RECRUITING

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

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Conditions

Duchenne Muscular DystrophyDMDGene Therapy

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

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)

Quick Facts

Study Start:2024-05-06
Study Completion:2031-05-06
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06138639

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.

Ages Eligible for Study:0 Years to 17 Years
Sexes Eligible for Study:MALE
Accepts Healthy Volunteers:No
Standard Ages:CHILD
Inclusion CriteriaExclusion Criteria
  1. * Cohort 1: 4 to \<7 years of age
  2. * Cohort 2: 7 to \<12 years of age
  3. * Cohort 3: 0 to \< 4 years of age
  4. * Cohort 4: 12 to \< 18 years of age
  5. * Cohort 5: 10 to \< 18 years of age
  6. * 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:
  7. * Cohorts 1, 2, and 4: Ambulatory
  8. * Cohort 3: Either ambulatory or non-ambulatory
  9. * Cohort 5: Non-ambulatory, but having been previously ambulatory by history
  10. * 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.
  11. * Negative for AAV antibodies.
  12. * Steroid regimen:
  13. * 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.
  14. * Cohort 3: N/A
  15. * Meet 10-meter walk/run time criteria
  16. * Meet time to rise from supine criteria
  17. * Cohort 5: Meet Performance of Upper Limb (PUL) 2.0 criteria
  18. * Participant has body weight: ≤ 90 kg
  1. * Treatment with dystrophin modifying drugs within 3 months prior to screening.
  2. * Current or prior treatment with an approved or investigational gene transfer drug.
  3. * Exposure to certain approved or investigational drugs within 3 months prior to screening or 5 half-lives since last administration, whichever is longer.
  4. * 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.

Contacts and Locations

Study Contact

Solid Bio Clinical Trials
CONTACT
617-337-4680
clinicaltrials@solidbio.com

Principal Investigator

Solid Bio Clinical Trials
STUDY_DIRECTOR
Solid Biosciences

Study Locations (Sites)

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
Rare Disease Research
Atlanta, Georgia, 30329
United States
Washington University in St. Louis
Saint Louis, Missouri, 63110
United States
Nationwide Children's Hospital
Columbus, Ohio, 43215
United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510
United States

Collaborators and Investigators

Sponsor: Solid Biosciences Inc.

  • Solid Bio Clinical Trials, STUDY_DIRECTOR, Solid Biosciences

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-06
Study Completion Date2031-05-06

Study Record Updates

Study Start Date2024-05-06
Study Completion Date2031-05-06

Terms related to this study

Keywords Provided by Researchers

  • DMD
  • Gene Therapy

Additional Relevant MeSH Terms

  • Duchenne Muscular Dystrophy