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

About

Brief Summary

This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single IV infusion of SGT-003 in participants with Duchenne muscular dystrophy. There will be 2 cohorts in this study, dosed sequentially. Cohort 1 will include participants 4 to <6 years of age, inclusive. Cohort 2 will only be opened after dosing and monitoring a subset of participants in Cohort 1. Cohort 2 will include participants 6 to <8 years of age, inclusive. All participants will receive SGT-003 and will be enrolled in the study for 5 total years for long-term follow up.

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase 1/Phase 2

Eligibility

Gender

Male

Healthy Volunteers

Minimum Age

4 Years

Maximum Age

7 Years

Inclusion Criteria:

Cohort 1: 4 to <6 years of age, inclusive
Cohort 2: 6 to <8 years of age, inclusive
Ambulatory as defined as "being able to walk without the use of an assistive device."
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.
Cohort 1: <18 kg body weight
Cohort 2: <30 kg body weight

Exclusion Criteria:

Current or prior treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen.
Current or prior treatment with an approved or investigational gene transfer drug.
Exposure to another investigational drug 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.

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