At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- SGT-003 (genetic)
- How long the study runs
- Study runs about 84 months (dates as stated)
- About the drug or intervention
- SGT-003 — genetic: Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
- Patient visit burden
- Not specified by the sponsor
In plain English
This study, called INSPIRE DUCHENNE, is testing a gene therapy called SGT-003 in people with Duchenne Muscular Dystrophy (DMD). It is run by Solid Biosciences Inc. DMD is a condition that causes muscle weakness. The trial has five groups (cohorts) covering different ages, from babies under 4 up to under 18 years old.
Who can take part
- Age 0 to under 18, depending on the group: Group 1 is 4 to under 7; Group 2 is 7 to under 12; Group 3 is 0 to under 4; Group 4 is 12 to under 18; Group 5 is 10 to under 18
- Groups 1, 2 and 4 must be able to walk (complete a 10-metre walk/run test in under 30 seconds). Group 3 can be able to walk or not. Group 5 must not be able to walk now but must have been able to walk in the past
- A confirmed diagnosis of DMD with a genetic test showing a change in the dystrophin gene. The sponsor's own genetic testing must confirm this. In some cases, a muscle biopsy may be needed to check dystrophin levels
- Must test negative for AAV (a virus used to carry the gene therapy) antibodies
- Groups 1, 2, 4 and 5 must have been on a stable daily steroid medicine (prednisone at least 0.5 mg per kg per day, or deflazacort at least 0.75 mg per kg per day) for at least 12 weeks before screening. Steroids are not required for Group 3
- Must meet certain movement test targets, including the 10-metre walk/run test and a test of standing up from lying down. Group 5 must also meet targets on a test called the Performance of Upper Limb (PUL) 2.0
- Body weight must be 90 kg or less
Who may not be able to
- Treatment with medicines that change dystrophin within 3 months before screening
- Current or past treatment with an approved or experimental gene therapy
- Taking certain approved or experimental medicines within 3 months before screening, or within 5 half-lives of the drug, whichever is longer
- A DMD diagnosis linked to certain gene changes that mean certain parts of the DMD gene are not made (not expressing exons 1 to 11, 42 to 45, or 57 to 69)
What taking part involves
- • Receiving SGT-003, a gene therapy, for Duchenne Muscular Dystrophy. How it is given is not stated — ask the trial team.
- • The sponsor will carry out genetic testing to confirm diagnosis. In some cases a muscle biopsy may be needed before joining.
Time commitment: Number of visits, how long the study lasts, and other details about what taking part involves are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 0 Years to 17 Years
- Who
- Male
- Number of participants
- 60
- Started
- 2024-05-06
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for duchenne muscular dystrophy
- • Phase1/Phase2 - 60 participants
- • This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy
Who can take part?
- • Ages 0 Years to 17 Years
- • Diagnosed with duchenne muscular dystrophy
- • Male only
Where?
- • London - Great Ormond Street Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study. Cohort 1 will include participants 4 to \< 7 years of age. Cohort 2 will include participants 7 to \< 12 years of age. Cohort 3 will include participants 0 to \< 4 years of age. Cohort 4 will include participants 12 to \< 18 years of age. Cohort 5 will include participants 10 to \< 18 years of age. Initiation of participant enrollment in Cohorts 4 and 5 will be subject to the accrual of safety and efficacy data from Cohorts 1-3. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 0 Years - 17 Years
- Who can join: Male only
Treatment history
Treatments you must have had:
- ✓ to determine eligibility under this criterion
What the study is looking for
- ✓Cohort 1: 4 to \<7 years of age
- ✓Cohort 2: 7 to \<12 years of age
- ✓Cohort 3: 0 to \< 4 years of age
- ✓Cohort 4: 12 to \< 18 years of age
- ✓Cohort 5: 10 to \< 18 years of age
Who cannot take part
- ✗Treatment with dystrophin modifying drugs within 3 months prior to screening.
- ✗Current or prior treatment with an approved or investigational gene transfer drug.
- ✗Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last...
- ✗Other inclusion or exclusion criteria apply.
See the full criteria
Where Is This Study? (1 UK site)
Great Ormond Street Hospital
London WC1N 3JH, United Kingdom
How to Get in Touch
Solid Bio Clinical Trials
Sponsor contactCONTACT