Back to Results

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

Interested in this Trial?

Contact Us

Trial Status Enrolling By Invitation

Trial Identifier

NCT05768386

Condition

Hemophilia A, Hemophilia a

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

The BMN 270 clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of BMN 270 for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of BMN 270 beyond 5 years and to assess the durability of efficacy.

Eligibility Criteria

Birth Sex

Male

Age

From 18 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

172

Study Started:

2023-01-01

Study Updated:

2026-01-12

Trial Locations

  • Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center

    Los Angeles, California, United States

  • UC Davis Hemophilia Treatment Center

    Sacramento, California, United States

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

    San Diego, California, United States

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

    San Francisco, California, United States

  • UCN Hemophilia and Thrombosis Center

    Shandon, California, United States

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

    Aurora, Colorado, United States

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

    Tampa, Florida, United States

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

    Louisville, Kentucky, United States

  • University of Michigan, Pediatric Hematology and Oncology

    Ann Arbor, Michigan, United States

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

    Detroit, Michigan, United States

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

    St Louis, Missouri, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, United States

  • Hemophilia Center of Western Pennsylvania

    Pittsburgh, Pennsylvania, United States

  • Royal Prince Alfred Hospital, Institute of Haematology

    Sydney, New South Wales, Australia

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, Australia

  • Royal Adelaide Hospital

    Adelaide, South Australia, Australia

  • The Alfred Hospital

    Melbourne, Victoria, Australia

  • Fiona Stanley Hospital

    Murdoch, Western Australia, Australia

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

    Leuven, Belgium

  • Holy Spirit Hematology and Hemotherapy Center

    Vitória, Espírito Santo, Brazil

  • Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter

    Rio de Janeiro, Rio de Janeiro, Brazil

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

    Campinas, São Paulo, Brazil

  • Sao Paulo University Clinical Hospital

    São Paulo, São Paulo, Brazil

  • CHRU Lille Hospital Center Heart-Lung Institute

    Lille, France

  • CHU La Timone - La Timone Children's Hospital

    Marseille, France

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

    Bonn, North Rhine-Westphalia, Germany

  • Vivantes Hospital in Friedrichshain

    Berlin, State of Berlin, Germany

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

    Ramat Gan, Israel

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

    Milan, Italy

  • Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center

    Johannesburg, South Africa

  • Kyung Hee University Hospital at Gangdong

    Seoul, South Korea

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

    Seville, Andalusia, Spain

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

    A Coruña, Galicia, Spain

  • Addenbrooke's Hospital, Hemophilia and Thrombophilia Center

    Cambridge, United Kingdom

  • Glasgow Royal Infirmary, Department of Hematology

    Glasgow, United Kingdom

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

    London, United Kingdom

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

    London, United Kingdom

  • Royal London Hospital, Barts and the London Hemophilia Center

    London, United Kingdom

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

    London, United Kingdom

  • Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center

    Oxford, United Kingdom

  • University Hospital Southampton NHS Foundation Trust

    Southampton, United Kingdom

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.

Contact BioMarin Clinical Trials

Please complete and submit the following form to express interest in participating and request more information about one of our active clinical trials

*required fields

"*" indicates required fields

Please select the trial that you are interested in from the dropdown below. Note that this list only includes currently active trials. If you cannot find the trial you are seeking, please enter the study identifier and title in the comments box below, along with any other details you would like us to know to help address your inquiry.
Consent*
Consent*
For more Information about BioMarin's privacy obligations or to exercise your privacy rights, please visit BioMarin's Data Privacy Center.
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form
This field is hidden when viewing the form

What’s Next?

Visit the following links to learn more about clinical trials.

Discuss this trial with your care team.

You may wish to print this page to share the details with your healthcare providers and discuss whether you may be eligible.

Print

Get in touch.

Contact Us