이 페이지는 자동 번역되었으며 번역의 정확성을 보장하지 않습니다. 참조하십시오 영문판 원본 텍스트의 경우.

중등도 내지 중증 혈우병 B형 성인 남성에서 PF-06838435를 이용한 인자 IX 유전자 치료의 효능 및 안전성을 평가하기 위한 연구 (BENEGENE-2)

2026년 11월 2일 업데이트: Pfizer

PHASE 3, OPEN-LABEL, SINGLE-ARM STUDY TO EVALUATE EFFICACY AND SAFETY OF FIX GENE TRANSFER WITH PF-06838435 (RAAV-SPARK100-HFIX-R338L) IN ADULT MALE PARTICIPANTS WITH MODERATELY SEVERE TO SEVERE HEMOPHILIA B (FIX:C ≤2%) (BENEGENE-2)

This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objective of the study will evaluate the annualized bleeding rate [ABR] for participants treated with gene therapy versus standard of care (SOC) therapy (FIX prophylaxis replacement regimen).

연구 개요

상태

모집하지 않고 적극적으로

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

51

단계

  • 3단계

연락처 및 위치

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

연구 장소

  • 그리스
      • Athens, 그리스, 11527
        • General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center
  • 대만
      • Changhua, 대만, 500
        • Changhua Christian Hospital
      • Kaohsiung City, 대만, 807
        • Kaohsiung Medical University Chung-Ho Memorial Hospital
      • Taichung, 대만, 40705
        • Taichung Veterans General Hospital
      • Taichung, 대만, 40201
        • Chung Shan Medical University Hospital
      • Taichung, 대만, 40201
        • Chung Shan Medical University
      • Taipei, 대만, 10002
        • National Taiwan University Hospital
  • 대한민국
      • Seoul, 대한민국, 05278
        • Kyung Hee University Hospital at Gangdong
  • 독일
      • Berlin, 독일, 10249
        • Vivantes Klinikum im Friedrichshain
      • Berlin, 독일, 10249
        • Vivantes Klinikum Friedrichshain
      • Bonn, 독일, 53127
        • Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts
      • Bonn, 독일, 53127
        • Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit
      • Bonn, 독일, 53127
        • Universitätsklinikum Bonn
  • 미국
    • California
      • San Francisco, California, 미국, 94143
        • University of California, San Francisco - Clinical Research Center
      • San Francisco, California, 미국, 94143
        • University of California, San Francisco - Outpatient Hematology Clinic
      • San Francisco, California, 미국, 94143
        • UCSF IDS Pharmacy
      • San Francisco, California, 미국, 94158
        • The Regents of the University of California, San Francisco campus
    • Colorado
      • Aurora, Colorado, 미국, 80045
        • Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
      • Aurora, Colorado, 미국, 80045
        • Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts
    • Indiana
      • Indianapolis, Indiana, 미국, 46260
        • Indiana Hemophilia and Thrombosis Center, Inc
      • Indianapolis, Indiana, 미국, 46260
        • St. Vincent Hospital & Health Care Center, Inc.
      • Indianapolis, Indiana, 미국, 46260
        • Indiana Hemophilia & Thrombosis Center, Inc.
      • Indianapolis, Indiana, 미국, 46260
        • Innovative Hematology, Inc.
    • Mississippi
      • Madison, Mississippi, 미국, 39110
        • Mississippi Center for Advanced Medicine
      • Madison, Mississippi, 미국, 39110
        • Madison Radiological Group
    • Pennsylvania
      • Philadelphia, Pennsylvania, 미국, 19104
        • Center for Human Phenomic Science
      • Philadelphia, Pennsylvania, 미국, 19104
        • Penn Blood Disorder Center
      • Philadelphia, Pennsylvania, 미국, 19104
        • Investigational Drug Service
      • Philadelphia, Pennsylvania, 미국, 19104
        • The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal
  • 브라질
