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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

Full criteria on EU CTIS (euclinicaltrials.eu)

Sites (8)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
UZ Leuven
Coordinator of this site? Confirm its status
LeuvenBelgiumCompleted
Vivantes Netzwerk fuer Gesundheit GmbH
Coordinator of this site? Confirm its status
BerlinGermanyCompleted
Universitaetsklinikum Bonn AöR
Coordinator of this site? Confirm its status
BonnGermanyCompleted
Complexo Hospitalario Universitario A Coruña
Coordinator of this site? Confirm its status
A CoruñaSpainCompleted
University Hospital Virgen Del Rocio S.L.
Coordinator of this site? Confirm its status
SevillaSpainCompleted
Centre Hospitalier Universitaire de Lille
Coordinator of this site? Confirm its status
LilleFranceCompleted
Centre Hospitalier Regional De Marseille
Coordinator of this site? Confirm its status
MarseilleFranceCompleted
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Coordinator of this site? Confirm its status
MilanItalyCompleted

This page is information, not medical advice. Discuss any trial with your doctor.