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Tutkimus tehosta ja turvallisuudesta PF-06741086 aikuisilla ja teini-ikäisillä potilailla, joilla on vaikea hemofilia A tai kohtalaisen vaikea tai vaikea hemofilia B

torstai 23. heinäkuuta 2026 päivittänyt: Pfizer

Avoin tutkimus nuorten ja aikuisten vaikeasta (koagulaatiotekijäaktiivisuus

PF-06741086-hoidon odotetaan osoittavan kliinisesti merkittävän edun ja/tai merkittävän panoksen potilaiden hoitoon verrattuna nykyisiin hemofilia A tai B hoitomenetelmiin, koska se toimii eri tavalla kuin tekijänkorvaustuotteet ja toimii estäjien läsnä ollessa. . Mahdollisuus antaa kerran viikossa (QW) ihonalaisesti (SC) tarjoaa hoitovaihtoehtoja luotettavan verisuonen pääsyn puuttuessa, lisää käyttömukavuutta ja saattaa mahdollistaa paremman hoitomyöntymisen. Yhdessä näiden ominaisuuksien pitäisi vähentää verenvuotojaksoja.

Tutkimuksen yleiskatsaus

Tila

Valmis

Ehdot

Interventio / Hoito

Opintotyyppi

Interventio

Ilmoittautuminen (Todellinen)

188

Vaihe

  • Vaihe 3

Yhteystiedot ja paikat

Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.

