A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Public ClinicalTrials.gov record NCT06138639. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT06138639
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 0 Years to 17 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 5, 2024
- Primary completion
- May 5, 2027
- Completion
- May 5, 2031
- Last update posted
- Jul 7, 2026
2024 – 2031
United States locations
- U.S. sites
- 12
- U.S. states
- 10
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | Recruiting |
| University of California, Los Angeles Medical Center | Los Angeles | California | 90095 | Recruiting |
| University of California, Davis | Sacramento | California | 95817 | Recruiting |
| University of California | San Diego | California | 92037 | Recruiting |
| Rare Disease Research | Atlanta | Georgia | 30329 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611-2605 | Recruiting |
| Washington University in St. Louis | St Louis | Missouri | 63110 | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43215 | Recruiting |
| Oregon Health and Sciences University | Portland | Oregon | 97239 | Recruiting |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23510 | Recruiting |
| Seattle Children's Hospital | Seattle | Washington | 98105 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06138639, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 7, 2026 · Synced Sep 2, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06138639 live on ClinicalTrials.gov.