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

Participation Requirements

• Sex:

  MALE

• Eligible Ages:

  4 to 11

Participation Criteria

Inclusion 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

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 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.

Other inclusion or exclusion criteria apply.