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

Sponsor

BioMarin Pharmaceutical (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05768386

Collaborator

(none)

172

Enrollment

Location

204

Anticipated Duration (Months)

0.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A

Detailed 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).

Study Design

Study Type:

Observational

Anticipated Enrollment :

172 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Actual Study Start Date :

Jan 1, 2023

Anticipated Primary Completion Date :

Jan 1, 2040

Anticipated Study Completion Date :

Jan 1, 2040

Outcome Measures

Primary Outcome Measures

Evaluate the long-term safety of BMN 270 [Duration of study (10 years)]
Occurrence of adverse drug reactions, serious adverse events, and events of special interest.

Secondary Outcome Measures

Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial [Duration of study (10 years)]
Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA)
Evaluate the use of hemostatic agents (ie, emicizumab) [Duration of study (10 years)]
Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)
Evaluate the long-term impact of BMN 270 on HRQoL [Duration of study (10 years)]
Changes in Haemo-QOL-A

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

Male

Accepts Healthy Volunteers:

Inclusion Criteria:

Subjects must have completed their primary treatment study. Subjects 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:

Subjects who do not directly enroll in 270-401 at the time of the study completion visit in their primary treatment study must enroll in 270-401 within 4 months of the date of that study completion visit.
Subjects must be overtly healthy and not have any condition that, in the opinion of the Investigator or Medical Monitor, would prevent the subject 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).