Search clinical trials for: valoctocogene roxaparvovec

Total 18 results

BioMarin Pharmaceutical

Completed

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (GENEr8-2)

Hemophilia A

United States
BioMarin Pharmaceutical

Active, not recruiting

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) (BMN 270-301)

Hemophilia A

United States, Taiwan, United Kingdom, Australia, Israel, Belgium, Brazil, France, Germany, Italy, Korea, Republic of, South Africa, Spain
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Haemophilia A Patients (270-201)

Severe Haemophilia A

United Kingdom
BioMarin Pharmaceutical

Active, not recruiting

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 (GENEr8-AAV5+)

Hemophilia A | Gene Therapy | Blood Disorder | Clotting Disorders

Taiwan, Korea, Republic of, United Kingdom, South Africa
BioMarin Pharmaceutical

Active, not recruiting

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)

Hemophilia A

United States, Australia, Taiwan, Brazil
BioMarin Pharmaceutical

Active, not recruiting

Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)

Hemophilia A

Japan
BioMarin Pharmaceutical

Recruiting

Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors (GENEr8-INH)

Hemophilia A With Inhibitor | Hemophilia A With Anti Factor VIII

United States, United Kingdom, Taiwan, Israel, Korea, Republic of, South Africa, Brazil, Italy, Germany
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) (ENVISION)

Duchenne Muscular Dystrophy

Belgium, United States, Japan, Spain, Italy, United Kingdom
Sarepta Therapeutics, Inc.

Completed

A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Enrolling by invitation

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study (EXPEDITION)

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Enrolling by invitation

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (ENDEAVOR)

Muscular Dystrophy, Duchenne

United States
Sarepta Therapeutics, Inc.

Completed

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

Muscular Dystrophy, Duchenne

United States
Hoffmann-La Roche
Sarepta Therapeutics, Inc.

Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Duchenne Muscular Dystrophy

Spain, Germany
Sarepta Therapeutics, Inc.
Hoffmann-La Roche

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) (EMBARK)

Duchenne Muscular Dystrophy

United States, Japan, Taiwan, Belgium, Spain, Germany, Hong Kong, Italy, United Kingdom
Pfizer

Recruiting

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

United States
Sarepta Therapeutics, Inc.
Hansa Biopharma AB

Active, not recruiting

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)

Duchenne Muscular Dystrophy

Spain
Pfizer

Active, not recruiting

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne

United States, Australia
Sarepta Therapeutics, Inc.

Recruiting

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy (ENDURE)

Duchenne Muscular Dystrophy

United States

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