NCT06138639 · Solid Biosciences Inc.
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
What this study is about
This is a conducted at multiple hospitals, where both patients and doctors know the treatment given, non-randomly assigned study to investigate the safety, tolerability, and effectiveness of a single given through a vein (IV) (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 5 cohorts in this study.
View original scientific description
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.
Interventions
GENETIC
SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Primary outcome measures
Incidence of treatment-emergent adverse events (AEs)
Time frame: Day 360
Change from baseline in Microdystrophin Protein Levels
Time frame: Day 90
Microdystrophin expression evaluation in muscle biopsies
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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
- Participant ambulatory status at the time of Screening Part A or Rescreening, as defined by the ability to complete a 10-meter walk/run test in \< 30 seconds:
- Cohorts 1, 2, and 4: Ambulatory
- Cohort 3: Either ambulatory or non-ambulatory
- Cohort 5: Non-ambulatory, but having been previously ambulatory by history
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing. In cases where a genotype may be predictive of residual dystrophin production and/or a clear clinical diagnosis of DMD cannot be made (e.g., due to age), evaluation of dystrophin levels in baseline muscle biopsies may be required to determine eligibility under this criterion.
- Negative for AAV antibodies.
- Steroid regimen:
Where
- Little Rock, Arkansas
- Los Angeles, California
- Sacramento, California
- San Diego, California
- Atlanta, Georgia
- Chicago, Illinois
- St Louis, Missouri
- Columbus, Ohio
- Portland, Oregon
- Philadelphia, Pennsylvania
- Norfolk, Virginia
- Seattle, Washington
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jul 8, 2026 · Source of record for eligibility and locations