En studie for å evaluere sikkerheten, effekten, farmakokinetikken og farmakodynamikken til Emicizumab hos deltakere med mild eller moderat hemofili A uten FVIII-hemmere (HAVEN 6)
5. august 2026 oppdatert av: Hoffmann-La Roche
En multisenter, åpen studie for å evaluere sikkerheten, effekten, farmakokinetikken og farmakodynamikken til Emicizumab hos pasienter med mild eller moderat hemofili A uten FVIII-hemmere
Dette er en multisenter, åpen enarmsstudie designet for å evaluere sikkerheten, effekten, farmakokinetikken og farmakodynamikken til emicizumab hos deltakere med mild eller moderat hemofili A uten hemmere mot faktor VIII (FVIII).
Studieoversikt
Status
Fullført
Forhold
Intervensjon / Behandling
Studietype
Intervensjonell
Registrering (Faktiske)
73
Fase
- Fase 3
Kontakter og plasseringer
Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.
Studiesteder
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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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Alberta
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Edmonton, Alberta, Canada, T6G 1Z1
- Kaye Edmonton Clinic
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Newfoundland and Labrador
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St. John's, Newfoundland and Labrador, Canada, A1B 3V6
- Eastern Health - General Hospital
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California
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Los Angeles, California, Forente stater, 90027
- Childrens Hospital LA
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Georgia
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Atlanta, Georgia, Forente stater, 30308
- Hemophilia of Georgia Center for Bleeding & Clotting Disorders
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Indiana
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Indianapolis, Indiana, Forente stater, 46260
- Indiana Hemophilia & Thrombosis center
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Michigan
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Ann Arbor, Michigan, Forente stater, 48109
- University of Michigan, C.S. Mott Children's Hospital
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Washington
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Seattle, Washington, Forente stater, 98101
- Washington Institute for Coagulation
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Bron, Frankrike, 69677
- Hopital Cardio-vasculaire Louis Pradel
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Le Kremlin-Bicêtre, Frankrike, 94275
- CH de Bicêtre
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Paris, Frankrike, 75015
- Groupe Hospitalier Necker Enfants Malades
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Amsterdam, Nederland, 1105 AZ
- Amsterdam UMC Location AMC
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Warsaw, Polen, 02-776
- Instytut Hematologii i Transfuzjologii
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Madrid, Spania, 28046
- Hospital Universitario La Paz
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Seville, Spania, 41013
- Hospital Universitario Virgen del Rocío
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Cardiff, Storbritannia, CF14 4XW
- Cardiff and Vale NHS Trust
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London, Storbritannia, NW3 2QG
- Royal Free Hospital
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London, Storbritannia, WC1N 3HR
- Great Ormond Street Hospital for Children NHS Foundation Trust
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Johannesburg, Sør-Afrika, 2193
- Charlotte Maxeke Johannesburg Hospital
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Bonn, Tyskland, 53127
- Universitatsklinikum Bonn
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München, Tyskland, 80336
- Klinikum der Universität München, Campus Innenstadt
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Deltakelseskriterier
Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
- Eldre voksen
Tar imot friske frivillige
Nei
Beskrivelse
Inklusjonskriterier:
- Diagnose av mild (FVIII nivå mellom >5 % og
- Vekt ≥3 kilogram (kg)
- Behov for profylakse basert på utreders vurdering
- En negativ test for inhibitor (dvs.
- Ingen dokumentert inhibitor (dvs.
