RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

This study is investigating a new gene therapy called SGT-003 for Duchenne muscular dystrophy. The main goal is to find out if this treatment is safe for patients and how well their bodies cope with it. Researchers also want to see if it helps with the condition. It’s an 'open-label' study, meaning everyone involved will know they are receiving the active treatment, SGT-003. There isn't a placebo group. Participants, who will be boys aged 0 to 17, will receive a single dose of SGT-003 through a drip into their vein. The study will separate participants into different age groups, and some groups might start later. Everyone who joins will be followed for a full five years to track the long-term effects of the gene therapy.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Solid Biosciences Inc.

Enrolment target

Start

06 May 2024

Estimated completion

06 May 2031

Duchenne Muscular Dystrophy

What is this study about?

This research study is called "INSPIRE DUCHENNE" and it's looking into a new treatment called SGT-003 for Duchenne muscular dystrophy (DMD). DMD is a serious condition that mainly affects boys, causing muscles to become weaker over time.

The SGT-003 treatment is a type of 'gene therapy'. This means it aims to help the body make a vital protein, called dystrophin, that is missing or faulty in people with Duchenne. The researchers want to see if this treatment is safe for people to use and if it can help improve their condition.

This is a 'Phase 1' and 'Phase 2' study. This means it's still in the early stages, focusing on safety first, and then looking at how well the treatment works. Participants will get one single treatment of SGT-003 through a drip into their arm. They will then be carefully watched by doctors and nurses for five years to see how they are doing in the long term. This long follow-up is very important to understand all the effects of the treatment.

Key takeaways

The study is testing a new gene therapy (SGT-003) for Duchenne muscular dystrophy.
It's an early-stage study (Phase 1/2) focused on safety and how well the treatment works.
Participants will receive a single treatment through an IV drip.
The study includes boys aged 0 to 17 with Duchenne.
Everyone in the study will be followed for five years to monitor long-term effects.
There are specific requirements for who can join, including age and walking ability.

Who may be eligible?

To join this study, participants must be boys aged between 0 and 17 years old with a confirmed diagnosis of Duchenne muscular dystrophy. Your doctor will need to confirm this diagnosis and also check your specific genetic changes.

There are different age groups, called 'cohorts', in the study. For most age groups (4 to under 7, 7 to under 12, and 12 to under 18), you would need to be able to walk a short distance (10 meters) in less than 30 seconds. Younger children (0 to under 4) can join whether they are walking or not. There's also a specific group for those aged 10 to under 18 who are no longer walking but could walk before. You also need to be taking a stable dose of steroid medication, unless you are in the youngest group.

There are also some reasons why you might not be able to join. For example, if you have recently had other experimental treatments for Duchenne, or if you have specific types of antibodies (natural immune responses) to the treatment being used in the study. Your weight must also be 90 kg or less.

Quick self-check

Are you a male with a confirmed Duchenne muscular dystrophy diagnosis?
Are you between 0 and 17 years old?
Are you currently on a stable steroid medication (if applicable for your age group)?
Have you spoken with your doctor about your specific gene changes related to Duchenne?
Can you meet the walking requirements for your age group (if applicable)?
Have you avoided other new Duchenne treatments recently?

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

What does participation involve?

If you decide to join the study, you will receive a single dose of the SGT-003 treatment through a drip into a vein. This is the main part of the intervention. After receiving the treatment, you will have regular check-ups and appointments with the study doctors and nurses. These visits will involve various tests and assessments to monitor your health, how your body is reacting to the treatment, and how your Duchenne muscular dystrophy is progressing.

These assessments might include blood tests, muscle function tests (like walking tests or strength tests), and other medical examinations. The study involves a long-term follow-up period, meaning you will be part of the study for a total of five years after receiving the treatment. This is to make sure researchers can fully understand the long-term effects of SGT-003. The exact number and frequency of visits will be explained to you by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. The potential benefit of this study is that the SGT-003 gene therapy might help to improve muscle strength and slow down the progression of Duchenne muscular dystrophy. However, as this is an early-stage study, we don't yet know for sure how effective it will be. There are also potential risks and side effects associated with any new treatment, especially gene therapy. These will be carefully explained to you by the study team before you consent to take part. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (15)

Arkansas Children's Hospital
Little Rock, United States· Recruiting
University of California, Los Angeles Medical Center
Los Angeles, United States· Recruiting
University of California, Davis
Sacramento, United States· Recruiting
University of California
San Diego, United States· Recruiting
Rare Disease Research
Atlanta, United States· Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States· Recruiting
Washington University in St. Louis
St Louis, United States· Recruiting
Nationwide Children's Hospital
Columbus, United States· Recruiting
Oregon Health and Sciences University
Portland, United States· Recruiting
Children's Hospital of Philadelphia
Philadelphia, United States· Recruiting
Children's Hospital of the King's Daughters
Norfolk, United States· Recruiting
Seattle Children's Hospital
Seattle, United States· Recruiting

+3 more sites — see the official record for the full list.

Common questions

What is SGT-003?

SGT-003 is an investigational gene therapy aiming to provide the body with a working copy of the gene needed to make dystrophin, a protein missing in Duchenne muscular dystrophy.

How will I receive the SGT-003 treatment?

You will receive the SGT-003 treatment as a single dose given through a drip directly into a vein, similar to receiving fluids in a hospital.

How long will I be in the study?

If you join the study, you will be followed by the research team for a total of five years to track the long-term effects of the treatment.

Is this treatment guaranteed to work?

No, this is a research study, and we are still investigating if SGT-003 is effective and safe. No treatment can be guaranteed to work.

Can girls participate in this study?

This particular study is specifically for male participants with Duchenne muscular dystrophy.

How to find out more

Solid Bio Clinical Trials

clinicaltrials@solidbio.com 617-337-4680 Official trial record

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