RecruitingPHASE3INTERVENTIONAL

A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)

This study, called IMPACT DUCHENNE, is looking into a new gene therapy called SGT-003 for Duchenne Muscular Dystrophy. It's for boys aged 7 to 11 who are still able to walk. The study is in its final main stage (Phase 3) and aims to check how well a single dose of SGT-003 works compared to a 'dummy' treatment (placebo), and to ensure it's safe. Participants will be split into two groups: one receiving SGT-003 first, then placebo, and the other receiving placebo first, then SGT-003. Everyone will be watched closely for at least 5 years after their treatment to see the long-term effects. This will help doctors understand if SGT-003 could be a new way to help manage Duchenne.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Solid Biosciences Inc.

Enrolment target

Start

22 Oct 2025

Estimated completion

01 Jan 2034

Duchenne Muscular Dystrophy

What is this study about?

Duchenne Muscular Dystrophy (DMD) is a serious genetic condition that causes muscles to weaken over time. This can lead to problems with walking, breathing, and heart function. Researchers are constantly looking for new ways to help manage DMD, and gene therapy is one promising area of research. This particular study, called IMPACT DUCHENNE, is exploring a new gene therapy called SGT-003.

The main aim of this study is to discover if a single treatment with SGT-003 can make a real difference for boys with DMD who are still able to move around fairly well. It's what's known as a 'double-blind, placebo-controlled' study. This means neither you, your child, nor the study doctors will know whether your child is receiving the active gene therapy or a placebo (a treatment that looks exactly the same but contains no active medicine). This helps to ensure the results are as fair and accurate as possible.

This study is divided into two parts, where participants will switch treatments. For example, if your child receives the active gene therapy in the first part, they will receive the placebo in the second part, and vice-versa. After this, all participants will be carefully monitored for at least 5 years to understand the long-term effects of SGT-003. This research is crucial for understanding whether SGT-003 could be a safe and effective new treatment option for boys living with Duchenne Muscular Dystrophy.

Key takeaways

This study is testing a new gene therapy (SGT-003) for Duchenne Muscular Dystrophy.
It's for boys aged 7-11 who can still walk.
All participants will receive both the gene therapy and a placebo during the study.
Your child will be closely monitored for at least 5 years.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is specifically for boys between 7 and 11 years old who have Duchenne Muscular Dystrophy and are still able to walk. They must have a confirmed medical diagnosis of DMD, including the specific genetic change that causes it. Also, they need to be taking a stable daily dose of certain steroid medications (like prednisone or deflazacort) for at least 6 months before joining the study.

There are also some physical requirements, such as being able to meet certain times for walking 10 meters and standing up from lying down. Your child's body weight must also be 50 kg or less. They must not have received any other gene therapies or certain other experimental or approved Duchenne-modifying drugs recently.

Finally, the specific genetic change causing the DMD must not be in certain areas of the DMD gene (exons 1 to 11, 42 to 45, or 57 to 69). And, importantly, your child must not have antibodies against the common cold virus (adeno-associated virus) that is used to deliver the gene therapy, as this could stop the treatment from working.

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 between 7 and 11 years old?
Does my child have a confirmed diagnosis of Duchenne Muscular Dystrophy and still able to walk?
Has my child been taking stable steroid medication for at least 6 months?
Has my child not recently received other gene therapies or certain experimental Duchenne drugs?
Is my child's body weight 50 kg or less?

Answer every question to see your result.

What does participation involve?

If your child joins this study, they will receive a single infusion (a drip into their vein) of either the active gene therapy (SGT-003) or a placebo. Neither you nor the study team will know which it is at first. The study is split into two parts, and participants will switch treatments between these parts.

During the study, your child will have regular check-ups, which will include various tests and assessments to see how they are doing and to check their health. These appointments will involve visits to the study clinic. After the main treatment parts are complete, your child will be carefully monitored for at least 5 years. This long-term follow-up is really important to understand how the treatment works over time. The study duration from the first treatment until the end of the long-term monitoring will be at least 5 years.

Potential risks and benefits

Participating in a study like this could potentially offer your child access to a new therapy that might help manage their Duchenne Muscular Dystrophy before it is widely available. However, there are also potential risks involved, as with any new medicine. Side effects could occur, and some may be serious. The study doctors will carefully monitor your child for any problems and will explain all known potential risks before you decide to take part. Remember, joining a clinical study is completely voluntary, and you are free to withdraw your child at any time, for any reason, without it affecting their current or future medical care.

Locations (5)

Arkansas Children's Hospital
Verified postcode
Little Rock, United States· Not yet recruiting
Neurology Rare Disease Center
Verified postcode
Flower Mound, United States· Not yet recruiting
Children's Hospital of the King's Daughters
Verified postcode
Norfolk, United States· Not yet recruiting
The Children's Hospital of Westmead
Verified postcode
Sydney, Australia· Recruiting
BC Children's Hospital
Verified postcode
Vancouver, Canada· Recruiting

Common questions

What is gene therapy?

Gene therapy aims to correct or replace faulty genes that cause diseases like Duchenne Muscular Dystrophy. In this study, SGT-003 is designed to deliver a working gene to muscle cells.

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 compare the real treatment's effects accurately.

Will my child definitely get the active gene therapy?

Yes, eventually. In this study, all participants will receive both SGT-003 and the placebo at different times. No one will only receive the placebo.

How long will the study last for my child?

Your child will be monitored for at least 5 years from when they first receive SGT-003. This is to understand the long-term effects of the treatment.

Can we stop participating at any time?

Yes, joining the study is completely your choice, and you can withdraw your child at any time, for any reason, without it affecting their medical care.

How to find out more

Solid Bio Clinical Trials

clinicaltrials@solidbio.com 6173374680 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.