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

Study Overview
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Brief Summary
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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
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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).

Official Title
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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

Conditions
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Hemophilia A

Participation Criteria
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Eligibility Criteria
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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).

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | 
| 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) | 
| Evaluate the long-term impact of BMN 270 on HRQoL | Changes in Haemo-QOL-A | Duration of study (10 years) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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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