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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. Identifier: NCT03368742
Recruitment Status : Recruiting
First Posted : December 11, 2017
Last Update Posted : August 24, 2021
Information provided by (Responsible Party):
Solid Biosciences, LLC

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.

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 16 participants
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
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 : December 2023
Estimated Study Completion Date : December 2028

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

No Intervention: Untreated Control
Untreated control group. After 1 year, treatment-eligible control patients will receive SGT-001 at the selected dose.

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

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

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.

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 identifier (NCT number): NCT03368742

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United States, California
David Geffen School of Medicine at UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Ummulwara Qasim    310-825-3264   
Principal Investigator: Perry Shieh, MD, PhD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Stephanie Salabarria    352-273-6582   
Principal Investigator: Barry Byrne, MD, PhD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Ishita Tejani, MSPH, CCRP    312-227-3019   
Principal Investigator: Vamshi Rao, MD         
United States, Massachusetts
University of Massachusetts Medical School-Worcester Recruiting
Worcester, Massachusetts, United States, 01655
Contact: Sarah Figueira    508-856-1604   
Principal Investigator: Brenda Wong, MD         
Sponsors and Collaborators
Solid Biosciences, LLC
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Study Director: Roxana Donisa Dreghici, MD Solid Biosciences
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Responsible Party: Solid Biosciences, LLC Identifier: NCT03368742    
Other Study ID Numbers: GX1001
First Posted: December 11, 2017    Key Record Dates
Last Update Posted: August 24, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked