A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) (NCT06138639)

🧪 Interventions in This Study

• SGT-003 (GENETIC)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. Cohort 1: 4 to \<7 years of age
2. Cohort 2: 7 to \<12 years of age
3. Cohort 3: 0 to \< 4 years of age
4. Cohort 4: 12 to \< 18 years of age
5. Cohort 5: 10 to \< 18 years of age
6. 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:
7. Cohorts 1, 2, and 4: Ambulatory
8. Cohort 3: Either ambulatory or non-ambulatory
9. Cohort 5: Non-ambulatory, but having been previously ambulatory by history
10. 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.
11. Negative for AAV antibodies.
12. Steroid regimen:

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (15 Sites)

• Arkansas Children's Hospital — Little Rock, Arkansas
• University of California, Los Angeles Medical Center — Los Angeles, California
• University of California, Davis — Sacramento, California
• University of California — San Diego, California
• Rare Disease Research — Atlanta, Georgia
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
• Washington University in St. Louis — St Louis, Missouri
• Nationwide Children's Hospital — Columbus, Ohio
• Oregon Health and Sciences University — Portland, Oregon
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
• Children's Hospital of the King's Daughters — Norfolk, Virginia
• Seattle Children's Hospital — Seattle, Washington
• The Hospital for Sick Children — Toronto, Ontario
• Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome,
• Great Ormond Street Hospital — London,

📞 Contact the Study Coordinator

Phone: (617) 337-4680

Email: clinicaltrials@solidbio.com

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
• 💵 Compensation indicated: All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

More studies in Duchenne Muscular Dystrophy

• Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy — Little Rock, Arkansas
• Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD — Columbus, Ohio
• Microdystrophin Gene Transfer Study in Adolescents and Children With DMD — Los Angeles, California
• NS-050/NCNP-03 in Boys With DMD (Meteor50) — Los Angeles, California
• A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) — Little Rock, Arkansas

Explore by condition: All Duchenne Muscular Dystrophy clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Contact the study coordinator using the phone/email above.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.