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Badanie mające na celu ocenę skuteczności i bezpieczeństwa terapii genowej czynnika IX za pomocą PF-06838435 u dorosłych mężczyzn z umiarkowanie ciężką do ciężkiej hemofilią B (BENEGENE-2)

2 listopada 2026 zaktualizowane przez: 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).

Przegląd badań

Status

Aktywny, nie rekrutujący

Warunki

Interwencja / Leczenie

Typ studiów

Interwencyjne

Zapisy (Rzeczywisty)

51

Faza

  • Faza 3

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Lokalizacje studiów

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

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

18 lat do 65 lat (Dorosły, Starszy dorosły)

Akceptuje zdrowych ochotników

Nie

Płeć kwalifikująca się do nauki

Męski

Opis

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 studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

  • Główny cel: Leczenie
  • Przydział: Nielosowe
  • Model interwencyjny: Zadanie dla jednej grupy
  • Maskowanie: Brak (otwarta etykieta)

Broń i interwencje

Grupa uczestników / Arm
Interwencja / Leczenie
Eksperymentalny: PF-06838435/ fidanakogen elaparwowek
Biologiczny: PF-06838435/ fidanacogene elaparvovec

Gene Therapy

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Annualized Bleeding Rate (ABR) for Total Bleeds (Treated and Untreated) From Week 12 to Month 15
Ramy czasowe: 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)

Miary wyników drugorzędnych

Miara wyniku
Opis środka
Ramy czasowe
ABR for Treated Bleeds From Week 12 to Month 15
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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)
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: 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
Ramy czasowe: Baseline, 6 years

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

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

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

Pfizer

Śledczy

  • Dyrektor Studium: Pfizer CT.gov Call Center, Pfizer

Publikacje i pomocne linki

Osoba odpowiedzialna za wprowadzenie informacji o badaniu dobrowolnie udostępnia te publikacje. Mogą one dotyczyć wszystkiego, co jest związane z badaniem.

Publikacje ogólne

Przydatne linki

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

29 lipca 2019

Zakończenie podstawowe (Rzeczywisty)

16 listopada 2022

Ukończenie studiów (Szacowany)

25 lutego 2028

Daty rejestracji na studia

Pierwszy przesłany

1 marca 2019

Pierwszy przesłany, który spełnia kryteria kontroli jakości

1 marca 2019

Pierwszy wysłany (Rzeczywisty)

4 marca 2019

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

4 listopada 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

2 listopada 2026

Ostatnia weryfikacja

1 listopada 2026

Więcej informacji

Terminy związane z tym badaniem

Słowa kluczowe

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • C0371002
  • 2022-502844-11-00 (Identyfikator rejestru: CTIS (EU))

Plan dla danych uczestnika indywidualnego (IPD)

Planujesz udostępniać dane poszczególnych uczestników (IPD)?

TAK

Opis planu 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.

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Tak

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

produkt wyprodukowany i wyeksportowany z USA

Tak

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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