Clinical trial NCT05768386: A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)
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
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Hemophilia A
Keywords: Gene Therapy
Clotting Disorders
Blood Disorder
Blood Coagulation Disorders
Inherited Blood Coagulation disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases
Inborn
Factor VIII
Coagulants
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2023-03-14
Enrollment: 172
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects must have completed their primary treatment study in which they were dosed BMN 270. Subjects may enroll in 270-401 even if they have restarted FVIII prophylaxis or other hemophilia A treatment.

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.

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluate the long-term safety of BMN 270
timeFrame: Duration of study (10 years)
Secondary outcomes:
measure: Evaluate the long-term effects of BMN 270 in subjects with hemophilia A previously treated in a BioMarin clinical trial
timeFrame: Duration of study (10 years)
measure: Evaluate the use of hemostatic agents (ie, emicizumab)
timeFrame: Duration of study (10 years)
measure: Evaluate the long-term impact of BMN 270 on HRQoL
timeFrame: Duration of study (10 years)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Orthopedic Institute for Children, Orthopedic Hemophilia Treatment Center
state: California
city: Sacramento
country: United States
facility: UC Davis Hemophilia Treatment Center
state: California
city: Shandon
country: United States
facility: UCN Hemophilia and Thrombosis Center
state: California
city: Aurora
country: United States
facility: Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus
state: Colorado
city: Tampa
country: United States
facility: St Joseph's Children's Hospital, Center for Bleeding and Clotting Disorders
state: Florida
city: Ann Arbor
country: United States
facility: University of Michigan, Pediatric Hematology and Oncology
state: Michigan
city: Pittsburgh
country: United States
facility: Hemophilia Center of Western Pennsylvania
state: Pennsylvania
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital, Hemophilia and Thrombophilia Center
city: London
country: United Kingdom
facility: Hammersmith Hospital, Clinical Research Facility, Centre for Translational and Experimental Medicine
city: London
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham. Adult Hemophilia Centre, West Midlands Adult Comprehensive Care Hemophilia Centre
city: London
country: United Kingdom
facility: Royal London Hospital, Barts and the London Hemophilia Center
city: London
country: United Kingdom
facility: St Thomas' Hospital, Centre for Hemophilia, Hemostasis and Thrombosis
city: Southampton
country: United Kingdom
facility: University Hospital Southampton NHS Foundation Trust
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: