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

Hemophilia A Clinical Trial

— GENEr8-2  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03392974
• Other study ID #: 270-302
• Secondary ID: 2017-003573-34
• Status: Completed
• Phase: Phase 3
• Start date: March 14, 2018
• Est. completion date: June 5, 2023

Study information

• Verified date: September 2023
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: June 5, 2023
• Est. primary completion date: May 22, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Valoctocogene Roxaparvovec
Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Locations

Country	Name	City	State
United States	Hemophilia Center of Western Pennsylvania	Pittsburgh	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of the Median Factor VIII (FVIII) Activity	Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.	Week 52
Secondary	Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy	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	Weeks 5 through Week 52
Secondary	Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment	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	Weeks 5 though Week 52