Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

About

Brief 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.

Primary Purpose

Treatment

Study Type

Interventional

Phase

Phase I/II

Eligibility

Gender

Male

Healthy Volunteers

Minimum Age

4 Years

Maximum Age

17 Years

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.

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