A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401) (GENEr8-LTE)

January 8, 2026 updated by: BioMarin Pharmaceutical

A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial

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.

Study Overview

Status

Enrolling by invitation

Conditions

Detailed Description

Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270).

Study Type

Observational

Enrollment (Estimated)

172

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2050
        • Royal Prince Alfred Hospital, Institute of Haematology
    • Queensland
      • Herston, Queensland, Australia, 4029
        • Royal Brisbane and Women's 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
  • Belgium
      • Leuven, Belgium, 3000
        • University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease
  • Brazil
    • Espírito Santo
      • Vitória, Espírito Santo, Brazil, 29040-090
        • Holy Spirit Hematology and Hemotherapy Center
    • Rio de Janeiro
      • Rio de Janeiro, Rio de Janeiro, Brazil, 24000-000
        • Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter
    • São Paulo
      • Campinas, São Paulo, Brazil, 13083-878
        • Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP)
      • São Paulo, São Paulo, Brazil, 05403 000
        • Sao Paulo University Clinical Hospital
  • France
      • Lille, France, 59000
        • CHRU Lille Hospital Center Heart-Lung Institute
      • Marseille, France, 13005
        • CHU La Timone - La Timone Children's Hospital
  • Germany
    • North Rhine-Westphalia
      • Bonn, North Rhine-Westphalia, Germany, 53127
        • University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center
    • State of Berlin
      • Berlin, State of Berlin, Germany, 10249
        • Vivantes Hospital in Friedrichshain
  • Israel
      • Ramat Gan, Israel, 5266202
        • Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation
  • Italy
      • Milan, Italy, 20122
        • Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"
  • South Africa
      • Johannesburg, South Africa, 2193
        • Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
  • South Korea
      • Seoul, South Korea, 05278
        • Kyung Hee University Hospital at Gangdong
  • Spain
    • Andalusia
      • Seville, Andalusia, Spain, 41013
        • University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics
    • Galicia
      • A Coruña, Galicia, Spain, 15006
        • Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis
  • United Kingdom
      • Cambridge, United Kingdom
        • Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
      • Glasgow, United Kingdom, G4 0SF
        • Glasgow Royal Infirmary, Department of Hematology
      • London, United Kingdom
        • Royal London Hospital, Barts and the London Hemophilia Center
      • London, 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
        • St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
      • Oxford, United Kingdom, OX3 7LD
        • Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center
      • Southampton, United Kingdom
        • University Hospital Southampton NHS Foundation Trust
  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
      • Sacramento, California, United States, 95817
        • UC Davis Hemophilia Treatment Center
      • San Diego, California, United States, 92121
        • University of California San Diego, Hemophilia & Thrombosis Treatment Center
      • San Francisco, California, United States, 94143
        • University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic
      • Shandon, California, United States, 27517
        • UCN Hemophilia and Thrombosis Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
    • Florida
      • Tampa, Florida, United States, 33614
        • St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
    • Kentucky
      • Louisville, Kentucky, United States, 40202-3229
        • University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-5718
        • University of Michigan, Pediatric Hematology and Oncology
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan, Hemophilia and Thrombosis Center
    • Missouri
      • St Louis, Missouri, United States, 63110-1093
        • Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Nationwide Children's Hospital
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Hemophilia Center of Western Pennsylvania

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate the long-term safety of BMN 270
Time Frame: Duration of study (10 years)
Occurrence of adverse drug reactions, serious adverse events, and events of special interest.
Duration of study (10 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial
Time Frame: Duration of study (10 years)
Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA)
Duration of study (10 years)
Evaluate the use of hemostatic agents (ie, emicizumab)
Time Frame: Duration of study (10 years)
Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
Duration of study (10 years)
Evaluate the long-term impact of BMN 270 on HRQoL
Time Frame: Duration of study (10 years)
Changes in Haemo-QOL-A
Duration of study (10 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Medical Director, MD, BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 1, 2023

Primary Completion (Estimated)

January 1, 2040

Study Completion (Estimated)

January 1, 2040

Study Registration Dates

First Submitted

February 23, 2023

First Submitted That Met QC Criteria

March 13, 2023

First Posted (Actual)

March 14, 2023

Study Record Updates

Last Update Posted (Estimated)

January 12, 2026

Last Update Submitted That Met QC Criteria

January 8, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 270-401
  • 2022-001246-38 (EudraCT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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