Microdystrophin Gene Transfer Study in Adolescents and Children With DMD (IGNITE DMD)

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

Tracking Information

• First Submitted Date: December 5, 2017
• First Posted Date: December 11, 2017
• Last Update Posted Date: August 24, 2021
• Actual Study Start Date: December 6, 2017
• Estimated Primary Completion Date: December 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
• 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

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: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

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: Duchenne Muscular Dystrophy

Intervention

Genetic: SGT-001

AAV9 vector containing muscle-specific promoter and microdystrophin construct

Study Arms

• 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

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: December 8, 2017): 16
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: December 2028
• Estimated Primary Completion Date: December 2023 (Final data collection date for primary outcome measure)

Eligibility 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 (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

• Sexes Eligible for Study: Male

• Ages: 4 Years to 17 Years (Child)
• Accepts Healthy Volunteers: No
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03368742
• Other Study ID Numbers: 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

• Plan to Share IPD: No

• Responsible Party: Solid Biosciences, LLC
• Study Sponsor: Solid Biosciences, LLC
• Collaborators: Not Provided

Investigators

• Study Director: Roxana Donisa Dreghici, MD; Solid Biosciences

• PRS Account: Solid Biosciences, LLC
• Verification Date: August 2021