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By invitation only NCT05768386

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

Conditions: Hemophilia A

Sponsor: BioMarin Pharmaceutical

trial.available_in: БГ
Overview
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.
Description
Study 270-401 will collect additional follow-up data in a single study for approximately 10 years among all subjects who consent to participate and have completed their primary treatment study (ie, for any study in which they received BMN 270).
Who can participate
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.
Locations 12
Australia (1)
Royal Prince Alfred Hospital, Institute of Haematology
Sydney , New South Wales
Belgium (1)
University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease
Leuven
Brazil (1)
Holy Spirit Hematology and Hemotherapy Center
Vitória , Espírito Santo
France (1)
CHRU Lille Hospital Center Heart-Lung Institute
Lille
Germany (1)
University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, Hemophilia Care Center
Bonn , North Rhine-Westphalia
Israel (1)
Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation
Ramat Gan
Italy (1)
Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Thrombosis "Angelo Bianchi Bonomi"
Milan
South Africa (1)
Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center
Johannesburg
South Korea (1)
Kyung Hee University Hospital at Gangdong
Seoul
Spain (1)
University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics
Seville , Andalusia
United Kingdom (1)
Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
Cambridge
United States (1)
Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
Los Angeles , California
Technical details
Status
By invitation only
Study type
OBSERVATIONAL
Sex
Male only
Minimum age
18 Years
Healthy volunteers
No
Start date
01.01.2023
Completion date
01.01.2040
Registry ID
NCT05768386
Source
anzctr
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