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

Duchenne Muscular Dystrophy Clinical Trial

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 Ambulant Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06138639
• Other study ID #: SGT-003-101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: April 5, 2024
• Est. completion date: January 15, 2030

Study information

• Verified date: March 2024
• Source: Solid Biosciences Inc.
• Contact: Solid Bio Clinical Trials
• Phone: 617-337-4680
• Email: clinicaltrials[@]solidbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 6
• Est. completion date: January 15, 2030
• Est. primary completion date: January 15, 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 7 Years

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• SGT-003
Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of California, Los Angeles Medical Center	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Solid Biosciences Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events (AEs)		Day 360
Secondary	Change from baseline in microdystrophin protein levels	Microdystrophin expression evaluation in muscle biopsies	Day 90, Day 360
Secondary	Change from baseline in North Star Ambulatory Assessment (NSAA) total score	Assessment of muscle function using a 17-item scale	Day 360
Secondary	Change from baseline in stride velocity 95th centile (SV95C)	Assessment of peak ambulatory performance captured by wearable activity monitoring device	Day 360