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

Официальное название

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

Обзор

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.

Подробное описание

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).

Первичные конечные точки

• Evaluate the long-term safety of BMN 270 [Срок оценки: Duration of study (10 years)]

Вторичные конечные точки (3)

• Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial [Срок оценки: Duration of study (10 years)]
• Evaluate the use of hemostatic agents (ie, emicizumab) [Срок оценки: Duration of study (10 years)]
• Evaluate the long-term impact of BMN 270 on HRQoL [Срок оценки: Duration of study (10 years)]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 13 центров

• Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center — Los Angeles
• UC Davis Hemophilia Treatment Center — Sacramento
• University of California San Diego, Hemophilia & Thrombosis Treatment Center — San Diego
• University of California San Francisco - Moffit Hospital, Outpatient Hematology Clinic — San Francisco
• UCN Hemophilia and Thrombosis Center — Shandon
• Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus — Aurora
• St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders — Tampa
• University Medical Center, Inc,; DBA University of Louisville Hospital, James Graham Brown — Louisville
• … и ещё 5 центров

Великобритания · 8 центров

• Addenbrooke's Hospital, Hemophilia and Thrombophilia Center — Cambridge
• Glasgow Royal Infirmary, Department of Hematology — Glasgow
• Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimenta — London
• Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehe — London
• Royal London Hospital, Barts and the London Hemophilia Center — London
• St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis — London
• Nuffield Orthopaedic Centre, Hemophilia & Thrombosis Center — Oxford
• University Hospital Southampton NHS Foundation Trust — Southampton

Австралия · 5 центров

• Royal Prince Alfred Hospital, Institute of Haematology — Sydney
• Royal Brisbane and Women's Hospital — Herston
• Royal Adelaide Hospital — Adelaide
• The Alfred Hospital — Melbourne
• Fiona Stanley Hospital — Murdoch

Бразилия · 4 центра

• Holy Spirit Hematology and Hemotherapy Center — Vitória
• Arthur De Siqueira Cavalcanti Hematology State Institute / Coordinating Hemocenter — Rio de Janeiro
• Campinas University Clinical Hospital, Hematology and Hemotherapy Center (Hemocentro UNICA — Campinas
• Sao Paulo University Clinical Hospital — São Paulo

Франция · 2 центра

• CHRU Lille Hospital Center Heart-Lung Institute — Lille
• CHU La Timone - La Timone Children's Hospital — Marseille

Германия · 2 центра

• University Hospital Bonn, Institute of Experimental Hematology and Transfusion Medicine, H — Bonn
• Vivantes Hospital in Friedrichshain — Berlin

Испания · 2 центра

• University Hospital Virgen del Rocio (HUVR), Hematology Pediatrics — Seville
• Hospital Teresa Herrera, Department of Hematology and Hemotherapy, Unit of Hemostasis and — A Coruña

Бельгия · 1 центр

• University Hospitals Leuven, Campus Gasthuisberg, Department of Heart and Vascular Disease — Leuven

Израиль · 1 центр

• Chaim Sheba Medical Center, National Institute of Hemophilia and Coagulation — Ramat Gan

Италия · 1 центр

• Maggiore Polyclinic Hospital, Foundation IRCCS Ca' Granda, Center for Hemophilia and Throm — Milan

ЮАР · 1 центр

• Charlotte Maxeke Johannesburg Academic Hospital, Hemophilia Comprehensive Care Center — Johannesburg

South Korea · 1 центр

• Kyung Hee University Hospital at Gangdong — Seoul

Идентификаторы

NCT: NCT05768386 · 270-401 · 2022-001246-38

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