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Hemophilia A

Current Clinical Trials: Hemophilia A

Study: 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: Enrolling participants

Additional information: View on clinicaltrials.gov.

Study: 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: Enrolling participants

Additional information: View on clinicaltrials.gov.

Study: Phase I/II 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 Haemophilia A

Status: Ongoing

Additional information: View on EU Clinical Trials Registry and clinicaltrials.gov.

BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Brineura
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

Global Medical Information

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