- Dokumentasjon av detaljene for profylaktisk eller episodisk FVIII-behandling og antall blødningsepisoder i minst de siste 24 ukene før innmelding
- Tilstrekkelig hematologisk lever- og nyrefunksjon
- For kvinner i fertil alder: avtale om å forbli avholdende eller bruke prevensjon (som definert i protokollen) under behandlingsperioden og i minst 24 uker etter siste dose av studiemedikamentet
Ekskluderingskriterier:
- Arvelig eller ervervet blødningsforstyrrelse annet enn mild eller moderat medfødt hemofili A
- Historie med ulovlig narkotika- eller alkoholmisbruk innen 48 uker før screening, etter etterforskerens vurdering
- Tidligere (innen de siste 12 månedene) eller nåværende behandling for tromboembolisk sykdom eller tegn på tromboembolisk sykdom
- Andre tilstander som for øyeblikket kan øke risikoen for blødning eller trombose
- Anamnese med klinisk signifikant overfølsomhet assosiert med monoklonale antistoffbehandlinger eller komponenter av emicizumab-injeksjonen
- Planlagt kirurgi under emicizumab ladedosefase (operasjoner hos deltakere på emicizumab fra uke 5 og utover er tillatt)
- Kjent HIV-infeksjon med CD4-teller
- Samtidig sykdom, tilstand, betydelig unormalitet ved screeningsevaluering eller laboratorietester, eller behandling som kan forstyrre gjennomføringen av studien, eller som etter etterforskerens mening vil utgjøre en ytterligere uakseptabel risiko ved administrering av studiemedikamentet til deltakeren
- Mottak av noe av følgende: Et undersøkelseslegemiddel for å behandle eller redusere risikoen for hemofile blødninger innen 5 halveringstider etter siste legemiddeladministrering med unntak av tidligere emicizumab-profylakse; Et ikke-hemofilirelatert undersøkelsesmiddel innen de siste 30 dagene eller 5 halveringstider, avhengig av hva som er kortest; eller ethvert annet undersøkelseslegemiddel som for tiden administreres eller planlegges administrert
- Manglende evne til å overholde studieprotokollen etter etterforskerens mening
- Gravid eller ammer, eller har til hensikt å bli gravid under studien (kvinner i fertil alder må ha et negativt serumgraviditetstestresultat innen 7 dager før oppstart av studiemedikamentet)
Studieplan
Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
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Eksperimentell: Emicizumab
Deltakere med mild og moderat hemofili A uten faktor VIII (FVIII)-hemmere vil bli registrert for å motta emicizumab-startdoseregimet etterfulgt av deltakerens preferanse for ett av 3 vedlikeholdsdoseregimer.
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Fire belastningsdoser av emicizumab 3 milligram per kilo kroppsvekt (mg/kg) vil bli administrert subkutant (SC) en gang i uken (QW) i 4 uker etterfulgt av deltakerens preferanse for ett av de tre følgende vedlikeholds SC doseregimer: 1,5 mg /kg QW, 3 mg/kg en gang hver 2. uke (Q2W), eller 6 mg/kg en gang hver 4. uke (Q4W).
Andre navn:
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Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
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Modellbasert årlig blødningsfrekvens for behandlede blødninger
Tidsramme: Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Antall behandlede blødninger over effektperioden ble estimert som en årlig blødningsrate (ABR) ved bruk av en negativ binomial regresjonsmodell, som tar hensyn til ulike oppfølgingstider.
En behandlet blødning ble definert som en blødning som ble direkte fulgt av en hemofilimedisin som ble rapportert å være en "behandling for blødning".
72-timersregelen ble implementert: to blødninger av samme type og på samme anatomiske plassering ble regnet som én blødning dersom den andre blødningen skjedde innen 72 timer fra siste behandling for den første blødningen.
Blødninger på grunn av operasjon/prosedyre ble utelukket.
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Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Mean Calculated ABR for Treated Bleeds
Tidsramme: 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
Tidsramme: 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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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Gjennomsnittlig beregnet årlig blødningsrate for behandlede spontane blødninger
Tidsramme: Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Antallet behandlede spontane blødninger i løpet av effektperioden presenteres her som en beregnet annualisert blødningsrate (ABR) som ble annualisert for hver deltaker ved å bruke følgende formel: ABR = (antall blødninger/antall dager i effektperioden) x 365,25 .
En behandlet spontan blødning ble definert som en behandlet blødning (blødning direkte etterfulgt av en hemofilimedisin rapportert å være en "behandling for blødning") uten annen kjent medvirkende faktor som traumer eller prosedyre/kirurgi.
72-timersregelen ble implementert: to blødninger av samme type og på samme anatomiske plassering ble regnet som én blødning dersom den andre blødningen skjedde innen 72 timer fra siste behandling for den første blødningen.
