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

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ClinicalTrials.gov Identifier: NCT03368742
Recruitment Status : Recruiting
First Posted : December 11, 2017
Last Update Posted : December 11, 2017
Sponsor:
Information provided by (Responsible Party):
Solid Biosciences, LLC

December 5, 2017
December 11, 2017
December 11, 2017
December 6, 2017
March 2020   (Final data collection date for primary outcome measure)
  • 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
Same as current
No Changes Posted
Not Provided
Not Provided
Not Provided
Not Provided
 
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
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
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.
Not Provided
Interventional
Phase 1
Phase 2
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Active treatment group vs untreated control group.
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
  • Experimental: SGT-001 - Dose Level 1
    Single IV infusion of SGT-001 at starting dose
    Intervention: Genetic: SGT-001
  • Experimental: SGT-001 - Dose Level 2
    Single IV infusion of SGT-001 at next ascending dose
    Intervention: Genetic: SGT-001
  • Experimental: SGT-001 - Dose Level 3
    Single IV infusion of SGT-001 at selected dose
    Intervention: Genetic: SGT-001
  • No Intervention: Untreated Control
    Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
16
Same as current
March 2021
March 2020   (Final data collection date for primary outcome measure)

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 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

Additional inclusion/exclusion criteria may apply.

Sexes Eligible for Study: Male
4 Years to 17 Years   (Child)
No
United States
 
 
NCT03368742
GX1001
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Solid Biosciences, LLC
Solid Biosciences, LLC
Not Provided
Study Director: Genevieve A. Laforet, MD, PhD Solid Biosciences
Study Director: Jorge A. Quiroz, MD, MBA Solid Biosciences
Solid Biosciences, LLC
December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP