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Clinical Trials List

Protocol Number270-401
NCT Number(ClinicalTrials.gov Identfier)NCT05768386
Not yet recruiting

2024-05-01 - 2039-02-01

Phase IV

Recruiting5

ICD-10D66

Hereditary factor VIII deficiency

ICD-9286.0

Congenital factor VIII disorder

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

  • Trial Applicant

    TAIWAN PSI HEALTH DEVELOPMENT COMPANY LIMITED

  • Sponsor

    BioMarin Pharmaceuticals Inc.

  • Trial scale

    Multi-Regional Multi-Center

  • Update

    2026/02/01

Investigators and Locations

Principal Investigator 林炫聿 Division of Hematology & Oncology
CHANGHUA CHRISTIAN HOSPITAL

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator JIAAN-DER WANG Division of Pediatrics
Taichung Veterans General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Sheng-chieh Chou Division of Hematology & Oncology
National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator 陳宇欽 Division of Hematology & Oncology
Tri-Service General Hospital

Co-Principal Investigator

  • 賴學緯 Division of Hematology & Oncology

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Shyh-Shin Chiou
Kaohsiung Medical Univeristy Chung-Ho Memorial Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Condition/Disease

Hemophilia A

Objectives

Primary Objective • To evaluate the long-term safety of BMN 270. Secondary Objectives • To evaluate the long-term efficacy of BMN 270 in participants with hemophilia A who have received treatment in a BioMarin clinical trial. • To assess the use of hemostatic medications (e.g., emicizumab). • To evaluate the long-term impact of BMN 270 on health-related quality of life (HRQoL).

Test Drug

BMN 270

Active Ingredient

AAV5-hFVIII-SQ

Dosage Form

27D

Dosage

2E13 VG/ML

Endpoints

• Incidence of adverse drug reactions, serious adverse events, and adverse events of special interest, including:
 o Hepatotoxicity
 o Thromboembolic events
 o Development of FVIII inhibitors
 o Vector transmission to third parties
 o Vector integration with potential tumorigenic risk

Inclution Criteria

Participants must meet all of the following criteria to be eligible for the study:

(1) Participants must have completed the end-of-study visit of the parent study, or be currently participating in the parent study and have completed at least 24 months of post-infusion follow-up in that study, which has been terminated by the sponsor. Participants who have restarted FVIII prophylaxis or other treatments for hemophilia A remain eligible for inclusion in Study 270-401.

(2) Participants must be able to provide a signed Informed Consent Form (ICF), including agreement to comply with the requirements and restrictions listed in the ICF.

Exclusion Criteria

(1) If a participant does not enroll directly into Study 270-401 at the time of the end-of-study visit of the parent study, the participant must enroll in Study 270-401 within 4 months from the date of that visit. If more than 4 months have passed, the participant may still be eligible to enroll in Study 270-401 provided that key data from the interim period between studies (specifically FVIII activity, bleeding events, FVIII usage, adverse events [AEs], serious adverse events [SAEs], and hemophilia-related medication use) are available, and the investigator or medical monitor determines that any missing data will not affect or interfere with the assessment or interpretation of this study.

(2) Participants must be in good general health and, in the opinion of the investigator or medical monitor, must have no condition that would prevent full compliance with study requirements and/or interfere with the assessment or interpretation of study data (including, when applicable, advanced HIV disease).

(3) Where national regulations apply, participants must not be under any judicial or administrative order of institutionalization (for example, confinement to a care or correctional facility).

The Estimated Number of Participants

  • Taiwan

    19 participants

  • Global

    200 participants

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