Blødninger på grunn av operasjon/prosedyre ble utelukket.
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Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Median beregnet årlig blødningsfrekvens for behandlede spontane blødninger
Tidsramme: Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Antallet behandlede spontane blødninger i løpet av effektperioden presenteres her som en beregnet annualisert blødningsrate (ABR) som ble annualisert for hver deltaker ved å bruke følgende formel: ABR = (antall blødninger/antall dager i effektperioden) x 365,25 .
En behandlet spontan blødning ble definert som en behandlet blødning (blødning direkte etterfulgt av en hemofilimedisin rapportert å være en "behandling for blødning") uten annen kjent medvirkende faktor som traumer eller prosedyre/kirurgi.
72-timersregelen ble implementert: to blødninger av samme type og på samme anatomiske plassering ble regnet som én blødning dersom den andre blødningen skjedde innen 72 timer fra siste behandling for den første blødningen.
Blødninger på grunn av operasjon/prosedyre ble utelukket.
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Fra dagen for første emicizumab-dose til minst 52 uker med emicizumab-behandling (median [område, min-maks] effektperiode: 55,64 [8,6-89,9] uker)
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Prosentandel av deltakere som foretrekker Emicizumab SC-behandling, deres tidligere hemofili IV-behandling eller ikke har noen preferanse, vurdert ved bruk av Emicizumab Preference Survey ved uke 17
Tidsramme: Uke 17
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Emicizumab Preference Survey er et egnet spørreskjema utviklet av sponsoren for å registrere deltakerens preferanse for behandling med subkutan (SC) emicizumab, intravenøs (IV) faktor VIIII (FVIII), eller ingen preferanse.
Konfidensintervallene på 95 % ble beregnet ved bruk av Pearson-Clopper-metoden.
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Uke 17
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Prosentandel av omsorgspersoner som foretrekker Emicizumab SC-behandling, barnets tidligere hemofili IV-behandling eller ikke har noen preferanse, vurdert ved bruk av Emicizumab Preference Survey ved uke 17
Tidsramme: Uke 17
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Emicizumab Preference Survey er et egnet spørreskjema utviklet av sponsoren for å registrere omsorgspersonens preferanse for barnets behandling med subkutan (SC) emicizumab, intravenøs (IV) faktor VIIII (FVIII), eller ingen preferanse.
Konfidensintervallene på 95 % ble beregnet ved bruk av Pearson-Clopper-metoden.
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Uke 17
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Endring fra baseline i gjennomsnittlig daglig toppaktivitetsvarighet over tid
Tidsramme: Baseline (uke 1-2) og uke 13 (uke 12-13 periode), 25 (uke 24-25 periode), 37 (uke 36-37 periode) og 49 (uke 48-49 periode)
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For vurdering av fysisk aktivitet ble deltakere ≥5 år instruert til å bære studiens akselerometriapparat på håndleddet kontinuerlig (24 timer/dag) hver dag i de angitte 2-ukers periodene i løpet av studien.
En deltaker ble ansett for å være i overensstemmelse med vurderingene av fysisk aktivitet hvis de brukte studieenheten kontinuerlig (≥8 timer/dag) hver dag i minst 8 dager i hver av de angitte 2-ukers periodene i løpet av studien.
Hvis dette samsvarskriteriet ikke ble nådd på et spesifikt tidspunkt, ble ikke deltakeren inkludert i analysen av de angitte 2-ukers periodene der samsvar ikke ble nådd.
Daglige målinger ble beregnet i gjennomsnitt over 14-dagers tidspunkt.
Aktivitetstelling var et mål på akselerasjonen målt av enheten.
Den daglige toppaktivitetsvarigheten ble definert som summen av moderat til kraftig aktivitet per dag (i minutter).
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Baseline (uke 1-2) og uke 13 (uke 12-13 periode), 25 (uke 24-25 periode), 37 (uke 36-37 periode) og 49 (uke 48-49 periode)
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Endring fra baseline i gjennomsnittlig daglig trinntelling over tid
Tidsramme: Baseline (uke 1–2) og uke 13 (uke 12–13), 25 (uke 24–25), 37 (uke 36–37) og 49 (uke 48–49)
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For vurdering av fysisk aktivitet ble deltakere ≥5 år instruert til å bære studiens akselerometriapparat på håndleddet kontinuerlig (24 timer/dag) hver dag i de angitte 2-ukers periodene i løpet av studien.
En deltaker ble ansett for å være i overensstemmelse med vurderingene av fysisk aktivitet hvis de brukte studieenheten kontinuerlig (≥8 timer/dag) hver dag i minst 8 dager i hver av de angitte 2-ukers periodene i løpet av studien.
Hvis dette samsvarskriteriet ikke ble nådd på et spesifikt tidspunkt, ble ikke deltakeren inkludert i analysen av de angitte 2-ukers periodene der samsvar ikke ble nådd.
Daglige målinger ble beregnet i gjennomsnitt over 14-dagers tidspunkt.
Aktivitetstelling var et mål på akselerasjonen målt av enheten.
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Baseline (uke 1–2) og uke 13 (uke 12–13), 25 (uke 24–25), 37 (uke 36–37) og 49 (uke 48–49)
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Model-Based ABR for All Bleeds
Tidsramme: 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 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)
|
|
Mean Calculated ABR for All Bleeds
Tidsramme: 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.
|
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 All Bleeds
Tidsramme: 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
Tidsramme: 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 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)
|
|
Mean Calculated ABR for Treated Joint Bleeds
Tidsramme: 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)
|
|
Median Calculated ABR for Treated Joint Bleeds
Tidsramme: 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)
|
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Model-Based ABR for Treated Target Joint Bleeds
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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.
|
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 on the Average Over Time
Tidsramme: 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)
|
|
CATCH Questionnaire for Pediatric Participants: Change From Baseline in the Daily Activity RP and Impact Domain Score Over Time
Tidsramme: 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, 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.
|
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 Pediatric Participants: Change From Baseline in the Social Activity Risk Perception and Impact Domain Score Over Time
Tidsramme: 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, 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.
|
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 Pediatric Participants: Change From Baseline in the Recreational Activity RP and Impact Domain Score Over Time
Tidsramme: 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.
|
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 Pediatric Participants: Change From Baseline in the School Impact Domain Score Over Time
Tidsramme: 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.
|
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 Pediatric Participants: Change From Baseline in the Preoccupation Domain Score Over Time
Tidsramme: 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.
|
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 Pediatric Participants: Change From Baseline in the Treatment Burden Domain Score Over Time
Tidsramme: 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.
|
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 Pediatric Participants: Number of Participants by Responses to Their Level of Hurt Associated With a Bleed Over Time
Tidsramme: Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
|
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.
|
Baseline, Weeks 13, 25, 37, 49, 61, 85, 97, 109, 121, 133, 145 and 157
|
|
CATCH Questionnaire for Pediatric Participants: Number of Participants by Responses to Their Level of Hurt at Its Worst Over Time
Tidsramme: 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)
|
|
CATCH Questionnaire for Caregivers: Change From Baseline in the Preoccupation Domain Score Over Time
Tidsramme: 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, 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)
|
|
CATCH Questionnaire for Caregivers: Change From Baseline in the Treatment Burden Domain Score Over Time
Tidsramme: 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.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
Change From Baseline in Hemophilia Joint Health Scores Over Time
Tidsramme: Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
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.
|
Baseline, Weeks 13, 25, 37, 49, 61, 73, 85, 97, 109,121, 133, 145, 157, 169 and SC/ED (up to approximately 274 weeks)
|
|
Menstrual Bleed Questionnaire (MBQ) for Female Participants of Childbearing Potential: Change From Baseline in the MBQ Total Score Over Time
Tidsramme: 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
|
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.
|
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
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Heaviness Subscale Score Over Time
Tidsramme: 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)
|
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.
|
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)
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the QoL Subscale Score Over Time
Tidsramme: 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.
|
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
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Irregularity Subscale Score Over Time
Tidsramme: 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.
|
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
|
|
MBQ for Female Participants of Childbearing Potential: Change From Baseline in the Pain Subscale Score Over Time
Tidsramme: 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
|
|
Menstruation Diary With the Pictorial Blood Assessment Chart (PBAC) for Female Participants of Childbearing Potential: PBAC Scores Over Time
Tidsramme: Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
|
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).
|
Baseline and monthly (on days of menstruation) until Study Completion (up to approximately 247 weeks)
|
|
Number of Participants With at Least One Adverse Event by Severity, According to the World Health Organization (WHO) Toxicity Grading Scale
Tidsramme: 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.
As per WHO toxicity grading, Grade 1= mild, 2= moderate, 3=severe, 4=life threatening.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With Adverse Events Leading to Study Drug Discontinuation
Tidsramme: 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.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Thromboembolic Event
Tidsramme: 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.
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).
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Event of Thrombotic Microangiopathy
Tidsramme: 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.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Injection-Site Reaction by Severity, According to the WHO Toxicity Grading Scale
Tidsramme: 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.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
Number of Participants With at Least One Severe Hypersensitivity, Anaphylaxis, and Anaphylactoid Event
Tidsramme: 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.
|
From enrollment until final completion date cutoff (median [range, min-max] observation period: 156.21 [24.1 - 274.0] weeks)
|
|
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
Tidsramme: 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.
|
Up to approximately 274 weeks
|
|
Number of Participants With at Least One Adverse Events of Changes From Baseline in Vital Signs
Tidsramme: 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.
|
Up to approximately 274 weeks
|
|
Change From Baseline in Electrocardiogram (ECG) Parameters Over Time: QT, QTcB, QTcF, RR, PR, and QRS Intervals
Tidsramme: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
|
|
Change From Baseline in Heart Rate Over Time, as Measured by ECG
Tidsramme: Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
Baseline, Weeks 5, 25, 49, 61, 73, 85, 97, 109, 121, 133, 145, 157, 169, 181, SC/ED (up to approximately 274 weeks)
|
|
|
Plasma Trough Concentration (Ctrough) of Emicizumab Over Time
Tidsramme: 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)
|
|
|
Number of Participants With Anti-Drug Antibodies Against Emicizumab at Baseline and Post-Baseline
Tidsramme: 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.
|
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)
|
|
Number of Participants Who Develop Anti-FVIII Inhibitors Over Time
Tidsramme: 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.
|
Up to approximately 274 weeks
|
Samarbeidspartnere og etterforskere
Det er her du vil finne personer og organisasjoner som er involvert i denne studien.
Sponsor
Etterforskere
- Studieleder: Clinical Trials, Hoffmann-La Roche
Publikasjoner og nyttige lenker
Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.
Studierekorddatoer
Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.
Studer hoveddatoer
Studiestart (Faktiske)
10. februar 2020
Primær fullføring (Faktiske)
30. oktober 2021
Studiet fullført (Faktiske)
19. desember 2025
Datoer for studieregistrering
Først innsendt
7. november 2019
Først innsendt som oppfylte QC-kriteriene
7. november 2019
Først lagt ut (Faktiske)
12. november 2019
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
27. august 2026
Siste oppdatering sendt inn som oppfylte QC-kriteriene
5. august 2026
Sist bekreftet
1. august 2026
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- BO41423
- 2019 (U.S. NIH-stipend/kontrakt: Chief Medical Office (CMO) Alberta Health Services)
- 2023 (U.S. NIH-stipend/kontrakt: GRAMMY Museum Foundation)
- 2019-002179-32 (EudraCT-nummer)
- 2023-506610-52-00 (Registeridentifikator: EU CT Number)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
JA
IPD-planbeskrivelse
Kvalifiserte forskere kan be om tilgang til individuelle pasientnivådata gjennom forespørselsplattformen (www.vivli.org). Ytterligere detaljer om Roches kriterier for kvalifiserte studier er tilgjengelig her (https://vivli.org/ourmember/roche/).
For ytterligere detaljer om Roches globale retningslinjer for deling av informasjon om kliniske studier og hvordan du ber om tilgang til relaterte kliniske studiedokumenter, se her (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Ja
Studerer et amerikansk FDA-regulert enhetsprodukt
Nei
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .