NCT05768386CompletedPhase 4
A study to investigate long-term follow-up in subjects who received BMN 270 in a prior BioMarin Clinical Trial with hemophilia A
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
Source: EU CTIS (euclinicaltrials.eu) · Last updated 23 Mar 2026
Brief summary (registry, in English)
Evaluate the long-term safety of BMN 270
- Status (registry)
- Completed
- Phase (registry)
- Phase 4
- Study type
- Interventional
- Enrollment
- 16 (actual)
- First submitted
- 16 Feb 2024
- Start date
- 16 Aug 2024
- Lead sponsor
- Biomarin Pharmaceutical Inc.
- Diseases
- Haemophilia
- NCT number
- NCT05768386
- EU CT number
- 2023-507749-27-00
What you commit to
- Study type: Interventional · Phase 4
- Participants enrolled: 16
- 8 sites in 5 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study, not your situation.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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, critical data (in particular FVIII activity, bleeding episodes, FVIII use, AEs, SAEs, and the use of hemophilia medications) must be available from the extended duration between studies, and, in the opinion of the Investigator or Medical Monitor, any missing data would not impact or interfere with evaluation and interpretation of the study.
- 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).
Other
- 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.
Who the study is looking for
- Participants must have completed the End of Study Visit in their primary treatment study (Study 270-201, 270-203, 270-205, 270-301, 270-302, or 270-303) or be currently enrolled in one of these studies and have completed a minimum of 24 months of post-infusion follow-up in that 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.
- Participants must be capable of giving signed informed consent as described in Appendix 10.1.3 of the protocol, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Age in the registry: 18 years to - · Sex: Male · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| UZ Leuven Coordinator of this site? Confirm its status | Leuven | Belgium | Completed |
| Vivantes Netzwerk fuer Gesundheit GmbH Coordinator of this site? Confirm its status | Berlin | Germany | Completed |
| Universitaetsklinikum Bonn AöR Coordinator of this site? Confirm its status | Bonn | Germany | Completed |
| Complexo Hospitalario Universitario A Coruña Coordinator of this site? Confirm its status | A Coruña | Spain | Completed |
| University Hospital Virgen Del Rocio S.L. Coordinator of this site? Confirm its status | Sevilla | Spain | Completed |
| Centre Hospitalier Universitaire de Lille Coordinator of this site? Confirm its status | Lille | France | Completed |
| Centre Hospitalier Regional De Marseille Coordinator of this site? Confirm its status | Marseille | France | Completed |
| Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico Coordinator of this site? Confirm its status | Milan | Italy | Completed |
This page is information, not medical advice. Discuss any trial with your doctor.