A study to investigate long-term follow-up in subjects who received BMN 270 in a prior BioMarin Clinical Trial with hemophilia A
- Conditions
- Hemophilia AMedDRA version: 20.0Level: LLTClassification code: 10060612Term: Hemophilia A Class: 10010331Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]
- Registration Number
- CTIS2023-507749-27-00
- Lead Sponsor
- Biomarin Pharmaceutical Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 300
Participants must have completed their primary treatment study (Study 270-201, 270- 203, 270-205, 270-301, 270-302, or 270-303) 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., 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.
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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: Evaluate the long-term safety of BMN 270;Secondary Objective: Evaluate the long-term effects of BMN 270 in participants with hemophilia A previously treated in a BioMarin clinical trial, Evaluate the use of hemostatic agents (eg, emicizumab, FVIII replacement therapy, efanesoctocog, and all approved hemostatic agents), Evaluate the long-term impact of BMN 270 on HRQoL, Assess clinically meaningful change in hemophilia A symptoms and impacts;Primary end point(s): Occurrence of adverse drug reactions, serious adverse events, and events of special interest, including: a) Hepatotoxicity; b) Thromboembolic events; c) Development of FVIII inhibitors; d)Transmission to third parties; e) Integration with theoretical risk of tumorigenesis
- Secondary Outcome Measures
Name Time Method Secondary end point(s):Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds);Secondary end point(s):FVIII activity measured over time (CSA and OSA);Secondary end point(s):Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate);Secondary end point(s):Changes in Haemo-QoL-A;Secondary end point(s):Change from Baseline in the Patient Global Impression of Severity (PGI-S) and Clinician Global Impression of Severity (CGI-S) item scores at Week 52 of the study post-BMN 270 infusion;Secondary end point(s):Patient Global Impression of Change (PGI-C) and Clinician Global Impression of Change (CGI-C) item scores at Week 52 of the study post-BMN 270 infusion