Opiskelupaikat

  • Bulgaria
      • Sofia, Bulgaria, 1756
        • National Specialized Hospital for the Active Treatment of Hematological Diseases - EAD, Sofia
      • Stara Zagora, Bulgaria, 6003
        • UMHAT "Prof.Dr. Stoyan Kirkovich"
  • Espanja
      • A Coruña, Espanja, 15006
        • Hospital Universitario A Coruna
      • Barcelona, Espanja, 08035
        • Hospital Universitario Vall d´Hebron
      • Madrid, Espanja, 28046
        • Hospital Universitario La Paz
      • Salamanca, Espanja, 37007
        • Hospital Universitario De Salamanca
      • Zaragoza, Espanja, 50009
        • Hospital Universitario Miguel Servet
  • Etelä -Korea
      • Daegu, Etelä -Korea, 41944
        • Kyungpook National University Hospital
      • Seoul, Etelä -Korea, 03722
        • Severance Hospital, Yonsei University Health System
      • Seoul, Etelä -Korea, 05278
        • Kyung Hee University Hospital at Gangdong
  • Etelä-Afrikka
    • Gauteng
      • Johannesburg, Gauteng, Etelä-Afrikka, 2193
        • Charlotte Maxeke Johannesburg Academic Hospital
  • Hong Kong
      • Hong Kong, Hong Kong
        • Queen Mary Hospital
      • Hong Kong, Hong Kong
        • Prince of Wales Hospital
  • Intia
    • Gujarat
      • Surat, Gujarat, Intia, 395002
        • Nirmal Hospital Pvt, Ltd
    • Maharashtra
      • Pune, Maharashtra, Intia, 411004
        • Sahyadri Super Speciality Hospital
      • Pune, Maharashtra, Intia, 411004
        • Sahyadri Clinical Research And Development Center
    • Tamil Nadu
      • Vellore, Tamil Nadu, Intia, 632004
        • Christian Medical College
  • Italia
      • Perugia, Italia, 06156
        • Università degli Studi di Perugia, Azienda Ospedaliera di Perugia, Ospedale Santa Maria della
    • Milan
      • Milan, Milan, Italia, 20122
        • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
    • RM
      • Roma, RM, Italia, 00161
        • Università degli Studi di Roma "Sapienza"-Policlinico Umberto I
  • Japani
      • Hiroshima, Japani, 734-8551
        • Hiroshima University Hospital
    • Aichi-ken
      • Nagoya, Aichi-ken, Japani, 466-8560
        • Nagoya University Hospital - Transfusion Medicine
    • Hokkaido
      • Sapporo, Hokkaido, Japani, 004-0041
        • Sapporo Tokushukai Hospital
    • Saitama
      • Iruma-gun, Saitama, Japani, 350-0495
        • Saitama Medical University Hospital
  • Kanada
    • Ontario
      • Hamilton, Ontario, Kanada, L8S 4K1
        • McMaster University
      • Hamilton, Ontario, Kanada, L8S 4K1
        • McMaster Children's Hospital
      • Hamilton, Ontario, Kanada, L8N 3Z5
        • McMaster University Medical Centre - Hamilton Health Sciences
      • Toronto, Ontario, Kanada, M5G 1X8
        • The Hospital for Sick Children
  • Kiina
      • Beijing, Kiina, 100045
        • Beijing Children's Hospital, Capital Medical University
    • Guangdong
      • Guangzhou, Guangdong, Kiina, 510515
        • Nanfang Hospital, Southern Medical University
    • Guizhou
      • Guiyang, Guizhou, Kiina, 550004
        • The Affiliated Hospital of Guizhou Medical University
    • Jiangxi
      • Nanchang, Jiangxi, Kiina, 306113
        • Jiangxi Provincial People's Hospital
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, Kiina, 300020
        • Institute of Hematology, Chinese Academy of Medical Sciences
  • Kroatia
      • Zagreb, Kroatia, 10000
        • Klinicki bolnicki centar Zagreb
  • Meksiko
    • Nuevo León
      • Monterrey, Nuevo León, Meksiko, 64460
        • Hospital Universitario "Dr Jose Eleuterio Gonzalez"
    • Yucatán
      • Mérida, Yucatán, Meksiko, 97130
        • Centro Multidisciplinario para el Desarrollo Especializado de la Inv. Clínica en Yucatán, S.C.P.
  • Oman
      • Muscat, Oman, 123
        • Sultan Qaboos University Hospital
  • Ranska
      • Paris, Ranska, 75015
        • Hôpital Necker Enfants malades
  • Saudi-Arabia
      • Jeddah, Saudi-Arabia, 21589
        • King AbdulAziz University Hospital
      • Riyadh, Saudi-Arabia
        • King Faisal Specialist Hospital & Research Center
  • Serbia
      • Belgrade, Serbia, 11000
        • Clinical Center of Serbia
      • Belgrade, Serbia, 11000
        • Institute for Mother and Child healthcare "Dr Vukan Cupic"
      • Kragujevac, Serbia, 34000
        • Clinical Center Kragujevac
      • Niš, Serbia, 18000
        • Clinical Center Niš
  • Taiwan
      • Taichung, Taiwan, 40705
        • Taichung Veterans General Hospital
    • Changhua County
      • Changhua, Changhua County, Taiwan, 500
        • Changhua Christian Hospital
  • Turkki (Türkiye)
      • Adana, Turkki (Türkiye), 01130
        • Acibadem Adana Hospital
      • Ankara, Turkki (Türkiye), 06230
        • Hacettepe University Medical Faculty
      • Ankara, Turkki (Türkiye), 06500
        • Gazi University Health Research and Practice Center Gazi Hospital
      • Antalya, Turkki (Türkiye), 07060
        • Akdeniz University Medical Faculty
      • Gaziantep, Turkki (Türkiye), 27310
        • Gaziantep University Sahinbey Research and Training Hospital
      • Istanbul, Turkki (Türkiye), 34093
        • Istanbul University Oncology Institute
      • Izmir, Turkki (Türkiye), 35040
        • Ege University Medical Faculty
      • Izmir, Turkki (Türkiye), 35210
        • Dr. Behcet Uz Child Diseases Surgery Education and Research Hospital
      • Kayseri, Turkki (Türkiye), 38039
        • Erciyes University Medical Faculty
      • Samsun, Turkki (Türkiye), 55280
        • Ondokuz Mayıs University Medical Faculty
      • Trabzon, Turkki (Türkiye), 61080
        • Karadeniz Technical University Medical Faculty
  • Venäjä
      • Samara, Venäjä, 443079
        • FGBOU VO "Samara State Medical University" of MoH of Russia
  • Yhdysvallat
    • Florida
      • Tampa, Florida, Yhdysvallat, 33612
        • USF Health Morsani Center for Advanced Healthcare
    • Iowa
      • Iowa City, Iowa, Yhdysvallat, 52242
        • University of Iowa
    • New York
      • New Hyde Park, New York, Yhdysvallat, 11040
        • Northwell Health HTC
    • Washington
      • Seattle, Washington, Yhdysvallat, 98101
        • Washington Institute for Coagulation

