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Clinical Trials in the UK / NCT05768386
Enrolling by invitation Observational

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

NCT05768386 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Observational
Started
2023-01-01
Last updated
2026-01-12

Condition(s) studied

Hemophilia A

Study 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

Sex
MALE
Min age
18 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (41)

FacilityCityRegionStatus
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center Los Angeles California
UC Davis Hemophilia Treatment Center Sacramento California
University of California San Diego, Hemophilia & Thrombosis Treatment Center San Diego California
University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic San Francisco California
UCN Hemophilia and Thrombosis Center Shandon California
Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus Aurora Colorado
St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders Tampa Florida
University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown Cancer Center Louisville Kentucky
University of Michigan, Pediatric Hematology and Oncology Ann Arbor Michigan
Children's Hospital of Michigan, Hemophilia and Thrombosis Center Detroit Michigan
Washington University School of Medicine, Department of Pediatrics, Division of Hematology/Oncology St Louis Missouri
Nationwide Children's Hospital Columbus Ohio
Hemophilia Center of Western Pennsylvania Pittsburgh Pennsylvania
Royal Prince Alfred Hospital, Institute of Haematology Sydney New South Wales
Royal Brisbane and Women's Hospital Herston Queensland
Royal Adelaide Hospital Adelaide South Australia
The Alfred Hospital Melbourne Victoria
Fiona Stanley Hospital Murdoch Western Australia
University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease Leuven Belgium
Holy Spirit Hematology and Hemotherapy Center Vitória Espírito Santo
Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter Rio de Janeiro Rio de Janeiro
Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICAMP) Campinas São Paulo
Sao Paulo University Clinical Hospital São Paulo São Paulo
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
Vivantes Hospital in Friedrichshain Berlin State of Berlin
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
Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and Thrombosis A Coruña Galicia
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

+ 1 more sites — see the full list on the official registry below.

More BioMarin Pharmaceutical trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05768386 on ClinicalTrials.gov ↗ ← All trials in the UK