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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):

Study Description
Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: SGT-001 Phase 1 Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Active treatment group vs untreated control group.
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : December 6, 2017
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2021


Arms and Interventions

Arm Intervention/treatment
Experimental: SGT-001 - Dose Level 1
Single IV infusion of SGT-001 at starting dose
Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
Experimental: SGT-001 - Dose Level 2
Single IV infusion of SGT-001 at next ascending dose
Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
Experimental: SGT-001 - Dose Level 3
Single IV infusion of SGT-001 at selected dose
Genetic: SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
No Intervention: Untreated Control
Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.


Outcome Measures

Primary Outcome Measures :
  1. Primary efficacy endpoint [ Time Frame: 12 months ]
    Change from baseline in microdystrophin protein in muscle biopsies (active treatment group)

  2. Primary safety endpoint [ Time Frame: 12 months ]
    Incidence of adverse events

  3. Primary safety endpoint [ Time Frame: 12 months ]
    Incidence of clinical laboratory abnormalities

  4. Primary safety endpoint [ Time Frame: 12 months ]
    Incidence of abnormalities in vital signs

  5. Primary safety endpoint [ Time Frame: 12 months ]
    Incidence of abnormalities in physical examinations

  6. Primary safety endpoint [ Time Frame: 12 months ]
    Incidence of abnormalities on ECGs


Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03368742


Locations
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Stephanie Salabarria    352-273-6582    SGT001Inquiries@peds.ufl.edu   
Principal Investigator: Barry Byrne, MD, PhD         
Sponsors and Collaborators
Solid Biosciences, LLC
Investigators
Study Director: Genevieve A. Laforet, MD, PhD Solid Biosciences
Study Director: Jorge A. Quiroz, MD, MBA Solid Biosciences
More Information

Responsible Party: Solid Biosciences, LLC
ClinicalTrials.gov Identifier: NCT03368742     History of Changes
Other Study ID Numbers: GX1001
First Posted: December 11, 2017    Key Record Dates
Last Update Posted: December 11, 2017
Last Verified: December 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked