Active not recruitingPHASE3INTERVENTIONAL

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)

This study is looking at a new gene therapy called delandistrogene moxeparvovec for boys with Duchenne Muscular Dystrophy (DMD). It's designed to see how safe the treatment is and how well it works. Boys who can walk and those who can't will be included. This is a scientific study where some participants will receive the active treatment and others will receive a placebo, which looks like the real thing but has no active medicine. This helps researchers understand the true effects of the new therapy. Everyone in the study will eventually have the chance to receive the actual gene therapy. The study will last for about two and a half years.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Sarepta Therapeutics, Inc.

Enrolment target

148

Start

31 May 2023

Estimated completion

30 Jun 2028

Duchenne Muscular Dystrophy

What is this study about?

This study is testing a new gene therapy called delandistrogene moxeparvovec. Gene therapy is a cutting-edge type of treatment that aims to correct the root cause of certain genetic conditions. In the case of Duchenne Muscular Dystrophy (DMD), this new therapy is designed to help the body make a missing protein called dystrophin, which is vital for healthy muscle function.

The study involves boys with DMD, including those who are still able to walk and those who are no longer able to. Researchers want to understand if this new therapy can slow down or stop the progression of the disease and improve muscle strength. They also need to make sure the treatment is safe.

It's important to remember that this is a research study, and while the goal is to find better treatments, there are no guarantees about the results. The study follows strict ethical guidelines to protect the participants.

Key takeaways

Tests a new gene therapy (delandistrogene moxeparvovec) for DMD.
Includes boys who can and cannot walk.
Compares the therapy to a placebo in the first stage.
All participants will eventually receive the active therapy.
The study lasts for about two and a half years.
Careful health checks and muscle tests will be done throughout.

Who may be eligible?

To join this study, boys must have been officially diagnosed with Duchenne Muscular Dystrophy (DMD) through medical tests and genetic testing. If they can't walk, they need to meet specific health checks. If they can walk, they need to be between 8 and under 18 years old.

Participants must be able to follow instructions for special muscle tests. They also need to be on a steady dose of steroid medication for at least 12 weeks before starting the study, and this dose shouldn't change much during the study. Boys cannot have certain immune system markers, which will be checked with a blood test. Also, their genetic test results for DMD must show a specific type of change in the DMD gene, but not changes in exon 8 or 9.

Boys cannot have had other gene therapies or experimental treatments for DMD recently. They also can't have other serious medical conditions or abnormal test results that the study doctors think would make participating unsafe. The study doctors will carefully review all these points.

Quick self-check

Do you have an official diagnosis of Duchenne Muscular Dystrophy?
Have you been on a steady dose of steroid medicine for at least 12 weeks?
If you can walk, are you between 8 and under 18 years old?
Have you had other gene therapies or experimental DMD treatments recently? (If yes, you might not be eligible.)
Do you have any other serious health conditions that a doctor might think makes this study unsafe for you?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will be in it for about two and a half years (128 weeks). In the first part, some participants will receive the active gene therapy, and others will receive a placebo, which looks like the real treatment but contains no medicine. This is given as a drip into a vein. Neither you, your family, nor your doctors will know whether you received the active treatment or the placebo during this first part.

Throughout the study, you will have regular visits to the clinic. These visits will include checks of your health, blood tests, and special tests to measure your muscle strength and movement. Every participant will eventually have the opportunity to receive the active delandistrogene moxeparvovec gene therapy, either at the beginning of the study or later on.

Potential risks and benefits

The potential benefit of joining this study is the chance to receive a new, experimental gene therapy that might help improve muscle function or slow down the progression of DMD. However, as with any new treatment, there are potential risks and side effects that will be explained in detail by the study team. It's crucial to understand that you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (46)

Arkansas Children's Hospital
Little Rock, United States
Lucile Packard Children's Hospital Stanford
Palo Alto, United States
University of California at Davis Medical Center
Sacramento, United States
Rady Children's Hospital-San Diego
San Diego, United States
University of Florida, UF Health Center for Pediatric Neuromuscular and Rare Diseases
Gainesville, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
The Johns Hopkins Hospital, Charlotte R. Bloomberg Children's Center, Pediatric Clinical Research Unit
Baltimore, United States
Boston Children's Hospital
Boston, United States
Washington University of St. Louis, St. Louis Children's Hospital
St Louis, United States
University of Rochester, Department of Neurology
Rochester, United States
Lenox Baker Children's Hospital (Duke University)
Durham, United States
Nationwide Children's Hospital
Columbus, United States

+34 more sites — see the official record for the full list.

Common questions

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy (DMD) is a genetic condition that causes muscles to become weaker over time.

What is gene therapy?

Gene therapy is a new type of treatment that tries to fix the genetic problem that causes a disease, like DMD.

What is a placebo?

A placebo is a dummy treatment that looks exactly like the real medicine but contains no active ingredients. It helps researchers safely compare the new treatment's effects.

How long will the study last?

The study will last for about two and a half years, which is roughly 128 weeks.

Will I or my doctor know if I'm getting the real treatment?

In the first part of the study, neither you, your family, nor your doctors will know if you are receiving the real treatment or the placebo. This is called 'double-blind'.