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Completed

NCT Number: NCT03392974

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Cardiovascular Diseases Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Hemostatic Disorders Vascular Diseases

Age range

18 year and older

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, 15213-4306, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history.
  • Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
  • Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days.
  • No previous documented history of a detectable FVIII inhibitor of less than 0.6 Bethesda Units (BU).

Exclusion criteria

  • Detectable pre-existing antibodies to the AAV5 capsid.
  • Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
  • Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver cirrhosis of any etiology or history of hepatic malignancy.
  • Evidence of any bleeding disorder not related to hemophilia A.
  • Active Hepatitis C.
  • Prior treatment with any vector/gene transfer agent.

Treatment and study plan

Valoctocogene Roxaparvovec

Biological

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Other names: BMN 270

Primary outcomes

  1. Change of the Median Factor VIII (FVIII) Activity

    Time frame: Week 52

    Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.

Secondary outcomes

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

    Time frame: 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

  2. Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment

    Time frame: 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

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study 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

Acronym: GENEr8-2

Important dates

Study start
2018
Primary completion
2019
Study completion
2023
First posted
Jan 8, 2018
Registry last updated
Oct 3, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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