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NCT Number: NCT03368742

Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants 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 participants were dosed.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

4 year–17 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

David Geffen School of Medicine at UCLA, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 participants)
  • 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 weeks

Exclusion criteria

  • Prior or ongoing medical condition or physical examination, ECG or laboratory findings that could adversely affect participant 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.

Treatment and study plan

SGT-001

Genetic

AAV9 vector containing muscle-specific promoter and microdystrophin construct

Primary outcomes

  1. Number of Participants with Treatment Emergent Adverse Events (TEAEs)

    Time frame: Up to 5 years

Secondary outcomes

  1. Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters

    Time frame: Up to 5 years

  2. Number of Participants with Clinically Significant Abnormalities in Vital Signs

    Time frame: Up to 5 years

  3. Number of Participants with Clinically Significant Abnormalities in Physical Examinations

    Time frame: Up to 5 years

  4. Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)

    Time frame: Up to 5 years

  5. Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)

    Time frame: Baseline, 12 months

  6. Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)

    Time frame: Baseline, 12 months

  7. Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants

    Time frame: Baseline, 12 months

  8. Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants

    Time frame: Baseline, 12 months

  9. Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score

    Time frame: Baseline, 12 months

  10. Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted

    Time frame: Baseline, 12 months

  11. Change from Baseline in Ejection Fraction, As Measured by Echocardiography

    Time frame: Baseline,12 months

  12. Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography

    Time frame: Baseline,12 months

  13. Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography

    Time frame: Baseline,12 months

  14. Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module

    Time frame: Baseline, 12 months

Sponsors and collaborators

Lead sponsor

Solid Biosciences Inc.

Industry

Registry information

Official study 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

Acronym: IGNITE DMD

Important dates

Study start
2017
Primary completion
2026
Study completion
2026
First posted
Dec 11, 2017
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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