      • Rio de Janeiro, 브라질, 20211-030
        • Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO
    • Espírito Santo
      • Vitória, Espírito Santo, 브라질, 29047-105
        • Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes
    • São Paulo
      • Campinas, São Paulo, 브라질, 13083-878
        • Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP
      • São Paulo, São Paulo, 브라질, 05403-010
        • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
  • 사우디 아라비아
      • Riyadh, 사우디 아라비아, 11211
        • King Faisal Specialist Hospital and Research Centre
      • Riyadh, 사우디 아라비아, 11481
        • King Abdullah International Medical Research Center
    • Kingdom of Saudi Arabia
      • Riyadh, Kingdom of Saudi Arabia, 사우디 아라비아, 14611
        • King Abdulaziz Medical City-Ministry of national guard
  • 스웨덴
      • Malmö, 스웨덴, 205 02
        • Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics
      • Malmö, 스웨덴, 211 24
        • ApoEx AB
  • 스페인
      • Barcelona, 스페인, 08035
        • Hospital Universitari Vall d´Hebrón
    • Murcia
      • El Palmar, Murcia, 스페인, 30120
        • Hospital Universitario Virgen de la Arrixaca
  • 영국
      • Glasgow, 영국, G4 0SF
        • Glasgow Royal Infirmary
      • London, 영국, SE1 7EH
        • Guy's and St. Thomas' NHS Foundation Trust
      • Newcastle upon Tyne, 영국, NE2 4HH
        • Newcastle upon Tyne Hospitals NHS Foundation Trust
    • TYNE & WEAR
      • Newcastle upon Tyne, TYNE & WEAR, 영국, NE1 4LP
        • Newcastle upon Tyne Hospitals NHS FT
      • Newcastle upon Tyne, TYNE & WEAR, 영국, NE2 4HH
        • Newcastle upon Tyne Hospitals NHS FT
  • 이탈리아
      • Florence, 이탈리아, 50134
        • Azienda Ospedaliero Universitaria Careggi
      • Naples, 이탈리아, 80131
        • Azienda Ospedaliera Universitaria Federico II
  • 일본
    • Hokkaido
      • Sapporo, Hokkaido, 일본, 004-0041
        • Sapporo Tokushukai Hospital
    • Nara
      • Kashihara, Nara, 일본, 634-8522
        • Nara Medical University Hospital
    • Saitama
      • Iruma-gun, Saitama, 일본, 350-0495
        • Saitama Medical University Hospital
    • Tokyo
      • Setagaya-ku, Tokyo, 일본, 157 8535
        • National Center for Child Health and Development
  • 캐나다
    • Ontario
      • Hamilton, Ontario, 캐나다, L8N 3Z5
        • McMaster University Medical Centre - Hamilton Health Sciences
      • Hamilton, Ontario, 캐나다, L8V 1C3
        • Juravinski Hospital - Hamilton Health Sciences
      • Toronto, Ontario, 캐나다, M5B 1W8
        • St. Michael's Hospital
  • 터키 (Türkiye)
      • Adana, 터키 (Türkiye), 01130
        • Acibadem Adana Hospital
      • Gaziantep, 터키 (Türkiye), 27310
        • Gaziantep University Sahinbey Training and Research Hospital
      • Istanbul, 터키 (Türkiye), 34093
        • Istanbul University Oncology Institute
      • Izmir, 터키 (Türkiye), 35040
        • Ege University Medical Faculty Hospital
  • 프랑스
      • Bron, 프랑스, 69677
        • Hopital Cardiologique Louis Pradel - CRTH
      • Paris, 프랑스, 75015
        • Hopital Necker
  • 호주
      • Herston, 호주, 4029
        • Royal Brisbane and Women's Hospital
    • New South Wales
      • Camperdown, New South Wales, 호주, 2050
        • Royal Prince Alfred Hospital
    • South Australia
      • Adelaide, South Australia, 호주, 5000
        • Royal Adelaide Hospital
    • Victoria
      • Melbourne, Victoria, 호주, 3004
        • The Alfred Hospital
    • Western Australia
      • Murdoch, Western Australia, 호주, 6150
        • Fiona Stanley 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

