Active not recruitingPHASE2INTERVENTIONAL

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)

This research study is testing a new gene therapy called delandistrogene moxeparvovec for young boys with Duchenne Muscular Dystrophy (DMD). DMD is a serious condition that causes muscles to weaken over time. The main aim of this study is to see how safe this new treatment is for boys aged between two and under four years old. Researchers also want to find out if the treatment helps the body produce a special protein called dystrophin, which is usually missing in boys with DMD and is needed for healthy muscles. Participants will be involved in the study for about five years to carefully monitor the effects of the treatment.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Hoffmann-La Roche

Enrolment target

Start

29 Nov 2023

Estimated completion

18 Feb 2030

Duchenne Muscular Dystrophy

What is this study about?

This study is looking at a new treatment called delandistrogene moxeparvovec for Duchenne Muscular Dystrophy (DMD). DMD is a genetic condition that mainly affects boys, causing their muscles to become weaker over time. This happens because their bodies can't make enough of a protein called dystrophin, which is vital for muscle strength and repair.

The new treatment, delandistrogene moxeparvovec, is a type of gene therapy. Gene therapy aims to correct the problem at its source by helping the body produce the missing dystrophin protein. Essentially, it tries to deliver instructions to the cells so they can start making this important protein themselves. This study is specifically designed to understand two key things about this new treatment in young boys: first, how safe it is, and second, if it effectively helps the body make dystrophin.

By running this study, doctors hope to learn more about how this gene therapy works and if it could be a helpful and safe way to treat DMD in the future. The information gathered will be very important for developing new treatments for this challenging condition.

Key takeaways

This study is for young boys with Duchenne Muscular Dystrophy (DMD).
It tests a new gene therapy called delandistrogene moxeparvovec.
The main goals are to check the treatment's safety and if it helps make a missing muscle protein.
Participation lasts about five years with regular check-ups.
It's important to discuss potential benefits and risks with your doctor.

Who may be eligible?

This study is looking for young boys with Duchenne Muscular Dystrophy (DMD).

To be considered for this study, boys must be between 2 and under 4 years old and have a clear diagnosis of DMD. Their diagnosis needs to be confirmed by genetic tests showing a specific type of change in their genes. They should also be able to take part in simple movement tests that are suitable for their age.

However, some boys might not be able to join. This includes boys who have already had gene therapy or other experimental treatments for DMD. Boys who are taking regular steroid medication for their DMD, or who plan to start it within a year, also cannot join. Additionally, if a boy has other serious health problems or if the doctors think it wouldn't be safe for them to take part, they would not be eligible.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child a boy?
Is my child between 2 and under 4 years old?
Does my child have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) from genetic testing?
Has my child NOT had any other gene therapy or similar experimental treatments for DMD?
Is my child NOT taking regular steroid medication for DMD, and do we not plan to start it within the next year?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you and your child will be involved for about five years (264 weeks). This will involve regular visits to the study clinic where your child will have various checks and tests. These tests will help the doctors understand how your child is responding to the treatment and to monitor their health carefully. The study is "open-label," meaning both you and the study team will know your child is receiving the study treatment. The treatment involves receiving the gene therapy called delandistrogene moxeparvovec. Throughout these five years, the study team will be there to support you and answer any questions you have.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that the new gene therapy might help your child's body produce the dystrophin protein, which could improve their muscle strength and health in the long term. However, it's also important to be aware that there may be side effects or risks from the treatment, some of which might not be fully known yet. The study team will explain all known risks to you. You are completely free to withdraw your child from the study at any time, for any reason, without it affecting your child's future medical care.

Locations (7)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Chr de La Citadelle
Verified postcode
Liège, Belgium
Hôpital Necker-Enfants Malades
Verified postcode
Paris, France
Universitätsklinikum Essen
Verified postcode
Essen, Germany
PU A. Gemelli, Università Cattolica del Sacro Cuore
Verified postcode
Rome, Italy
Hospital Sant Joan De Deu
Unverified
Esplugues de Llobregas, Spain
Great Ormond Street Hospital for Children
Verified postcode
London, United Kingdom
John Radcliffe Hospital
Verified postcode
Oxford, United Kingdom

Common questions

What is Duchenne Muscular Dystrophy (DMD)?

DMD is a serious genetic condition that causes muscles to become weak over time, mainly in boys. It happens because their bodies can't make enough of a protein called dystrophin, which is needed for healthy muscles.

What is delandistrogene moxeparvovec?

It's a new type of gene therapy being tested. It aims to help the body make the missing dystrophin protein that is needed for muscle strength.

How long will the study last?

If your child participates, they will be involved in the study for about five years (specifically, 264 weeks) to carefully monitor the treatment's effects.

Will my child receive the actual treatment or a dummy treatment?

This is an "open-label" study, which means your child will receive the delandistrogene moxeparvovec treatment, and both you and the doctors will know it.

Can I take my child out of the study if we change our mind?

Yes, you can choose to withdraw your child from the study at any time, for any reason, and it will not affect their future medical care.