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One-Time gene therapy could slash bleeding in hemophilia b

NCT ID NCT03861273

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a one-time gene therapy for adult men with moderate to severe hemophilia B, a bleeding disorder. The therapy delivers a working gene to help the body produce a clotting protein called Factor IX. Researchers will track how many bleeding episodes participants have over 6 years compared to their usual treatment. The goal is to see if the gene therapy can reduce or prevent bleeds.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

51 people

The number who actually took part.

Started

Jul 2019

Expected to finish

Feb 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

hematologic disorder hemophilia A hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia B

Contacts and locations

Locations

  • Acibadem Adana Hospital

    Adana, 01130, Turkey (Türkiye)

  • ApoEx AB

    Malmö, 211 24, Sweden

  • Azienda Ospedaliera Universitaria Federico II

    Naples, 80131, Italy

  • Azienda Ospedaliero Universitaria Careggi

    Florence, 50134, Italy

  • Center for Human Phenomic Science

    Philadelphia, Pennsylvania, 19104, United States

  • Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes

    Vitória, Espírito Santo, 29047-105, Brazil

  • Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP

    Campinas, São Paulo, 13083-878, Brazil

  • Changhua Christian Hospital

    Changhua, 500, Taiwan

  • Chung Shan Medical University

    Taichung, 40201, Taiwan

  • Chung Shan Medical University Hospital

    Taichung, 40201, Taiwan

  • Ege University Medical Faculty Hospital

    Izmir, 35040, Turkey (Türkiye)

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150, Australia

  • Gaziantep University Sahinbey Training and Research Hospital

    Gaziantep, 27310, Turkey (Türkiye)

  • General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center

    Athens, 11527, Greece

  • Glasgow Royal Infirmary

    Glasgow, G4 0SF, United Kingdom

  • Guy's & St Thomas' NHS Foundation Trust, St Thomas' Hospital

    London, SE1 7EH, United Kingdom

  • Guy's and St. Thomas' NHS Foundation Trust

    London, SE1 7EH, United Kingdom

  • Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus

    Aurora, Colorado, 80045, United States

  • Hopital Cardiologique Louis Pradel - CRTH

    Bron, 69677, France

  • Hopital Necker

    Paris, 75015, France

  • Hospital Universitari Vall d´Hebrón

    Barcelona, 08035, Spain

  • Hospital Universitario Virgen de la Arrixaca

    El Palmar, Murcia, 30120, Spain

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

    São Paulo, São Paulo, 05403-010, Brazil

  • Indiana Hemophilia & Thrombosis Center, Inc.

    Indianapolis, Indiana, 46260, United States

  • Indiana Hemophilia and Thrombosis Center, Inc

    Indianapolis, Indiana, 46260, United States

  • Innovative Hematology, Inc.

    Indianapolis, Indiana, 46260, United States

  • Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti- HEMORIO

    Rio de Janeiro, 20211-030, Brazil

  • Investigational Drug Service

    Philadelphia, Pennsylvania, 19104, United States

  • Istanbul University Oncology Institute

    Istanbul, 34093, Turkey (Türkiye)

  • Juravinski Hospital - Hamilton Health Sciences

    Hamilton, Ontario, L8V 1C3, Canada

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • King Abdulaziz Medical City-Ministry of national guard

    Riyadh, Kingdom of Saudi Arabia, 14611, Saudi Arabia

  • King Abdullah International Medical Research Center

    Riyadh, 11481, Saudi Arabia

  • King Faisal Specialist Hospital and Research Centre

    Riyadh, 11211, Saudi Arabia

  • Kyung Hee University Hospital at Gangdong

    Seoul, 05278, South Korea

  • Madison Radiological Group

    Madison, Mississippi, 39110, United States

  • McMaster University Medical Centre - Hamilton Health Sciences

    Hamilton, Ontario, L8N 3Z5, Canada

  • Mississippi Center for Advanced Medicine

    Madison, Mississippi, 39110, United States

  • Nara Medical University Hospital

    Kashihara, Nara, 634-8522, Japan

  • National Center for Child Health and Development

    Setagaya-ku, Tokyo, 157 8535, Japan

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • Newcastle upon Tyne Hospitals NHS FT

    Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom

  • Newcastle upon Tyne Hospitals NHS FT

    Newcastle upon Tyne, TYNE & WEAR, NE2 4HH, United Kingdom

  • Newcastle upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE2 4HH, United Kingdom

  • Penn Blood Disorder Center

    Philadelphia, Pennsylvania, 19104, United States

  • Regents of the University of Colorado University of Colorado Denver, Office of Grants and Contracts

    Aurora, Colorado, 80045, United States

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane and Women's Hospital

    Herston, 4029, Australia

  • Royal Prince Alfred Hospital

    Camperdown, New South Wales, 2050, Australia

  • Saitama Medical University Hospital

    Iruma-gun, Saitama, 350-0495, Japan

  • Sapporo Tokushukai Hospital

    Sapporo, Hokkaido, 004-0041, Japan

  • Skåne University Hospital, Department of Hematology, Oncology and Radiation Physics

    Malmö, 205 02, Sweden

  • St. Michael's Hospital

    Toronto, Ontario, M5B 1W8, Canada

  • St. Vincent Hospital & Health Care Center, Inc.

    Indianapolis, Indiana, 46260, United States

  • Taichung Veterans General Hospital

    Taichung, 40705, Taiwan

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • The Regents of the University of California, San Francisco campus

    San Francisco, California, 94158, United States

  • The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal

    Hamilton, Pennsylvania, 19104, United States

  • The Trustees of the University of Pennsylvania, Office of Clinical Research- Legal

    Philadelphia, Pennsylvania, 19104, United States

  • UCSF IDS Pharmacy

    San Francisco, California, 94143, United States

  • University of California, San Francisco - Clinical Research Center

    San Francisco, California, 94143, United States

  • University of California, San Francisco - Outpatient Hematology Clinic

    San Francisco, California, 94143, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

  • Universitätsklinikum Bonn Institut für Klinische Chemie und Klinische Pharmakologie -Phase 1-Einheit

    Bonn, 53127, Germany

  • Universitätsklinikum Bonn, Anstalt des öffentlichen Rechts

    Bonn, 53127, Germany

  • Vivantes Klinikum Friedrichshain

    Berlin, 10249, Germany

  • Vivantes Klinikum im Friedrichshain

    Berlin, 10249, Germany

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Other studies related to the condition(s) this trial covers.