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Une étude pour évaluer l'efficacité et l'innocuité de la thérapie génique par le facteur IX avec le PF-06838435 chez les hommes adultes atteints d'hémophilie B modérément sévère à sévère (BENEGENE-2)

2 novembre 2026 mis à jour par: 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).

Aperçu de l'étude

Statut

Actif, ne recrute pas

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Réel)

51

Phase

  • Phase 3

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Lieux d'étude

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

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

18 ans à 65 ans (Adulte, Adulte plus âgé)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Homme

La description

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

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Non randomisé
  • Modèle interventionnel: Affectation à un seul groupe
  • Masquage: Aucun (étiquette ouverte)

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Expérimental: PF-06838435/ fidanacogene elaparvovec
Biologique: PF-06838435/ fidanacogene elaparvovec

Gene Therapy

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
Délai: 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)

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
ABR for Treated Bleeds From Week 12 to Month 15
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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)
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: 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
Délai: Baseline, 6 years

Results would be posted at secondary completion date.
Baseline, 6 years
Number of Target Joint Bleeds Through the Study
Délai: 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
Délai: 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
Délai: Baseline, 6 years

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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Pfizer

Les enquêteurs

  • Directeur d'études: Pfizer CT.gov Call Center, Pfizer

Publications et liens utiles

La personne responsable de la saisie des informations sur l'étude fournit volontairement ces publications. Il peut s'agir de tout ce qui concerne l'étude.

Publications générales

Liens utiles

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

29 juillet 2019

Achèvement primaire (Réel)

16 novembre 2022

Achèvement de l'étude (Estimé)

25 février 2028

Dates d'inscription aux études

Première soumission

1 mars 2019

Première soumission répondant aux critères de contrôle qualité

1 mars 2019

Première publication (Réel)

4 mars 2019

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

4 novembre 2026

Dernière mise à jour soumise répondant aux critères de contrôle qualité

2 novembre 2026

Dernière vérification

1 novembre 2026

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • C0371002
  • 2022-502844-11-00 (Identificateur de registre: CTIS (EU))

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

OUI

Description du régime IPD

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.

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Oui

Étudie un produit d'appareil réglementé par la FDA américaine

Non

produit fabriqué et exporté des États-Unis.

Oui

Ces informations ont été extraites directement du site Web clinicaltrials.gov sans aucune modification. Si vous avez des demandes de modification, de suppression ou de mise à jour des détails de votre étude, veuillez contacter register@clinicaltrials.gov. Dès qu'un changement est mis en œuvre sur clinicaltrials.gov, il sera également mis à jour automatiquement sur notre site Web .

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