Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

Duchenne Muscular Dystrophy Clinical Trial

— IGNITE DMD  

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03368742
• Other study ID #: GX1001
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: December 6, 2017
• Est. completion date: December 2027

Study information

• Verified date: October 2023
• Source: Solid Biosciences Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Patients 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 subjects were dosed. Subjects currently enrolling are assigned to active treatment. Control subjects enrolled under original protocol will continue through the study per the original protocol.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 16
• Est. completion date: December 2027
• Est. primary completion date: December 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years to 17 Years

Eligibility

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 patients)

• 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 wks

Exclusion Criteria:

• Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect subject 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. Patients over 30 kg will not be eligible for treatment at this time. A weight limit of = 18 kg will be implemented for the next two patients to be dosed.

Related Conditions & MeSH terms

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

Intervention

Genetic:
• SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida
United States	David Geffen School of Medicine at UCLA	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	Primary efficacy endpoint	Change from baseline in microdystrophin protein in muscle biopsies (active treatment group)	12 months
Primary	Primary safety endpoint	Incidence of adverse events	12 months
Primary	Primary safety endpoint	Incidence of clinical laboratory abnormalities	12 months
Primary	Primary safety endpoint	Incidence of abnormalities in vital signs	12 months
Primary	Primary safety endpoint	Incidence of abnormalities in physical examinations	12 months
Primary	Primary safety endpoint	Incidence of abnormalities on ECGs	12 months