© 2021 BioMarin. All rights reserved.

Current Studies


 

Study 270-205

Recruiting

A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    valoctocogene roxaparvovec (BMN 270)

  • Study Type

    Phase 2

  • Goal

    Evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe hemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors

  • Status

    This study is currently recruiting participants

Visit Study 270-205 Now
 

Study 270-303

Recruiting

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

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    valoctocogene roxaparvovec (BMN 270)

  • Study Type

    Phase 3

  • Goal

    Evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A

  • Status

    This study is currently recruiting participants

Visit Study 270-303 Now
 

Study 270-701

Recruiting

A Prospective, Observational Study To Evaluate Seroprevalence and The Rate of Seroconversion of Adeno-Associated Virus Serotypes and Exploratory Vectors in Patients with Hemophilia A [SAAVY]

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    None

  • Study Type

    Observational

  • Goal

    To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate in patients with Hemophilia A, over a 3 month, or 6 month timepoint

  • Status

    Active, recruiting

Visit Study 270-701 Now
 

Study 270-301

A Phase 3 Open-Label, Single Arm-Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec (BMN 270), an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    valoctocogene roxaparvovec (BMN 270)

  • Study Type

    Phase 3

  • Goal

    Assess the efficacy of valoctocogene roxaparvovec (BMN 270) defined as median FVIII activity during weeks 48–52 following intravenous infusion of valoctocogene roxaparvovec

  • Status

    This study is active, but not recruiting participants

Visit Study 270-301 Now
 

Study 270-302

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec (BMN 270), an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    valoctocogene roxaparvovec (BMN 270)

  • Study Type

    Phase 3

  • Goal

    Assess the efficacy of valoctocogene roxaparvovec at a dose of 4E13 vg/kg defined as median FVIII activity during Weeks 48-52 following intravenous infusion of valoctocogene roxaparvovec

  • Status

    This study is active, but not recruiting participants

Visit Study 270-302 Now
 

Study 270-201

Phase 1/2 Gene Therapy Study of Valoctocogene Roxaparvovec (BMN 270) for Hemophilia A

  • Indication

    Hemophilia A

  • Investigational Therapeutic

    valoctocogene roxaparvovec (BMN 270)

  • Study Type

    Phase 1/2

  • Goal

    A Phase 1/2, dose-escalation safety, tolerability and efficacy study of valoctocogene roxaparvovec (BMN 270), an adenovirus-associated virus vector mediated gene transfer of human factor VIII in patients with severe hemophilia A

  • Status

    This study is active, but not recruiting participants

Visit Study 270-201 Now