18년 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

남성

설명

Inclusion Criteria

  • Males who completed 6 months of Factor IX prophylaxis therapy during the lead-in study (C0371004) prior to providing consent at the screening visit for this study.
  • Documented moderately severe to severe hemophilia B (Factor IX activity < =2%)
  • Previous experience with FIX therapy (=>50 documented exposure days to a FIX protein product)
  • Suspension of prophylaxis therapy for hemophilia B after administration of the study drug
  • Laboratory values (hemoglobin, platelets and creatinine) within study specified limits
  • Agree to contraception until components of the drug are eliminated from their body
  • Capable of giving signed informed consent

Exclusion Criteria

  • Anti-AAVRh74var neutralizing antibodies (nAb) titer above the established threshold (ie, positive for nAb).
  • History of inhibitor to Factor IX or inhibitor detected during screening. Clinical signs or symptoms of decreased response to Factor IX
  • Hypersensitivity to Factor IX replacement product or IV immunoglobulin administration
  • History of chronic infection or other chronic disease
  • Any conditions associated with increased thromboembolic risk
  • Concurrent clinically significant major disease or condition unsuitable for participation and/or may interfere with the interpretation of study results
  • Laboratory values at screening visit that are abnormal or outside acceptable study limits
  • Current unstable liver or biliary disease
  • Currently on antiviral therapy for hepatitis B or C
  • Planned surgical procedure requiring Factor IX surgical prophylactic factor treatment 15 months from screening visit
  • Use of restricted therapies (e.g., blood products, acetylsalicylic acid [aspirin] or ibuprofen, other investigational therapy, and by-passing agents)
  • Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 12 weeks of screening visit
  • Active hepatitis B or C; hepatitis B surface antigen, hepatitis B virus deoxyribonucleic acid positivity, or hepatitis C virus ribonucleic acid positivity
  • Significant liver disease
  • Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count <=200 mm3 and/or a viral load >20 copies/mL
  • Study and sponsor staff involved in the conduct of the study and their families
  • Unable to comply with study procedures
  • Sensitivity to heparin or heparin induced thrombocytopenia
  • Sensitivity to any of the study interventions, or components thereof, or drug or other allergy

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: 무작위화되지 않음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: PF-06838435/fidanacogene elaparvovec
생물학적: PF-06838435/ fidanacogene elaparvovec

Gene Therapy

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

2차 결과 측정

결과 측정
측정값 설명
기간
ABR for Treated Bleeds From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Treated Bleed: An event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding (protocol definition, unless specifically referring to untreated bleed). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
Annualized Infusion Rate (AIR) of Exogenous FIX From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

AIR = number of exogenous infusions (for any reason) received during given time period *365.25/ (Date of last day - date of first day +1) in that time period. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
Steady State Circulating Factor IX (FIX:C) From Week 12 to Month 15
기간: Week 12 to Month 15

The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent. Data reported in this Outcome Measure is the geometric mean of all assessments from week 12 to Month 15.
Week 12 to Month 15
Circulating Factor IX (FIX:C) at Week 12, Week 24, Week 65
기간: Week 12, Week 24, Week 65

The certified central clinical laboratory analyzed the sample by two one-stage assays and a chromogenic assay. The first one-stage assay was performed on BCSXP analyzer with Actin-FSL reagent. The second one-stage assay used the same analyzer but SynthAsil as reagent.
Week 12, Week 24, Week 65
Annualized Factor IX (FIX) Consumption From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

Annualized FIX consumption was reported by International Units per kilogram (IU/kg). This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002. Data reported in this Outcome Measure is average of all assessments from Week 12 to Month 15.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
ABR for Spontaneous Bleeds From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Spontaneous Bleeds: Bleeding for no apparent/known reason particularly into the joints, muscles, and soft tissues. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
ABR for Traumatic Bleeds From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Traumatic Bleeds: Bleeding event occurring for an apparent/known reason. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
ABR for Untreated Bleeds From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

ABR = number of total bleeding episodes on study during the given time period) *365.25/ (Date of last day - date of first day +1) in that time period. Surgical procedures were excluded from summary/analyses. Surgical procedures were excluded from summary/analyses. Untreated Bleed: A bleeding event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
Number of Target Joint Bleeds From Week 12 to Month 15
기간: Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)

