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

Phase 1

Recruiting

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Genetic: SGT-003

• Registration Number: NCT06138639
• Lead Sponsor: Solid Biosciences Inc.

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. Cohort 1 will include participants 4 to \<6 years of age, inclusive. 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-11-14
• Study First Submitted QC: 2023-11-14
• Study First Posted: 2023-11-18
• Study Start: 2024-05-06
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-13
• Last Update Posted: 2024-12-16
• Primary Completion: 2027-05-06
• Study Completion: 2031-05-06

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 6

Inclusion Criteria

• Cohort 1: 4 to <6 years of age, inclusive

• Cohort 2: 6 to <8 years of age, inclusive

• 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.

• Participant has body weight:

  • Cohort 1: ≤25 kg
  • Cohort 2: <30 kg

• Time to rise from supine of <5 seconds.

Read More

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.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 2: SGT-003	SGT-003	All participants will receive a single IV infusion of SGT-003 on Day 1.
Cohort 1: SGT-003	SGT-003	All participants will receive a single IV infusion of SGT-003 on Day 1.

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (AEs)	Day 360

Secondary Outcome Measures

Name	Time	Method
Change from baseline in microdystrophin protein levels	Day 90, Day 360	Microdystrophin expression evaluation in muscle biopsies
Change from baseline in North Star Ambulatory Assessment (NSAA) total score	Day 540	Assessment of muscle function using a 17-item scale with each item scored from 0 to 2 and a higher score meaning a better outcome. The NSAA total score is defined as the sum of all 17 items, ranging from 0 to 34, with a higher score meaning a better outcome.
Change from baseline in stride velocity 95th centile (SV95C)	Day 540	Assessment of peak ambulatory performance captured by wearable activity monitoring device

Trial Locations

Locations (6)

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

University of California, Los Angeles Medical Center; 🇺🇸 Los Angeles, California, United States

University of California, Davis; 🇺🇸 Sacramento, California, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Children's Hospital of the King's Daughters; 🇺🇸 Norfolk, Virginia, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Ontario, Canada