Long-term follow-up of people with severe hemophilia A after BMN 270 gene therapy
Follows adults with severe hemophilia A years after one BMN 270 infusion.
Plain-language title generated from the registry record
Registry title: A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
Key facts
- Where
- A Coruña and Sevilla, SpainSee the 2 sites
- Phase
- Phase 4
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for men with severe hemophilia A who took part in an earlier BioMarin trial of BMN 270 gene therapy. It follows them over the long term for safety and how long the effect lasts.
- Age
- 18 years or older
- Sex
- Men only
- Disease stage
- Severe hemophilia A
- Earlier treatment
- Must have finished, or be taking part in, an earlier BioMarin BMN 270 treatment study when it was closed by the Sponsor. Restarting FVIII prophylaxis or other hemophilia A treatment is allowed.
Main reasons for not taking part
- Did not join within 4 months of finishing the earlier study, unless the Medical Monitor approves
- Has a condition that would stop them from following the study rules or would interfere with the study data, including advanced HIV disease
- Is committed to an institution by a court or administrative order, where country rules apply
Read by machine on 9 November 2026.
Where in Spain
- Complexo Hospitalario Universitario A CoruñaA Coruña, SpainNo longer taking part
- University Hospital Virgen Del Rocio S.L.Sevilla, SpainNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- BioMarin Pharmaceutical
- Trial code
- NCT05768386EU 2023-507749-27-00
- Record updated
- 12 January 2026
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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: 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.