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

NCT06138639 | Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: SGT-003-1012024-514501-57-001010251
• Study Type: interventional
• Target: 60
• Locations: 4 countries
• Sites: 15

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Gene Therapy

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (AEs)

  Day 360

• Change from baseline in Microdystrophin Protein Levels

  Microdystrophin expression evaluation in muscle biopsies

  Day 90

Secondary Outcomes (12)

• Change from Baseline of Microdystrophin Tissue Distribution by Immunofluorescence (IF)

  Day 90, Day 360

• Change from baseline in Microdystrophin Protein Levels

  Day 360

• Change from Baseline in Time to Rise Velocity

  Day 360, Day 540

• Change from baseline in Stride Velocity 95th Centile (SV95C)

  Day 360, Day 540

• Change from baseline in 10-meter walk/run velocity

  Day 360, Day 540

Study Arms (5)

Cohort 1: SGT-003

EXPERIMENTAL

All ambulatory participants from age 4 to \< 7 years will receive a single IV infusion of SGT-003 on Day 1.

Genetic: SGT-003

Cohort 2: SGT-003

EXPERIMENTAL

All ambulatory participants from age 7 to \< 12 years will receive a single IV infusion of SGT-003 on Day 1.

Genetic: SGT-003

Cohort 3: SGT-003

EXPERIMENTAL

All participants from age 0 to \< 4 years will receive a single IV infusion of SGT-003 on Day 1.

Genetic: SGT-003

Cohort 4: SGT-003

EXPERIMENTAL

All ambulatory participants from age 12 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.

Genetic: SGT-003

Cohort 5: SGT-003

EXPERIMENTAL

All non-ambulatory participants from age 10 to \< 18 years will receive a single IV infusion of SGT-003 on Day 1.

Genetic: SGT-003

Interventions

SGT-003 GENETIC

Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Cohort 1: SGT-003; Cohort 2: SGT-003; Cohort 3: SGT-003; Cohort 4: SGT-003; Cohort 5: SGT-003

Eligibility Criteria

• Age: 0 Years - 17 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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:
• Cohorts 1, 2, 4, and 5: A stable daily oral steroid regimen of at least 0.5 mg/kg/day of prednisone or 0.75 mg/kg/day of deflazacort for ≥12 weeks prior to Screening Part A or Rescreening, allowing for weight-based modifications consistent with clinical practice.
• Cohort 3: N/A
• Meet 10-meter walk/run time criteria

You may not qualify if:

• 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 administration, whichever is longer.
• Established clinical diagnosis of DMD that is associated with any deletion mutation invariant or variant predicted to not express exons 1 to 11 or, exons 42 to 45, or exons 57 to 69, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Sponsors & Collaborators

• Solid Biosciences Inc.: lead

Study Sites (15)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

University of California, Los Angeles Medical Center

Los Angeles, California, 90095, United States

RECRUITING

University of California, Davis

Sacramento, California, 95817, United States

RECRUITING

University of California

San Diego, California, 92037, United States

RECRUITING

Rare Disease Research

Atlanta, Georgia, 30329, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611-2605, United States

RECRUITING

Washington University in St. Louis

St Louis, Missouri, 63110, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

RECRUITING

Oregon Health and Sciences University

Portland, Oregon, 97239, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Children's Hospital of the King's Daughters

Norfolk, Virginia, 23510, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 0A4, Canada

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, 00168, Italy

RECRUITING

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Solid Bio Clinical Trials

  Solid Biosciences

  STUDY DIRECTOR

Central Study Contacts

Solid Bio Clinical Trials

CONTACT

617-337-4680; clinicaltrials@solidbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2023
• First Posted: November 18, 2023
• Study Start: May 6, 2024
• Primary Completion (Estimated): May 6, 2027
• Study Completion (Estimated): May 6, 2031
• Last Updated: March 3, 2026

Record last verified: 2026-03

Locations

CA (1); IT (1); GB (1); US (12)