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

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)

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.

Study Overview

• Status: Recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Genetic: SGT-003

• Study Type: Interventional
• Enrollment (Estimated): 6
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Solid Bio Clinical Trials; Phone Number: 617-337-4680; Email: clinicaltrials@solidbio.com

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90095
      • Recruiting
      • University Of California, Los Angeles Medical Center
      • Principal Investigator: Perry Shieh, MD, PhD
      • Contact: Ummi Qasim; Phone Number: 310-825-3264; Email: UQasim@mednet.ucla.edu
  • Ohio
    • Columbus, Ohio, United States, 43215
      • Recruiting
      • Nationwide Children's Hospital
      • Contact: Rox Ann Sullivan; Phone Number: 614-355-3635; Email: RoxAnn.Sullivan@nationwidechildrens.org
      • Principal Investigator: Kevin Flanigan, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1: SGT-003; All participants will receive a single IV infusion of SGT-003 on Day 1.	Genetic: SGT-003; Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)
Experimental: Cohort 2: SGT-003; All participants will receive a single IV infusion of SGT-003 on Day 1.	Genetic: SGT-003; Adeno-associated virus serotype SLB101 containing the human microdystrophin gene (h-µD5)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-emergent adverse events (AEs)	Day 360

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in microdystrophin protein levels	Microdystrophin expression evaluation in muscle biopsies	Day 90, Day 360
Change from baseline in North Star Ambulatory Assessment (NSAA) total score	Assessment of muscle function using a 17-item scale	Day 360
Change from baseline in stride velocity 95th centile (SV95C)	Assessment of peak ambulatory performance captured by wearable activity monitoring device	Day 360

Collaborators and Investigators

• Sponsor: Solid Biosciences Inc.
• Investigators: Study Director: Solid Bio Clinical Trials, Solid Biosciences

Study record dates

Study Major Dates

• Study Start (Estimated): April 5, 2024
• Primary Completion (Estimated): January 15, 2026
• Study Completion (Estimated): January 15, 2030

Study Registration Dates

• First Submitted: November 14, 2023
• First Submitted That Met QC Criteria: November 14, 2023
• First Posted (Actual): November 18, 2023

Study Record Updates

• Last Update Posted (Actual): March 29, 2024
• Last Update Submitted That Met QC Criteria: March 27, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Gene Therapy; DMD
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: SGT-003-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No