.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
Aikaikkuna: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for irregularity subscale ranges from 0 to 6. Higher score indicates more irregularity.
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Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 85, 89, 93, 97, 101, 104, 109, 113, 117, 121, 125, 129, 133, 137, 141 and 145
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MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
Aikaikkuna: Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
|
The MBQ is a validated measure for menorrhagia and used to assess impact on the menstrual bleed-related heaviness, pain, irregularity, and quality of life (QoL).
The MBQ is a 20-item measure covering four aspects regarding heavy menstrual bleeding: heaviness (8 items), QoL (8 items), irregularity (3 item), and pain (1 item).
The total score for pain subscale ranges from 0 to 3. Higher score indicates severe pain.
|
Baseline, Weeks 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 53, 57, 61, 65, 69, 73, 77, 81, 89, 93, 97, 101, 104, 109, 113, 121, 125, 129, 133 and 137
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Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
Aikaikkuna: Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
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The Menstruation Diary used a PBAC, which has shown good correlation with menstrual blood loss.
The PBAC is a chart that records the occurrence and size of clots, and the number of episodes of heavy bleeding (flooding), as well as depicts the amount of blood loss during a cycle.The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month.
The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
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Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
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Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With Adverse Events Leading to Study Drug Discontinuation
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Thromboembolic Event
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
The following signs and symptoms were recognized as potential thromboembolism (i.e., dyspnea, chest pain, leg pain, or swelling; or if in the head, headache, numbness in the face, eye pain or swelling, or vision impairment).
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Event of Thrombotic Microangiopathy
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
TMA is used to describe a group of disorders with clinical features of microangiopathic hemolytic anemia, thrombocytopenia, and organ damage that can include the kidneys, gastrointestinal system, central nervous system, etc.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
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Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
Injection-site reactions are defined as AEs related that occur within 24 hours after study drug administration and are judged to be related to the study drug injection.
Local injection-site reactions included erythema, hematoma, rash, discomfort, pain, and pruritus and were mostly of mild and moderate intensity.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
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Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
Aikaikkuna: From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
An AE was defined as any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment.
An AE can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptoms, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product.
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From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
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Number of Participants With at Least One Shifts in Clinical Laboratory Parameters From Baseline WHO Toxicity Scale Grade 0-2 to Post-baseline Grade 3 or 4
Aikaikkuna: Up to approximately 274 weeks
|
An abnormal laboratory value was defined as a laboratory test result outside of the normal range for hematology or serum chemistry parameters.
The WHO toxicity grading scale, which ranges from Grades 1 to 4 (least severe to most severe, respectively; Grade 0 is within normal range), was used for assessing the severity of laboratory abnormalities and adverse events (WHO 2003).
Not every laboratory abnormality qualified as an adverse event; an abnormality was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
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Up to approximately 274 weeks
|
|
Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
Aikaikkuna: Up to approximately 274 weeks
|
The number of participants with adverse events of changes from baseline in vital signs is reported here.
Vital signs measurements consisted of heart and respiratory rate, temperature, and systolic and diastolic blood pressures, with an abnormal vital sign value being outside of the normal range.
An abnormal vital sign result was reported as an adverse event if it met any of the following criteria: was accompanied by clinical symptoms; resulted in a change in study treatment (e.g., dosage modification, treatment interruption or discontinuation); resulted in a medical intervention or a change in concomitant therapy; or was clinically significant in the investigator's judgment.
All of the adverse events reported here were assessed independently by the investigator as not related to treatment with emicizumab.
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Up to approximately 274 weeks
|
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Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
Aikaikkuna: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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|
|
Change From Baseline in Heart Rate Over Time, as Measured by ECG
Aikaikkuna: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
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Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
Aikaikkuna: Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
|
Pre-dose at Weeks 1, 2, 3, 4, 5, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169 and 181 (up to approximately 274 weeks)
|
|
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Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
Aikaikkuna: Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
|
Participants were considered to be ADA positive if they were ADA negative at baseline but develop an ADA response following study drug administration (treatment-induced ADA response), or if they were ADA positive at baseline and the titer of one or more post-baseline samples is at least 4-fold greater than the titer of the baseline sample (treatment-enhanced ADA response).
The sum of all visits has been reported here.
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Baseline, Weeks 5, 13, 25, 33, 41, 49, 61, 73, 85, 97, 109, 121, 133, 145, 169, 181 and SC/ED (up to approximately 274 weeks)
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Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
Aikaikkuna: Up to approximately 274 weeks
|
Central laboratory measurement of anti-FVIII inhibitory antibodies (inhibitors) was performed using a Chromogenic Bethesda Assay (CBA).
Participants who develop anti-FVIII inhibitors (titer ≥ 0.6 Bethesda units per milliliter [BU/mL]) following study drug administration were summarized.
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Up to approximately 274 weeks
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Yhteistyökumppanit ja tutkijat
Täältä löydät tähän tutkimukseen osallistuvat ihmiset ja organisaatiot.
Sponsori
Tutkijat
- Opintojohtaja: Clinical Trials, Hoffmann-La Roche
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Tutkimusta koskevien tietojen syöttämisestä vastaava henkilö toimittaa nämä julkaisut vapaaehtoisesti. Nämä voivat koskea mitä tahansa tutkimukseen liittyvää.
Opintojen ennätyspäivät
Nämä päivämäärät seuraavat ClinicalTrials.gov-sivustolle lähetettyjen tutkimustietueiden ja yhteenvetojen edistymistä. National Library of Medicine (NLM) tarkistaa tutkimustiedot ja raportoidut tulokset varmistaakseen, että ne täyttävät tietyt laadunvalvontastandardit, ennen kuin ne julkaistaan julkisella verkkosivustolla.
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Todellinen)
Maanantai 10. helmikuuta 2020
Ensisijainen valmistuminen (Todellinen)
Lauantai 30. lokakuuta 2021
Opintojen valmistuminen (Todellinen)
Perjantai 19. joulukuuta 2025
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Torstai 7. marraskuuta 2019
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Torstai 7. marraskuuta 2019
Ensimmäinen Lähetetty (Todellinen)
Tiistai 12. marraskuuta 2019
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Torstai 27. elokuuta 2026
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Keskiviikko 5. elokuuta 2026
Viimeksi vahvistettu
Lauantai 1. elokuuta 2026
Lisää tietoa
Tähän tutkimukseen liittyvät termit
Avainsanat
Muita asiaankuuluvia MeSH-ehtoja
Muut tutkimustunnusnumerot
- BO41423
- 2019 (Yhdysvaltain NIH-apuraha/sopimus: Chief Medical Office (CMO) Alberta Health Services)
- 2023 (Yhdysvaltain NIH-apuraha/sopimus: GRAMMY Museum Foundation)
- 2019-002179-32 (EudraCT-numero)
- 2023-506610-52-00 (Rekisterin tunniste: EU CT Number)
Yksittäisten osallistujien tietojen suunnitelma (IPD)
Aiotko jakaa yksittäisten osallistujien tietoja (IPD)?
JOO
IPD-suunnitelman kuvaus
Pätevät tutkijat voivat pyytää pääsyä yksittäisten potilastason tietoihin pyyntöalustan (www.vivli.org) kautta. Lisätietoja Rochen tukikelpoisten opintojen kriteereistä on saatavilla täältä (https://vivli.org/ourmember/roche/).
Lisätietoja Rochen kliinisten tutkimustietojen jakamista koskevasta maailmanlaajuisesta käytännöstä ja siihen liittyvien kliinisten tutkimusten asiakirjojen pyytämisestä on täällä (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
Lääke- ja laitetiedot, tutkimusasiakirjat
Tutkii yhdysvaltalaista FDA sääntelemää lääkevalmistetta
Joo
Tutkii yhdysvaltalaista FDA sääntelemää laitetuotetta
Ei
Nämä tiedot haettiin suoraan verkkosivustolta clinicaltrials.gov ilman muutoksia. Jos sinulla on pyyntöjä muuttaa, poistaa tai päivittää tutkimustietojasi, ota yhteyttä register@clinicaltrials.gov. Heti kun muutos on otettu käyttöön osoitteessa clinicaltrials.gov, se päivitetään automaattisesti myös verkkosivustollemme .