Recruiting

Phase 1

Phase 2

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

Sponsor:

Solid Biosciences Inc.

Code:

NCT06138639

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 0 - 17

Healthy Volunteers: Not accepted

Interventions

SGT-003

Study Details

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

Study ID

NCT06138639

Start date

May 6, 2024

Status verified date

Jul, 2026

Completion date

May 6, 2031

Anticipated

Primary completion date

May 6, 2027

Anticipated

Eligibility Criteria

Sex: Male

Age: 0 - 17

Healthy Volunteers: Not accepted

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:

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
Meet time to rise from supine criteria
Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
Participant has body weight: ≤ 90 kg

Exclusion Criteria:

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.

Other inclusion or exclusion criteria apply.

Study Design

Enrollment

60 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Cohort 1: SGT-003

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

experimental: Cohort 2: SGT-003

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

experimental: Cohort 3: SGT-003

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

experimental: Cohort 4: SGT-003

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

experimental: Cohort 5: SGT-003

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

Interventions

SGT-003

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

Primary outcome measure

Incidence of treatment-emergent adverse events (AEs) [ Time Frame: Day 360 ]
Change from baseline in Microdystrophin Protein Levels [ Time Frame: Day 90 ]

Central Contacts and Locations

Central contacts

Solid Bio Clinical Trials

617-337-4680 clinicaltrials@solidbio.com

Locations

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Contacts

Amber Kellogg

501-364-3389 aveerapandiyan@uams.edu

Principal Investigator:

Aravindhan Veerapandiyan, MD

University of California, Los Angeles Medical Center

Recruiting

Los Angeles, California, United States, 90095

Contacts

Ummi Qasim

310-825-3264 UQasim@mednet.ucla.edu

Principal Investigator:

Perry Shieh, MD, PhD

University of California, Davis

Recruiting

Sacramento, California, United States, 95817

Contacts

Neuromuscular Research Lab

916-734-5057 NMRL@ucdavis.edu

Principal Investigator:

Craig McDonald, MD

University of California

Recruiting

San Diego, California, United States, 92037

Contacts

Gabriella Penner

858-382-3061 gpenner@health.ucsd.edu

Principal Investigator:

Chamindra Laverty, MD

Rare Disease Research

Recruiting

Atlanta, Georgia, United States, 30329

Contacts

678-883-6897 subjectrecruiting@rarediseaseresearch.com

Principal Investigator:

Renata Shih, MD

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

Recruiting

Chicago, Illinois, United States, 60611-2605

Contacts

Alka Maheshwari

312-227-3813 amaheshwari@luriechildrens.org

Washington University in St. Louis

Recruiting

St Louis, Missouri, United States, 63110

Contacts

Natalie Goedeker

314-362-4919 neuromusclepediatricresearch@wustl.edu

Principal Investigator:

Craig Zaidman, MD

Nationwide Children's Hospital

Recruiting

Columbus, Ohio, United States, 43215

Contacts

Destany McCain

614-722-6961 dbs001@nationwidechildrens.org

Principal Investigator:

Kevin Flanigan, MD

Oregon Health and Sciences University

Recruiting

Portland, Oregon, United States, 97239

Contacts

Beata Dyar

503-494-8216 dyar@ohsu.edu

Principal Investigator:

Erika Finanger, MD

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Brandt Parlanti

215-850-7769 parlantib@chop.edu

Principal Investigator:

John Brandsema, MD

Children's Hospital of the King's Daughters

Recruiting

Norfolk, Virginia, United States, 23510

Contacts

E.Ann Walker

757-668-6543 Proud.Research@CHKD.org

Principal Investigator:

Crystal Proud, MD

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Janaki O'Brien

206-987-1642 Janaki.OBrien@seattlechildrens.org

Principal Investigator:

Alicia Henriquez, MD

The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada, M5G 0A4

Contacts

Ana Stosic

416-648-6831 Ana.Stosic@SickKids.ca

Principal Investigator:

Hernan Gonorazky, MD

More Information

Sponsor

Solid Biosciences Inc.

Last update posted

Jul 8, 2026

Last verified

Jul, 2026

Keywords

DMD
Gene Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Solid Biosciences Inc. on 2026-07-08.