RECRUITING

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

Description

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
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Related Conditions:

Duchenne Muscular Dystrophy

Study Overview

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Study Details

Study overview

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.

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)

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

Condition
Duchenne Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Little Rock

Arkansas Children's Hospital, Little Rock, Arkansas, United States, 72202

Los Angeles

University of California, Los Angeles Medical Center, Los Angeles, California, United States, 90095

Sacramento

University of California, Davis, Sacramento, California, United States, 95817

Atlanta

Rare Disease Research, Atlanta, Georgia, United States, 30329

Saint Louis

Washington University in St. Louis, Saint Louis, Missouri, United States, 63110

Columbus

Nationwide Children's Hospital, Columbus, Ohio, United States, 43215

Norfolk

Children's Hospital of the King's Daughters, Norfolk, Virginia, United States, 23510

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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 in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.

Ages Eligible for Study

0 Years to 17 Years

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Solid Biosciences Inc.,

Solid Bio Clinical Trials, STUDY_DIRECTOR, Solid Biosciences

Study Record Dates

2031-05-06