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Trial record 1 of 1 for:    SGT-001
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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03368742
Recruitment Status : Recruiting
First Posted : December 11, 2017
Last Update Posted : January 12, 2021
Sponsor:
Information provided by (Responsible Party):
Solid Biosciences, LLC

Tracking Information
First Submitted Date  ICMJE December 5, 2017
First Posted Date  ICMJE December 11, 2017
Last Update Posted Date January 12, 2021
Actual Study Start Date  ICMJE December 6, 2017
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 8, 2017)
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
Official Title  ICMJE 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
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:
Active treatment group for all patients enrolled after June 2019. Total of approximately 16 to 32 patients.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
Study Arms  ICMJE
  • 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
  • No Intervention: Untreated Control
    Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 8, 2017)
16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2027
Estimated Primary Completion Date April 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 4 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03368742
Other Study ID Numbers  ICMJE GX1001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Solid Biosciences, LLC
Study Sponsor  ICMJE Solid Biosciences, LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Cathryn M Clary, MD, MBA Solid Biosciences
PRS Account Solid Biosciences, LLC
Verification Date October 2020

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