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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

key information

study id #: NCT03368742

condition: Duchenne Muscular Dystrophy

status: recruiting


This is a randomized, 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). Eligible patients will be randomized to an active treatment group or an untreated control group. Patients in the active treatment group will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. Patients in the untreated control group who continue to meet treatment criteria will receive SGT-001 after 1 year on study.

intervention: SGT-001

mechanism of action: Gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT03368742

last updated: August 27, 2019

study details

start date: December 6, 2017

estimated completion: March 2020

phase of development: Phase 1/Phase 2

size / enrollment: 16

primary outcomes:

  • Primary efficacy endpoint [Time Frame: 12 months]
    Change from baseline in microdystrophin protein in muscle biopsies (active treatment group)
  • Primary safety endpoint [Time Frame: 12 months]
    Incidence of adverse events
  • Primary safety endpoint [Time Frame: 12 months]
    Incidence of clinical laboratory abnormalities
  • Primary safety endpoint [Time Frame: 12 months]
    Incidence of abnormalities in vital signs
  • Primary safety endpoint [Time Frame: 12 months]
    Incidence of abnormalities in physical examinations
  • Primary safety endpoint [Time Frame: 12 months]
    Incidence of abnormalities on ECGs

inclusion criteria:

• Eligible Sexes:

• 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 of oral corticosteroids >= 24 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

study contacts

sponsor: Solid Biosciences, LLC

contacts: Stephanie Salabarria, 352-273-6582, SGT001Inquiries@peds.ufl.edu

investigators: Genevieve A. Laforet, MD, PhD; Jorge A. Quiroz, MD, MBA

trial center locations: United States