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NCT06138639PHASE1, PHASE2Recruiting

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

Solid Biosciences Inc.

Start Date

5/6/2024

Completion Date

5/6/2031

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.

Eligibility Criteria

Age Range: No minimum to 17 years

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.

Interventions

GENETIC

SGT-003

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Conditions

Duchenne Muscular Dystrophy

Locations

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

University of California, Los Angeles Medical Center

Los Angeles, California 90095

United States

University of California, Davis

Sacramento, California 95817

United States

University of California

San Diego, California 92037

United States

Rare Disease Research

Atlanta, Georgia 30329

United States

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

Chicago, Illinois 60611-2605

United States

Washington University in St. Louis

St Louis, Missouri 63110

United States

Nationwide Children's Hospital

Columbus, Ohio 43215

United States

Oregon Health and Sciences University

Portland, Oregon 97239

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

Children's Hospital of the King's Daughters

Norfolk, Virginia 23510

United States

Seattle Children's Hospital

Seattle, Washington 98105

United States

The Hospital for Sick Children

Toronto, Ontario M5G 0A4

Canada

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, 00168

Italy

Great Ormond Street Hospital

London, WC1N 3JH

United Kingdom