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.

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ClinicalTrials.gov public records Last synced Jul 21, 2026, 10:07 AM EDT

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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

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

Hemophilia A

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: Dec 31, 2022
Primary completion: Dec 31, 2039
Completion: Dec 31, 2039
Last update posted: Jan 11, 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 11, 2026 · Synced Jul 21, 2026

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The complete protocol, eligibility criteria, and contact information for NCT05768386 live on ClinicalTrials.gov.

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