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Gene therapy for hemophilia a: how long does it last?

NCT ID NCT05768386

Disease control Sponsor: BioMarin Pharmaceutical 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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 checks the long-term safety and effectiveness of a one-time gene therapy (BMN 270) for people with severe hemophilia A. Participants from earlier BioMarin trials will be followed for many years to see if the treatment continues to work and remains safe. The goal is to understand how well the therapy controls bleeding over time.

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.

Participants

About 172 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2023

Expected to finish

Jan 2040

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.

Who is studied

Subjects must have completed their primary treatment study in which they were dosed BMN 270. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.

Ages

18 years and older

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: * Participants must have completed their primary treatment study or be currently enrolled in the primary treatment study at the time of closure by the Sponsor. Participants may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment. * Subjects must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. If the subject is unable to provide consent, a legally authorized representative may provide written informed consent. Exclusion Criteria: * Participants who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study should enroll in 270-401 within 4 months of the date of that study completion visit. If a participant wishes to enroll in 270-401 after 4 months, they must receive prior approval from the Medical Monitor. * Participants must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the participant from fully complying with the requirements of the study and/or would impact or interfere with evaluation and interpretation of the study data (including, if applicable, advanced HIV disease). * Where applicable, per country regulation, the participant must not currently be committed to an institution by virtue of an order issued either by judicial or administrative authorities.

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Conditions

The condition(s) this trial relates to.

blood coagulation disease coagulation protein disease hematologic disorder hemophilia A hemorrhagic disease hereditary disease inherited blood coagulation disorder severe hemophilia A vascular hemostatic disease

As listed by the trial registrant

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

Hemophilia A

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    41 sites in 12 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Addenbrooke's Hospital, Hemophilia and Thrombophilia Center

    Cambridge, United Kingdom

  • Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter

    Rio de Janeiro, Rio de Janeiro, 24000-000, Brazil

  • CHRU Lille Hospital Center Heart-Lung Institute

    Lille, 59000, France

  • CHU La Timone - La Timone Children's Hospital

    Marseille, 13005, France

  • Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)

    Campinas, São Paulo, 13083-878, Brazil

  • Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation

    Ramat Gan, 5266202, Israel

  • Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center

    Johannesburg, 2193, South Africa

  • Children's Hospital of Michigan, Hemophilia and Thrombosis Center

    Detroit, Michigan, 48201, United States

  • Fiona Stanley Hospital

    Murdoch, Western Australia, 6150, Australia

  • Glasgow Royal Infirmary, Department of Hematology

    Glasgow, G4 0SF, United Kingdom

  • Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine

    London, United Kingdom

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania, 15213, United States

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

    Aurora, Colorado, 80045, United States

  • Holy Spirit Hematology and Hemotherapy Center

    Vitória, Espírito Santo, 29040-090, Brazil

  • Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis

    A Coruña, Galicia, 15006, Spain

  • Kyung Hee University Hospital at Gangdong

    Seoul, 05278, South Korea

  • Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"

    Milan, 20122, Italy

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center

    Oxford, OX3 7LD, United Kingdom

  • Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center

    Los Angeles, California, 90007, United States

  • Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre

    London, United Kingdom

  • Royal Adelaide Hospital

    Adelaide, South Australia, 5000, Australia

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029, Australia

  • Royal London Hospital, Barts and the London Hemophilia Center

    London, United Kingdom

  • Royal Prince Alfred Hospital, Institute of Haematology

    Sydney, New South Wales, 2050, Australia

  • Sao Paulo University Clinical Hospital

    São Paulo, São Paulo, 05403 000, Brazil

  • St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders

    Tampa, Florida, 33614, United States

  • St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis

    London, United Kingdom

  • The Alfred Hospital

    Melbourne, Victoria, 3004, Australia

  • UC Davis Hemophilia Treatment Center

    Sacramento, California, 95817, United States

  • UCN Hemophilia and Thrombosis Center

    Shandon, California, 27517, United States

  • University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center

    Bonn, North Rhine-Westphalia, 53127, Germany

  • University Hospital Southampton NHS Foundation Trust

    Southampton, United Kingdom

  • University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics

    Seville, Andalusia, 41013, Spain

  • University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease

    Leuven, 3000, Belgium

  • University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center

    Louisville, Kentucky, 40202-3229, United States

  • University of California San Diego, Hemophilia & Thrombosis Treatment Center

    San Diego, California, 92121, United States

  • University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic

    San Francisco, California, 94143, United States

  • University of Michigan, Pediatric Hematology and Oncology

    Ann Arbor, Michigan, 48109-5718, United States

  • Vivantes Hospital in Friedrichshain

    Berlin, State of Berlin, 10249, Germany

  • Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology

    St Louis, Missouri, 63110-1093, United States

More trials for these conditions

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