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Un estudio para evaluar la eficacia y la seguridad de la terapia génica del factor IX con PF-06838435 en hombres adultos con hemofilia B de moderadamente grave a grave (BENEGENE-2)

2 de noviembre de 2026 actualizado por: 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).

Descripción general del estudio

Estado

Activo, no reclutando

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Actual)

51

Fase

  • Fase 3

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

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

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años a 65 años (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Masculino

Descripción

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 de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: No aleatorizado
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: PF-06838435/ fidanacogén elaparvovec
Biológico: PF-06838435/ fidanacogene elaparvovec

Gene Therapy

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
Periodo de tiempo: 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)

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
ABR for Treated Bleeds From Week 12 to Month 15
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: Baseline, 6 years

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

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

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Pfizer

Investigadores

  • Director de estudio: Pfizer CT.gov Call Center, Pfizer

Publicaciones y enlaces útiles

La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.

Publicaciones Generales

Enlaces Útiles

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

29 de julio de 2019

Finalización primaria (Actual)

16 de noviembre de 2022

Finalización del estudio (Estimado)

25 de febrero de 2028

Fechas de registro del estudio

Enviado por primera vez

1 de marzo de 2019

Primero enviado que cumplió con los criterios de control de calidad

1 de marzo de 2019

Publicado por primera vez (Actual)

4 de marzo de 2019

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

4 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

2 de noviembre de 2026

Última verificación

1 de noviembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • C0371002
  • 2022-502844-11-00 (Identificador de registro: CTIS (EU))

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan 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.

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

Sí

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