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Clinical Trial NCT03368742 (IGNITE DMD) for Duchenne Muscular Dystrophy is active, not recruiting. See the Trial Radar Card View and AI discovery tools for all the details. Or ask anything here.
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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD) Phase 1, Phase 2 12 Adolescent Open-Label

Active, not recruiting
Clinical Trial NCT03368742 (IGNITE DMD) is designed to study Treatment for Duchenne Muscular Dystrophy. It is a Phase 1 Phase 2 interventional study that is active, not recruiting, having started on 6 December 2017, with plans to enroll 12 participants. Led by Solid Biosciences Inc., it is expected to complete by 15 October 2026. The latest data from ClinicalTrials.gov was last updated on 3 March 2026.
Brief Summary
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.

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

Conditions
Duchenne Muscular Dystrophy
Publications
Scientific articles and research papers published about this clinical trial:
Other Study IDs
  • IGNITE DMD
  • GX1001
NCT ID Number
NCT03368742
Start Date (Actual)
2017-12-06
Last Update Posted
2026-03-03
Completion Date (Estimated)
2026-10-15
Enrollment (Estimated)
12
Study Type
Interventional
PHASE
Phase 1
Phase 2
Status
Active, not recruiting
Primary Purpose
Treatment
Design Allocation
Non-Randomized
Interventional Model
Single Group
Masking
None (Open Label)
Arms / Interventions
Participant Group/ArmIntervention/Treatment
ExperimentalSGT-001 - Dose Level 1
Single IV infusion of SGT-001 at starting dose
SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
ExperimentalSGT-001 - Dose Level 2
Single IV infusion of SGT-001 at next ascending dose
SGT-001
AAV9 vector containing muscle-specific promoter and microdystrophin construct
No InterventionUntreated Control
Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.
N/A
Primary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Up to 5 years
Secondary Outcome Measures
Outcome MeasureMeasure DescriptionTime Frame
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters
Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Vital Signs
Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations
Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)
Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)
Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)
Baseline, 12 months
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants
Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants
Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score
Baseline, 12 months
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
Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography
Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography
Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography
Baseline,12 months
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
Baseline, 12 months
Participation Assistant
Eligibility Criteria

Eligible Ages
Child
Minimum Age
4 Years
Eligible Sexes
Male
  • 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

  • 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.

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No contact data.
2 Study Locations in 1 Countries

California

David Geffen School of Medicine at UCLA, Los Angeles, California, 90095, United States

Florida

University of Florida, Gainesville, Florida, 32610, United States