Target Joint: Defined as a major joint (e.g., hip, elbow, wrist, shoulder, knee, and ankle) into which repeated bleeds occurred (three or more spontaneous bleeds into a single joint within a consecutive 6-month period). A target joint was considered resolved when there were =<2 bleeds into the joint within a 12-month period. Joint Bleed: A 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. This outcome measure compared data collected from lead-in study C0371004 and from current study C0371002. Pre-infusion period = at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy in lead-in study C0371004 up to dosing in current study C0371002.
Pre-infusion period: minimum of 6 months through first dose of current study, an average of 1.31 years (FIX Prophylaxis arm); Week 12 to Month 15 post infusion in current study (PF-06838435 arm)
Percentage of the Participants Without Bleeds From Week 12 to Month 15
기간: Week 12 to Month 15

Percentage of participants without bleeds (total bleeds and treated bleeds) were summarized by type from Week 12 to Month 15.
Week 12 to Month 15
Change From Baseline in Joint Health as Measured by the Hemophilia Joint Health Score (HJHS) Instrument at Month 12
기간: Baseline, Month 12

A qualified healthcare professional assessed six joints (left ankle, right ankle, left elbow, right elbow, left knee, right knee) scored from 0 to 20 based on: duration of swelling, muscle atrophy, crepitus, flexion loss, extension loss, instability, joint pain, and strength. Gait was scored (0 to 4) based on walking, stairs, running, hopping on one leg. Total score = sum of scores from all joints + gait score ranged from 0 to 124, with the higher the number equating to more severe joint damage.
Baseline, Month 12
Change From Baseline in Hemophilia Quality of Life (Haem A QoL) Physical Health Domain at Month 12
기간: Baseline, Month 12

The Haem-A-QoL questionnaire contained 46 items with ten 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; and Partnership and Sexuality. The physical health domain was considered as the primary domain in this questionnaire, had a transformed score range from 0 to 100, with lower scores representing higher quality of life. In this Outcome Measure Physical Health domain scores of Haem A QoL are reported.
Baseline, Month 12
Change From Baseline in Hemophilia Activities List (HAL) Complex Lower Extremity Activities Component Score at Month 12
기간: Baseline, Month 12

The HAL was a multiple domain measure of the impact of hemophilia on functional abilities in adults. 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. Selected items from five of the domains were used to create three components: upper extremity; basic lower extremity; and complex lower extremity activities. The component score of "complex lower extremity activities" was the most important in this questionnaire, had a transformed score range from 0 to 100, higher values indicated less functional limitations in performing tasks.
Baseline, Month 12
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Number of Participants With Adverse Events of Special Interest
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Number of Participants With Positive Neutralizing Antibody (nAb) to Adeno-associated Virus Vector (AAV) and Anti-Drug Antibody (ADA)
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
ABR for Total Bleeds (Treated and Untreated) Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
ABR for Treated Bleeds Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
AIR of Exogenous Factor IX Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
FIX: C Level Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Annualized Factor IX Consumption Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
ABR for Spontaneous and Traumatic, and Untreated Bleeds Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Change From Baseline in Joint Health as Measured by the HJHS Instrument Through the Study
기간: Baseline, 6 years

Results would be posted at secondary completion date.
Baseline, 6 years
Number of Target Joint Bleeds Through the Study
기간: Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Maximum up to 6 years (Week 312) after PF-06838435 infusion
Change From Baseline in Haem A QoL Physical Health Domain Through the Study
기간: Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion

Results would be posted at secondary completion date.
Baseline, Maximum up to 6 years (Week 312) after PF-06838435 infusion
Change From Baseline in HAL Complex Lower Extremity Activities Component Score Through the Study
기간: Baseline, 6 years

Results would be posted at secondary completion date.
Baseline, 6 years

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)

2019년 7월 29일

기본 완료 (실제)

2022년 11월 16일

연구 완료 (추정된)

2028년 2월 25일

연구 등록 날짜

최초 제출

2019년 3월 1일

QC 기준을 충족하는 최초 제출

2019년 3월 1일

처음 게시됨 (실제)

2019년 3월 4일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 11월 4일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 11월 2일

마지막으로 확인됨

2026년 11월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • C0371002
  • 2022-502844-11-00 (레지스트리 식별자: CTIS (EU))

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

미국에서 제조되어 미국에서 수출되는 제품

예

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. .

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