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

Phase 3

Completed

• Conditions: Hemophilia A
• Interventions: Biological: Valoctocogene Roxaparvovec

• Registration Number: NCT03392974
• Lead Sponsor: BioMarin Pharmaceutical

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-12-22
• Study First Submitted QC: 2018-01-04
• Study First Posted: 2018-01-08
• Study Start: 2018-03-14
• Primary Completion: 2019-05-22
• Results First Submitted: 2021-08-09
• Results First Submitted QC: 2021-09-14
• Results First Posted: 2021-10-08
• Study Completion: 2023-06-05
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-26
• Last Update Posted: 2023-10-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 1

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Valoctocogene Roxaparvovec Open Label	Valoctocogene Roxaparvovec	Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg

Primary Outcome Measures

Name	Time	Method
Change of the Median Factor VIII (FVIII) Activity	Week 52	Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

Secondary Outcome Measures

Name	Time	Method
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy	Weeks 5 through Week 52	Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy during Week 5 to Week 52 post-BMN 270 infusion from the baseline utilization of exogenous FVIII replacement therapy
Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment	Weeks 5 though Week 52	Change in the annualized number of bleeding episodes requiring exogenous FVIII replacement treatment (annualized bleeding rate, ABR) during Week 5 to Week 52 of the study post-BMN 270 infusion from the baseline ABR

Trial Locations

Locations (1)

Hemophilia Center of Western Pennsylvania; 🇺🇸 Pittsburgh, Pennsylvania, United States