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

Solid Biosciences

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Genetic: SGT-001

Study type: Interventional

Funder types: Industry

Identifiers

NCT03368742

GX1001

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: Male

Ages: 4 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

12 participants in 3 patient groups

SGT-001 - Dose Level 1

Experimental group

Description:

Single IV infusion of SGT-001 at starting dose

Treatment:

Genetic: SGT-001

SGT-001 - Dose Level 2

Experimental group

Description:

Single IV infusion of SGT-001 at next ascending dose

Treatment:

Genetic: SGT-001

Untreated Control

No Intervention group

Description:

Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.