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 2 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. 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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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 2 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. 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 Ambulant 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

Columbus

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

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
  • * Participants who are ambulatory. Ambulatory as defined as "being able to walk without the use of an assistive device."
  • * Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing.
  • * Negative for AAV antibodies.
  • * On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
  • * Meet 10-meter walk/run time criteria
  • * Meet time to rise from supine criteria
  • * Participant has body weight: ≤50 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

4 Years to 11 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