Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Brief Title

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Official Title

A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients

Brief Summary

      This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene
      roxaparvovec in combination with prophylactic corticosteroids in patients with severe
      hemophilia A.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Change in median FVIII activity as measured by chromogenic substrate assay, after administration of BMN 270 with prophylactic corticosteroids

Secondary Outcome

 Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy or emicizumab for subjects receiving FVIII or emicizumab prophylaxis respectively after administration of BMN 270 with prophylactic corticosteroids

Condition

Hemophilia A

Intervention

valoctocogene roxaparvovec

Study Arms / Comparison Groups

 valoctocogene roxaparvovec

Description:  Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Biological

Estimated Enrollment

Start Date

November 10, 2020

Completion Date

September 2026

Primary Completion Date

September 2022

Eligibility Criteria

        Inclusion Criteria:

          1. Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as
             evidenced by medical history, at the time of signing the informed consent.

          2. Must have been on prophylactic hemophilia therapy for at least 12 months prior to
             study entry.

          3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure
             days (EDs).

          4. No previous documented history of a detectable FVIII inhibitor, <0.6 Bethesda Units
             (BU).

          5. Sexually active participants must agree to use an acceptable method of effective
             contraception. Participants must agree to contraception use for at least 12 weeks
             post-infusion.

        Exclusion Criteria:

          1. Detectable pre-existing antibodies to the AAV5 capsid. Up to 25% of subjects may have
             detectable pre-existing AAV5 capsid antibodies, so long as the titer level is below
             the minimum required dilution (< 20).

          2. Any evidence of active infection or any immunosuppressive disorder; patients with HIV
             infection and undetectable viral load are not excluded.

          3. Significant renal dysfunction or liver dysfunction, infection or history of hepatic
             malignancy.

          4. Evidence of any bleeding disorder not related to hemophilia A.

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Monitor, MD, 1-800-983-4587, [email protected]

Location Countries

Australia

Location Countries

Australia

Administrative Informations

NCT ID

NCT04323098

Organization ID

BMN 270-303

Secondary IDs

2018-004616-21

Responsible Party

Sponsor

Study Sponsor

BioMarin Pharmaceutical

Study Sponsor

Medical Monitor, MD, Study Director, BioMarin Pharmaceutical

Verification Date

July 2021