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Hemophilia A Clinical Trials

Hemophilia A is a rare inheritable bleeding disorder caused by a mutation in the gene that provides instructions to make the protein FVIII, which is essential for blood to clot normally.

Learn more about our hemophilia A clinical trials below.

Valoctocogene roxaparvovec-rvox

Studies

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

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Indication

Hemophilia A

Goal

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

This study is currently recruiting participants

Study Type

Phase 3

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

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Indication

Hemophilia A

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

This study is currently recruiting participants

Study Type

Phase 2

Study 270-303 Active, Not 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

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Indication

Hemophilia A

Goal

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

Active, not recruiting

Study Type

Phase 3

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]

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Indication

Hemophilia A

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

Investigational therapeutic

None

Status

Active, recruiting

Study Type

Observational

Study Website

https://saavy-study.com/

Study 270-301 Active, Not Recruiting

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

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Indication

Hemophilia A

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

This study is active, but not recruiting participants

Study Type

Phase 3

Study 270-302 Active, Not Recruiting

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

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Indication

Hemophilia A

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

This study is active, but not recruiting participants

Study Type

Phase 3

Study 270-201 Active, Not Recruiting

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

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Indication

Hemophilia A

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

Investigational therapeutic

valoctocogene roxaparvovec-rvox (BMN 270)

Status

This study is active, but not recruiting participants

Study Type

Phase 1/2