Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.

The protocol was amended to drop the control arm after 4 participants were dosed.

Study Overview

• Status: Active, not recruiting
• Conditions: Duchenne Muscular Dystrophy
• Intervention / Treatment: Genetic: SGT-001

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90095
      • David Geffen School of Medicine at UCLA
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
• Confirmed absence of dystrophin as determined by muscle biopsy (ambulatory participants)
• Anti-AAV9 antibodies below protocol-specified thresholds
• Stable cardiac and pulmonary function
• Adolescents: non-ambulatory by protocol-specified criteria
• Children: ambulatory by protocol-specified criteria
• Stable daily dose (or equivalent) of oral corticosteroids ≥ 12 weeks

Exclusion Criteria:

• Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant safety, compromise completion of treatment and follow-up, or impair assessment of study results
• Abnormal liver function
• Abnormal renal function
• Clinically significant coagulation abnormalities
• Impaired cardiovascular function based on cardiac MRI or ECHO
• Impaired respiratory function based on FVC % predicted or need for daytime ventilatory support
• Significant spinal deformity or presence of spinal rods
• Body mass index ≥ 95th percentile for age
• Exposure to another investigational drug within 3 months or 5 half-lives prior to screening
• Exposure to drugs affecting dystrophin or utrophin expression within 6 months prior to screening

Additional inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SGT-001 - Dose Level 1; Single IV infusion of SGT-001 at starting dose	Genetic: SGT-001; AAV9 vector containing muscle-specific promoter and microdystrophin construct
Experimental: SGT-001 - Dose Level 2; Single IV infusion of SGT-001 at next ascending dose	Genetic: SGT-001; AAV9 vector containing muscle-specific promoter and microdystrophin construct
No Intervention: Untreated Control; Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Treatment Emergent Adverse Events (TEAEs)	Up to 5 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters	Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Vital Signs	Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations	Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)	Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)	Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)	Baseline, 12 months
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants	Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants	Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score	Baseline, 12 months
Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted	Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography	Baseline,12 months
Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module	Baseline, 12 months

Collaborators and Investigators

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

Publications and helpful links

General Publications

• Boehler JF, Brown KJ, Ricotti V, Morris CA. N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy. Skelet Muscle. 2024 Jan 16;14(1):2. doi: 10.1186/s13395-023-00334-y.

Study record dates

Study Major Dates

• Study Start (Actual): December 6, 2017
• Primary Completion (Estimated): October 15, 2026
• Study Completion (Estimated): October 15, 2026

Study Registration Dates

• First Submitted: December 5, 2017
• First Submitted That Met QC Criteria: December 8, 2017
• First Posted (Actual): December 11, 2017

Study Record Updates

• Last Update Posted (Actual): June 2, 2026
• Last Update Submitted That Met QC Criteria: June 1, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne
• Other Study ID Numbers: GX1001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No