Summary

Eligibility
for males ages 4-17 (full criteria)
Location
at UCLA
Dates
study started
estimated completion

Description

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.

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

Keywords

Duchenne Muscular Dystrophy Muscular Dystrophies Muscular Dystrophy, Duchenne SGT-001

Eligibility

You can join if…

Open to males ages 4-17

  • 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

You CAN'T join if...

  • 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.

Locations

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

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Solid Biosciences, LLC
ID
NCT03368742
Phase
Phase 1/2 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
Expecting 16 study participants
Last Updated