ROCTAVIAN™ (valoctocogene roxaparvovec-rvox)

Current Studies

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Study 270-304

Recruiting

Study 270-304; 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 in Japanese Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

• Indication

  Hemophilia A

• Investigational Therapeutic

  valoctocogene roxaparvovec-rvox (BMN 270)

• Study Type

  Phase 3

• Goal

  This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A

• Status

  This study is currently recruiting participants

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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-rvox (BMN 270)

• Study Type

  Phase 2

• Goal

  Evaluate the safety and efficacy of valoctocogene roxaparvovec-rvox 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

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Study 270-303

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-rvox (BMN 270)

• Study Type

  Phase 3

• Goal

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

• Status

  Active, not recruiting

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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

• Website

  http://saavy-study.com/

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-rvox (BMN 270)

• Study Type

  Phase 3

• Goal

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

• Status

  This study is active, but not recruiting participants

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Study 270-302

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec-rvox (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-rvox (BMN 270)

• Study Type

  Phase 3

• Goal

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

• Status

  This study is active, but not recruiting participants

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Study 270-201

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

• Indication

  Hemophilia A

• Investigational Therapeutic

  valoctocogene roxaparvovec-rvox (BMN 270)

• Study Type

  Phase 1/2

• Goal

  A Phase 1/2, dose-escalation safety, tolerability and efficacy study of valoctocogene roxaparvovec-rvox (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 ⌃