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

Hemophilia A Clinical Trial

— GENEr8-LTE  

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05768386
• Other study ID #: 270-401
• Secondary ID: 2022-001246-38
• Status: Enrolling by invitation
• Start date: January 1, 2023
• Est. completion date: January 2040

Study information

• Verified date: April 2024
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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).

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 172
• Est. completion date: January 2040
• Est. primary completion date: January 2040
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Locations

Country	Name	City	State
United Kingdom	Addenbrooke's Hospital, Hemophilia and Thrombophilia Center	Cambridge
United Kingdom	Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine	London
United Kingdom	Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre	London
United Kingdom	Royal London Hospital, Barts and the London Hemophilia Center	London
United Kingdom	St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis	London
United Kingdom	University Hospital Southampton NHS Foundation Trust	Southampton
United States	University of Michigan, Pediatric Hematology and Oncology	Ann Arbor	Michigan
United States	Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus	Aurora	Colorado
United States	Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center	Los Angeles	California
United States	Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania
United States	UC Davis Hemophilia Treatment Center	Sacramento	California
United States	UCN Hemophilia and Thrombosis Center	Shandon	California
United States	St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

United States,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluate the long-term safety of BMN 270	Occurrence of adverse drug reactions, serious adverse events, and events of special interest.	Duration of study (10 years)
Secondary	Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial	Changes in annualized bleeding rate (ABR) (treated bleeds and all bleeds) and FVIII activity measured over time (CSA and OSA)	Duration of study (10 years)
Secondary	Evaluate the use of hemostatic agents (ie, emicizumab)	Annualized use of concomitant hemostatic medications (annualized FVIII utilization and annualized FVIII infusion rate)	Duration of study (10 years)
Secondary	Evaluate the long-term impact of BMN 270 on HRQoL	Changes in Haemo-QOL-A	Duration of study (10 years)