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Enrolling by invitation No phase listed Observational

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

ClinicalTrials.gov ID: NCT05768386

Public ClinicalTrials.gov record NCT05768386. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 26, 2026, 5:46 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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 identification

NCT ID
NCT05768386
Recruitment status
Enrolling by invitation
Study type
Observational
Phase
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Enrollment
172 participants

Conditions and interventions

Conditions

Interventions

Not listed

Eligibility (public fields only)

Age range
18 Years and older
Sex
Male
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jan 1, 2023 (Actual)
Primary completion
Jan 2040 (Estimated)
Completion
Jan 2040 (Estimated)
Last update posted
Jan 12, 2026

2023 – 2040

United States locations

U.S. sites
13
U.S. states
8
U.S. cities
13
Facility City State ZIP Site status
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center Los Angeles California 90007 —
UC Davis Hemophilia Treatment Center Sacramento California 95817 —
University of California San Diego, Hemophilia & Thrombosis Treatment Center San Diego California 92121 —
University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic San Francisco California 94143 —
UCN Hemophilia and Thrombosis Center Shandon California 27517 —
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus Aurora Colorado 80045 —
St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders Tampa Florida 33614 —
University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center Louisville Kentucky 40202-3229 —
University of Michigan, Pediatric Hematology and Oncology Ann Arbor Michigan 48109-5718 —
Children's Hospital of Michigan, Hemophilia and Thrombosis Center Detroit Michigan 48201 —
Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology St Louis Missouri 63110-1093 —
Nationwide Children's Hospital Columbus Ohio 43205 —
Hemophilia Center of Western Pennsylvania Pittsburgh Pennsylvania 15213 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 28 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05768386, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jan 12, 2026 · Synced Oct 26, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT05768386 live on ClinicalTrials.gov.

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