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

12 vuotta - 74 vuotta (Lapsi, Aikuinen, Vanhempi Aikuinen)

Hyväksyy terveitä vapaaehtoisia

Ei

Kuvaus

Sisällyttämiskriteerit

  • Osallistujat, joilla on diagnosoitu vaikea hemofilia A tai kohtalaisen vaikea tai vaikea hemofilia B ja joiden paino on seulonnassa vähintään 35 kg.
  • Osallistuja tai laillisesti valtuutettu edustaja tai osallistujan hoitaja, joka pystyy antamaan allekirjoitetun tietoon perustuvan suostumuksen (tai vähäisen suostumuksen, jos sovellettavissa).

Osallistujien, jotka ovat ilmoittautuneet ei-inhibiittoreiden kohorttiin, on myös täytettävä seuraavat kriteerit:

  • Ei havaittavaa tai dokumentoitua estäjien historiaa
  • FVIII/FIX-rutiiniprofylaksia suorittavat osallistujat, jotka ovat osoittaneet vähintään 80-prosenttisesti noudattavan suunniteltua ennaltaehkäisyohjelmaa 6 kuukauden aikana ennen ilmoittautumista ja jotka ovat valmiita jatkamaan rutiininomaista profylaksihoitoa FVIII/FIX-korvaushoidolla tarkkailuvaiheen aikana.
  • Osallistujat, joilla on tarpeen mukaan annettava hoito, jossa oli vähintään 6 akuuttia verenvuotojaksoa (spontaani tai traumaattinen), jotka vaativat hyytymistekijäinfuusiota 6 kuukauden aikana ennen ilmoittautumista ja jotka olivat valmiita jatkamaan tarpeen mukaan annettavaa hoitoa tarkkailuvaiheen aikana.

Inhibiittorikohorttiin ilmoittautuneiden osallistujien on myös täytettävä seuraavat kriteerit:

  • Dokumentaatio nykyisestä korkean tiitterin estäjän (≥ 5 BU/ml) tai nykyisen matalatiitteriisen estäjän (< 5 BU/ml), joka on vastustuskykyinen FVIII- tai FIX-korvaukselle ja FVIII- tai FIX-palautus <60 % odotetusta edellisen 6 kuukauden aikana ennen ilmoittautumista havainnointivaihe
  • Hemofilia A -osallistujat, joilla on tarpeen mukaan hoito-ohjelma, jossa on ≥6 verenvuotojaksoa, tai hemofilia B -potilaat, joilla on vähintään 4 verenvuotojaksoa (spontaani tai traumaattinen), jotka vaativat ohitustekijähoitoa 6 kuukauden aikana ennen havaintovaiheeseen ilmoittautumista ja jotka ovat valmiita jatkamaan hoitoa - vaativat hoitoa havaintovaiheen aikana.
  • Osallistujat, joilla on dokumentoitu estäjiä tekijäkorvaushoidon aikana, mutta jotka eivät täytä edellisessä kohdassa kuvattuja kvantitatiivisia inhibiittorikriteerejä seulonnan aikana (esim. osallistuja, jolla on aiemmin dokumentoitu korkeatiitteriinen inhibiittori (≥5 BU/ml) ja joiden tila estää uudelleen haastamisen FVIII- tai FIX-korvauksella) voidaan harkita kelpoisuutta tapauskohtaisesti Pfizer Medical Monitorin etukäteen antamalla suostumuksella.
  • Osallistujat, jotka täyttävät yllä mainitut verenvuotokriteerit ja jotka ovat rutiininomaisessa profylaksiassa (määritelty hoitona suonensisäisellä ohitustekijän injektiolla verenvuodon estämiseksi) ja jotka ovat osoittaneet vähintään 80-prosenttisesti noudattavan suunniteltua ennaltaehkäisyohjelmaa 6 kuukauden aikana ennen ilmoittautumista, voidaan saada. kelpoisuutta harkitaan tapauskohtaisesti Pfizerin lääketieteellisen monitorin kanssa keskustelun ja suostumuksen perusteella.

Poissulkemiskriteerit

  • Sepelvaltimotaudin, laskimo- tai valtimotromboosin tai iskeemisen sairauden aiempi tai nykyinen hoito ja/tai aiempi hoito
  • Tunnettu suunniteltu kirurginen toimenpide suunnitellulla tutkimusjaksolla.
  • Muu tunnettu hemostaattinen vika kuin hemofilia A tai B.
  • Epänormaali munuaisten tai maksan toiminta
  • Nykyinen epävakaa maksa- tai sappisairaus
  • Epänormaalit hematologiset parametrit
  • Muu akuutti tai krooninen lääketieteellinen tai psykiatrinen tila tai laboratoriopoikkeavuus, joka voi lisätä tutkimukseen osallistumiseen tai tutkimustuotteen antamiseen liittyvää riskiä tai häiritä tutkimustulosten tulkintaa ja tutkijan harkinnan mukaan
  • Nykyinen rutiiniprofylaksia ohitusaineella tai ei-hyytymistekijäkorvaushoidolla tai mikä tahansa aikaisempi hoito hemofilian hoitoon tarkoitetulla geeniterapiatuotteella (osallistujat, joita on hoidettu ennaltaehkäisyllä ohitusaineilla tai jotka ovat aiemmin saaneet hoitoa ei-tekijävalmisteilla harkitaan tapauskohtaisesti).

  • Säännöllinen, samanaikainen hoito immunomoduloivilla lääkkeillä

    - Jatkuva tai suunniteltu immuunitoleranssin induktion käyttö tarkkailuvaiheen tai aktiivisen hoitovaiheen aikana tai profylaksi FVIII- tai FIX-korvauksella milloin tahansa hoidon aloittamisen jälkeen tutkimusinterventiolla aktiivisen hoitovaiheen aikana.

  • Aiempi altistuminen PF 06741086:lle osallistuessaan tutkimuksiin B7841002 ja B7841003.
  • Osallistuminen muihin tutkimuksiin, joihin liittyy tutkimuslääke(itä) tai tutkimusrokotteita 30 päivän (tai paikallisten vaatimusten mukaisesti) tai 5 puoliintumisajan kuluessa ennen tutkimukseen osallistumista ja/tai tutkimukseen osallistumisen aikana.
  • CD4-solujen määrä ≤200/uL, jos ihmisen immuunikatovirus (HIV) -positiivinen
  • Seulonta-EKG, joka osoittaa kliinisesti merkittäviä poikkeavuuksia, jotka voivat vaikuttaa osallistujien turvallisuuteen tai tutkimustulosten tulkintaan.
  • Henkilöt, joilla on yliherkkyys tai allerginen reaktio hamsterin proteiinille tai muille tutkimusintervention komponenteille.
  • Tutkimuspaikan henkilöstön jäsenet, jotka ovat suoraan mukana tutkimuksen suorittamisessa, ja heidän perheenjäsenensä, tutkijan muutoin valvomat toimipaikan työntekijät tai osallistujat, jotka ovat Pfizerin työntekijöitä, mukaan lukien heidän perheenjäsenensä, jotka osallistuvat suoraan tutkimuksen suorittamiseen.

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: Crossover-tehtävä
  • Naamiointi: Ei mitään (avoin tarra)

Aseet ja interventiot

Osallistujaryhmä / Arm
Interventio / Hoito
Kokeellinen: PF-06741086
Osallistujat määrätään hoitoon PF-06741086:lla 6 kuukauden tarkkailuvaiheen jälkeen heidän nykyisen hemofiliahoitonsa mukaisesti.
Lääke: PF-06741086
300 milligrammaa (mg) ihonalainen (sc) latausannos, jota seuraa 150 mg neliömetriä kerran viikossa (qw). 300 mg sc qw määrätään osallistujille, jotka täyttävät annoksen korotuskriteerit.

Mitä tutkimuksessa mitataan?

Ensisijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP)
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With Prior OD Therapy at OP)
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Treated Bleeding Events: Non-Inhibitor Cohort (Participants With RP at OP)
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR is defined as number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or OD medication). A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Number of Participants With Adverse Events (AEs): Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. AEs included both serious AEs and all other AEs.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Serious Adverse Events (SAEs): Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
AE: any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE: any untoward medical occurrence at any dose that resulted in any of the following outcomes: death; life-threatening; required inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect; or that was considered as an important medical event.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Events: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Thrombotic events are when a blood clot (thrombus) forms in a blood vessel in the arm, leg, lung, or head and can be life-threatening. A thrombus can occur in veins (venous thrombosis) or arteries (arterial thrombosis).
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Thrombotic Microangiopathy: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Thrombotic microangiopathy: pathological state where micro-vessels are occluded by platelet rich thrombi leading to thrombocytopenia (low platelets) and microangiopathic haemolytic anaemia (red blood cell destruction) and potential end organ damage.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Disseminated Intravascular Coagulation/ Consumption Coagulopathy: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Disseminated intravascular coagulation is characterized by widespread clotting in small blood vessels, and consumption of platelets and clotting factors by the clots, leading to bleeding.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Anti-drug Antibodies (ADA) and Neutralizing Antibodies (NAb) Against PF- 06741086: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: During prophylaxis treatment in ATP (12 months)
ADA positive: A participant with >=1 treatment induced or treatment-boosted ADA response. NAb positive: An ADA positive participant with >=1 treatment induced or treatment boosted NAb response.
During prophylaxis treatment in ATP (12 months)
Number of Participants With Transient and Persistent ADA and NAb Against PF-06741086: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: During prophylaxis treatment in ATP (12 months)
Persistent ADA: participant with treatment-induced or treatment-boosted ADA detected at 2 or more times during treatment including any follow-up, where first and last ADA positive samples separated by >=16 weeks. Transient ADA: treatment-induced or treatment-boosted ADA detected at only 1 time during treatment or follow-up. Persistent NAb: NAb-positive participant with first and last positive NAb samples detected >=16 weeks posttreatment, irrespective of any negative samples in between. Transient NAb: NAb-positive participant with (1) treatment-induced/treatment-boosted NAb sample detected at only 1 time posttreatment or (2) treatment-induced/treatment-boosted NAb samples detected at 2 or more times where first and last positive samples separated by <16 weeks, and participant's last sample was NAb or ADA negative.
During prophylaxis treatment in ATP (12 months)
Number of Participants With Injection Site Reactions (ISRs): Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: During prophylaxis treatment in ATP (12 months)
ISR included: injection site haematoma, injection site pain, injection site bruising, injection site erythema, injection site induration, injection site oedema, Injection site pruritus and Injection site swelling. ISR graded according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 where grade 1: tenderness with or without associated symptoms (warmth, erythema, itching), grade 2: pain, lipodystrophy, edema, phlebitis, grade 3: ulceration or necrosis, severe tissue damage, operative intervention indicated, grade 4: life-threatening consequences, urgent intervention indicated and grade 5: death. In this outcome measure only categories with non-zero values reported.
During prophylaxis treatment in ATP (12 months)
Number of Participants With Clinically Significant Changes in Physical Examinations: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Physical examination included assessments of the head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. Height and weight were also measured and recorded. Clinical significance in physical examinations was determined by investigator.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Changes in Vital Signs: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Vital signs included temperature, pulse rate, respiratory rate, and blood pressure. Blood pressure and pulse rate were measured in a supine position using completely automated device after at least 5 minutes of rest for the participant in a quiet setting without distractions. Respiratory rate was measured by observing and counting the respirations of the participant for 30 seconds and multiplied by 2. Clinical significance of vital signs was determined by investigator.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Hematology included: hemoglobin; leukocytes; neutrophils; and platelets. Chemistry included: potassium; aspartate aminotransferase; creatinine; alkaline phosphatase; total bilirubin; albumin; calcium corrected, estimated decreased; sodium; and calcium. Clinical significance of laboratory parameters was determined by investigator.
OP reporting group: Up to 6 months; ATP reporting group: up to 13 months (includes 28-35 days of safety follow-up post last dose of study drug)
Number of Participants With Severe/ Systematic Hypersensitivity and Anaphylactic Reactions: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: During prophylaxis treatment in ATP (12 months)
Systemic hypersensitivity is a complex immune response where the immune system reacts excessively to an antigen, often leading to severe allergic reactions, including widespread symptoms (which may affect multiple organs), such as rash, swelling of your face, lips, mouth, or tongue, trouble breathing, wheezing, dizziness, fainting, fast heartbeat, pounding in your chest or sweating. Anaphylaxis is a severe, potentially fatal, systemic allergic reaction that occurs suddenly after contact with an allergy causing substance.
During prophylaxis treatment in ATP (12 months)

Toissijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Model-Based ABR of Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of the following: pain or an unusual sensation in the joint, palpable swelling, and warmth of the skin over the joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Spontaneous Bleeds: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR: number of bleeding episodes per year. ABR was calculated as the number of bleeds requiring treatments/ (days on treatment period/365.25). If a participant did not complete a treatment period, the days on treatment ended at the last dosing date + 6 days. Spontaneous bleed: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Target Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete a treatment period, days on treatment ended at last dosing date + 6 days. Target joint: a major joint into which repeated bleeds occurred. Joint bleed: bleeding episode characterized by rapid loss of range of motion as compared with baseline that was associated with any combination of following: pain or unusual sensation in joint, palpable swelling, and warmth of the skin over joint. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Model-Based ABR of Total Bleeds: Inhibitor and Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
ABR: number of bleeding episodes per year. ABR was calculated as number of bleeds requiring treatments/ (days on treatment period/365.25). If participant did not complete treatment period, days on treatment ended at last dosing date + 6 days. Treated bleed: If a bleed was treated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication). Untreated bleed: If a bleed was untreated with factor replacement or bypass agents within 48 hours of start of bleeding, regardless of the type of treatment (preventive, prophylaxis or on-demand medication), it was considered as an untreated bleed. Total bleeds: Treated bleeds + untreated bleeds. A model-based ABR using a repeated measures negative binomial model is reported for this outcome measure.
OP reporting group: Up to 6 months; ATP reporting group: up to 12 months
Change From Baseline in Hemophilia Joint Health Score (HJHS) at Month 6: Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling [0-3], duration of swelling [0-1], muscle atrophy [0-2], crepitus on motion [0-2], flexion loss [0-3], extension loss [0-3], joint pain [0-2], and strength [0-4]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HJHS at Month 6: Inhibitor Cohort
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
HJHS evaluated total joint score for 6 joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) and the global gait score. Joint total score per joint ranged from 0 to 20 (evaluated as: swelling [0-3], duration of swelling [0-1], muscle atrophy [0-2], crepitus on motion [0-2], flexion loss [0-3], extension loss [0-3], joint pain [0-2], and strength [0-4]). Global gait score ranged from 0 to 4 based on walking, stairs, running, and hopping on 1 leg. HJHS total score was sum of total joint score for 6 joints (0 to 120) and global gait score (0 to 4). The total HJHS score ranged from 0 to 124, where higher scores indicated worse joint health.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) Total Score and Physical Health Domain at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haem-A-QoL Total Score and Physical Health Domain at Month 6: Inhibitor Cohort, Participants >=17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Haem-A-QoL assessed health-related quality of life (QoL) in adult participants (>=17 years of age) with hemophilia. It contained 46 items with 10 domains that assessed health in the following areas: physical health; feelings; view of self; sports and leisure; work and school; dealing with haemophilia; treatment; future; family planning; partnership and sexuality. All items were based on a 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then each domain score was rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A-QoL score was averaged across the 46 items values and then rescaled from 0 to 100, lower Haem-A-QoL scores signified better QoL.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Quality of Life Questionnaire for Children (Haemo-QoL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Haemo-QoL assessed health-related QoL in adolescent participants (12 to <17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Haemo-QoL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Haemo-QoL assessed health-related QoL in adolescent participants (12 to <17 years of age) with hemophilia. It contains 12 items with 77 domains that assesses health in the following areas: physical health; feelings; attitude/view; family; friends; other people; sport and school; coping/dealing; treatment; perceived support; future and relationship. All items were based on 5-point Likert type scale (1= never, 2= rarely, 3= sometimes, 4= often, 5= all the time). Scoring was performed by averaging non-missing item responses for each domain, then rescaled from 0 to 100, lower scores signified higher QoL. Total Haem-A QoL score was averaged across the 77 items values and then rescaled from 0 to 100, lower Haemo-QoL scores signified better QoL.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Hemophilia Activities List (HAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants >=17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in HAL Total Score at Month 6: Inhibitor Cohort, Participants >=17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults (>=17 years of age). The 7 domains of this instrument contained 42 items in total, as follows: lying/sitting/kneeling/standing; lower (leg) functioning; upper (arm) functioning; transportation; self-care; household tasks; and sports/leisure. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. HAL total score was sum of all items and scored 42 to 252, which were transformed to 0 - 100, where higher scores indicated better functional status.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in Pediatric Hemophilia Activities List (pedHAL) Total Score at Month 6: Non-Inhibitor Cohort, Participants 12 to <17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to <17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in pedHAL Total Score at Month 6: Inhibitor Cohort, Participants 12 to <17 Years
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
The pedHAL was a multiple domain measure of the impact of hemophilia on functional abilities in adolescents (12 to <17 years of age). The 7 domains of this instrument contained 53 items in total, as follows: sitting/kneeling/standing; functions of the legs; functions of the arms; use of transportation; self-care; household tasks; leisure activities and sports. Items were rated on 6-point scale 1 (impossible) to 6 (never) that described difficulty due to hemophilia. pedHAL total score was normalized in a range of 0 - 100, where higher scores indicated better functional status.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Patient Global Impression of Change-Hemophilia (PGIC-H) at Month 6: Non-Inhibitor Cohort
Aikaikkuna: Month 6
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Month 6
PGIC-H at Month 6: Inhibitor Cohort
Aikaikkuna: Month 6
The PGIC-H was a single item assessment of the participant's overall impression of change in their life with hemophilia, on a 7-point scale (1= greatly improved to 7= greatly worsened, where lower scores indicated better improvement).
Month 6
Change From Baseline in EuroQol 5 Dimensions 5 Level (EQ-5D-5L) Index and VAS Scores at Month 6: Non-Inhibitor Cohort
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
Change From Baseline in EQ-5D-5L Index and VAS Scores at Month 6: Inhibitor Cohort
Aikaikkuna: OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6
EQ-5D-5L consisted of participant completed questionnaire with 2 components: health state profile index and visual analogue scale (VAS). EQ-5D health state profile index has 5 domains (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), with 5 levels (1= no problems, 2= slight problems, 3= moderate problems, 4= severe problems, 5= extreme problems). Responses to 5 dimensions comprised health state index value. E.g. if a participant responds "no problems" for each 5 dimensions, then health state was coded as "11111" with predefined index value to it. EQ-5D-5L index score ranged from -0.594 to 1, United Kingdom look-up value was applied to all participants, higher scores indicated better health states. The EQ- VAS measured participant's self-rated health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state) by VAS, where higher scores indicated better heath states.
OP reporting group: Baseline (Day 1, day of study enrollment), Month 6; ATP reporting group: Baseline (before dose on Day 1 of ATP), Month 6

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Muita asiaankuuluvia MeSH-ehtoja

Muut tutkimustunnusnumerot

  • B7841005
  • 2018-003660-31 (EudraCT-numero)

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