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중증 혈우병 A 또는 중등도 내지 중증 혈우병 B를 앓고 있는 성인 및 청소년 참여자에 대한 PF-06741086의 효능 및 안전성 연구

2026년 7월 23일 업데이트: Pfizer

청소년 및 성인 중증(응고 인자 활동)에 대한 공개 라벨 연구

PF-06741086을 사용한 치료는 인자 대체 제품과 다르게 작용하고 억제제의 존재 하에서 작용하기 때문에 혈우병 A 또는 B에 대한 현재의 치료 방법과 비교하여 임상적으로 관련된 이점 및/또는 환자 치료에 대한 주요 기여를 입증할 것으로 예상됩니다. . 주 1회(QW) 피하(SC) 투여 가능성은 신뢰할 수 있는 혈관 접근이 없는 경우 치료 옵션을 제공하고 편의성을 높이며 더 나은 순응도를 가능하게 할 수 있습니다. 이러한 특성을 결합하면 출혈 에피소드가 감소합니다.

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

188

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

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

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

12년 (어린이, 성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

설명

포함 기준

  • 중증 혈우병 A 또는 중등도에서 중증 혈우병 B 진단을 받은 참가자(심사 시 최소 체중 35kg).
  • 참가자 또는 법적 권한을 부여받은 대리인 또는 서명된 정보에 입각한 동의(또는 해당되는 경우 경미한 동의)를 제공할 수 있는 참가자의 간병인.

비억제제 코호트에 등록된 참가자는 또한 다음 기준을 충족해야 합니다.

  • 감지할 수 없거나 문서화된 억제제 이력 없음
  • 등록 전 6개월 동안 예정된 예방 요법을 80% 이상 순응하고 관찰 단계 동안 FVIII/FIX 교체로 일상적인 예방 치료를 계속 받을 의향이 있는 FVIII/FIX 정기 예방 요법 참가자.
  • 등록 전 6개월 동안 응고 인자 주입이 필요하고 관찰 단계 동안 주문형 치료를 계속 받을 의향이 있는 6회 이상의 급성 출혈 에피소드(자발적 또는 외상성)가 있는 주문형 치료 요법을 받는 참가자.

억제제 코호트에 등록된 참가자는 또한 다음 기준을 충족해야 합니다.

  • 현재 고역가 억제제(≥5 BU/mL) 또는 현재 저역가 억제제(<5 BU/mL)가 FVIII 또는 FIX 대체에 불응하고 FVIII 또는 FIX 회복이 등록 전 6개월 이내에 예상된 것의 60% 미만임을 기록 관찰 단계
  • 6회 이상 출혈 에피소드가 있는 온디맨드 치료 요법을 받는 혈우병 A 참가자 또는 4회 이상 출혈 에피소드(자발적 또는 외상성)가 있는 혈우병 B 참가자는 관찰 단계에 등록하기 전 6개월 동안 바이패스 인자로 치료가 필요하고 계속해서 치료를 받을 의향이 있습니다. -관찰 단계 동안 치료를 요구합니다.
  • 인자 대체 요법을 받는 동안 기록된 억제제가 있지만 스크리닝 시 이전 항목에 설명된 정량적 억제제 기준을 충족하지 않는 참가자(예: 이전에 문서화된 고역가 억제제(≥5 BU/mL) 및 FVIII 또는 FIX 교체로 재도전할 수 없는 상태)는 화이자 의료 모니터의 사전 동의를 받아 사례별로 적격성을 고려할 수 있습니다.
  • 위에 언급된 출혈 기준을 충족하고 일상적인 예방(출혈을 예방하기 위해 바이패스 인자를 IV 주사하는 치료로 정의됨) 중이고 등록 전 6개월 동안 예정된 예방 요법에 최소 80% 순응도를 입증한 참가자는 다음과 같을 수 있습니다. 화이자 의료 모니터와의 논의 및 동의를 통해 사례별로 자격 여부를 고려합니다.

제외 기준

  • 관상 동맥 질환, 정맥 또는 동맥 혈전증 또는 허혈성 질환에 대한 이전 또는 현재 치료 및/또는 병력
  • 계획된 연구 기간 동안 알려진 계획된 수술 절차.
  • 혈우병 A 또는 B 이외의 알려진 지혈 결함.
  • 비정상적인 신장 또는 간 기능
  • 현재 불안정한 간 또는 담도 질환
  • 비정상적인 혈액학적 매개변수
  • 연구 참여 또는 연구 제품 투여와 관련된 위험을 증가시킬 수 있거나 연구 결과의 해석을 방해할 수 있는 기타 급성 또는 만성 의학적 또는 정신과적 상태 또는 검사실 이상, 그리고 연구자의 판단에 따라,
  • 바이패싱 제제 또는 비응고 비인자 대체 요법 또는 혈우병 치료를 위한 유전자 치료 제품을 사용한 이전 치료를 통한 현재 일상적인 예방 사례별로 고려).

  • 면역 조절 약물을 사용한 정기적인 병용 요법

    - 관찰 단계 또는 활성 치료 단계 동안 면역 관용 유도의 진행 중이거나 계획된 사용, 또는 활성 치료 단계 동안 연구 개입으로 치료 개시 후 언제든지 FVIII 또는 FIX 대체를 통한 예방

  • 연구 B7841002 및 B7841003에 참여하는 동안 PF 06741086에 대한 이전 노출.
  • 연구 참여 전 및/또는 연구 참여 중 30일(또는 현지 요건에 따라 결정) 또는 5 반감기 이내에 연구 약물(들) 또는 연구 백신과 관련된 다른 연구에 참여.
  • 인간 면역결핍 바이러스(HIV) 양성인 경우 CD4 세포 수 ≤200/uL
  • 참가자의 안전 또는 연구 결과의 해석에 영향을 미칠 수 있는 임상적으로 관련된 이상을 입증하는 ECG 스크리닝.
  • 햄스터 단백질 또는 연구 개입의 다른 구성 요소에 대한 과민성 또는 알레르기 반응이 있는 개인.
  • 연구 수행에 직접 관여한 조사 현장 직원 및 그 가족, 조사자가 감독하는 현장 직원 또는 연구 수행에 직접 관여한 가족을 포함하여 화이자 직원인 참가자.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 크로스오버 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: PF-06741086
참가자는 현재 혈우병 요법에 대한 6개월의 관찰 단계 후에 PF-06741086 치료에 배정됩니다.
의약품: PF-06741086
300밀리그램(mg) 피하(sc) 부하 용량 후 매주 1회(qw) 150mg sq. 300mg sc qw는 용량 증량 기준을 충족하는 참가자에게 처방됩니다.

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Model-Based Annualized Bleeding Rate (ABR) of Treated Bleeding Events: Inhibitor Cohort (Participants With Prior OD Therapy at OP)
기간: 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)
기간: 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)
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)

2차 결과 측정

결과 측정
측정값 설명
기간
Model-Based ABR of Joint Bleeds: Inhibitor and Non-Inhibitor Cohort
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Pfizer

수사관

  • 연구 책임자: Pfizer CT.gov Call Center, Pfizer

간행물 및 유용한 링크

연구에 대한 정보 입력을 담당하는 사람이 자발적으로 이러한 간행물을 제공합니다. 이것은 연구와 관련된 모든 것에 관한 것일 수 있습니다.

일반 간행물

유용한 링크

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2020년 3월 9일

기본 완료 (실제)

2025년 4월 29일

연구 완료 (실제)

2025년 4월 29일

연구 등록 날짜

최초 제출

2019년 5월 2일

QC 기준을 충족하는 최초 제출

2019년 5월 2일

처음 게시됨 (실제)

2019년 5월 6일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 8월 14일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 7월 23일

마지막으로 확인됨

2026년 7월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

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

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

화이자는 식별되지 않은 개별 참가자 데이터 및 관련 연구 문서(예: 프로토콜, 통계 분석 계획(SAP), 임상 연구 보고서(CSR)) 자격을 갖춘 연구자의 요청에 따라 특정 기준, 조건 및 예외가 적용됩니다. 화이자의 데이터 공유 기준 및 액세스 요청 프로세스에 대한 자세한 내용은 https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests에서 확인할 수 있습니다.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다