Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Brief Title

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Official Title

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions

Brief Summary

      This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity,
      during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270
      on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week
      5 to week 52.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Change of the Median Factor VIII (FVIII) Activity

Secondary Outcome

 Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy

Condition

Hemophilia A

Intervention

Valoctocogene Roxaparvovec

Study Arms / Comparison Groups

 Valoctocogene Roxaparvovec Open Label

Description:  Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg

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

March 14, 2018

Completion Date

May 2023

Primary Completion Date

May 22, 2019

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.

          2. Must have been on prophylactic FVIII replacement 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.

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

        Exclusion Criteria:

          1. Detectable pre-existing antibodies to the AAV5 capsid.

          2. Any evidence of active infection or any immunosuppressive disorder, including HIV
             infection.

          3. Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver
             cirrhosis of any etiology or history of hepatic malignancy.

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

          5. Active Hepatitis C.

          6. Prior treatment with any vector/gene transfer agent.

Gender

Male

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Medical Director, MD, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT03392974

Organization ID

BMN 270-302

Secondary IDs

2017-003573-34

Responsible Party

Sponsor

Study Sponsor

BioMarin Pharmaceutical

Study Sponsor

Medical Director, MD, Study Director, BioMarin Pharmaceutical

Verification Date

August 2022