A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Not Applicable
Not yet recruiting
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Drug: Placebo
- Registration Number
- NCT07160634
- Lead Sponsor
- Solid Biosciences Inc.
- Brief Summary
This is a Phase 3, double-blind, placebo-controlled study with the primary objective of evaluating the efficacy of a single IV infusion of SGT-003 in pediatric ambulant male participants with DMD. The secondary objectives include the evaluation of additional efficacy and safety outcomes. The study will be divided into 2 parts. Participants will be randomized 1:1 to either SGT-003 in Part 1 followed by placebo in Part 2 or to placebo in Part 1 followed by SGT-003 in Part 2. Participants will continue to be monitored in long term follow up (LTFU) for at least 5 years from their SGT-003 dosing date.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- Male
- Target Recruitment
- 80
Inclusion Criteria
- Participant is ambulatory.
- Established clinical diagnosis of DMD and documented DMD gene mutation predictive of DMD phenotype.
- Negative for antibodies against adeno-associated virus.
- On a stable daily oral regimen of at least 0.5 mg/kg/day prednisone or 0.75 milligrams per kilogram per day (mg/kg/day) deflazacort for at least 6 months prior to entering the study, allowing for weight-based dose modifications in accordance with clinical practice.
- Meet 10-meter walk/run time criteria.
- Meet time to rise from supine criteria.
- Participant has bodyweight ≤50 kg.
Exclusion Criteria
- Current or prior treatment with an approved or investigational gene transfer drug or gene editing therapy.
- Exposure to vamorolone, givinostat, approved or investigational dystrophin- or disease-modifying drugs (such as eteplirsen, golodirsen, casimersen, viltolarsen, and ataluren), or another investigational drug for any indication within 6 months or 5 half-lives, whichever is longer, prior to enrollment.
- Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or exons 42 to 45, inclusive, of the DMD gene as documented by a genetic report.
Other Inclusion/Exclusion criteria to be applied as per protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description SGT-003 followed by Placebo SGT-003 Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2. SGT-003 followed by Placebo Placebo Enrolled participants will receive a single intravenous (IV) infusion of SGT-003 in Part 1 and a single IV infusion of matching Placebo in Part 2. Placebo followed by SGT-003 SGT-003 Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2. Placebo followed by SGT-003 Placebo Enrolled participants will receive a single intravenous (IV) infusion of matching Placebo in Part 1 and a single IV infusion of SGT-003 in Part 2.
- Primary Outcome Measures
Name Time Method Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 Baseline, Day 540
- Secondary Outcome Measures
Name Time Method Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540 Baseline, Day 540 Change From Baseline in 10-meter Walk/Run Velocity (m/s) at Day 540 Baseline, Day 540 Change From Baseline in 4-Stair Climb Velocity (tasks/s) at Day 540 Baseline, Day 540 Change From Baseline in North Star Ambulatory Assessment (NSAA) total score at Day 540 Baseline, Day 540 Cumulative Loss of Function in NSAA Items at Day 540 At Day 540 Change From Baseline in Microdystrophin Protein Levels by western blot (% of normal dystrophin) at Day 90 Baseline, Day 90 Change from baseline in Percent Predicted Forced Vital Capacity (FVC) at Day 540 Baseline, Day 540 Change from baseline in Percent Predicted Peak Expiratory Flow (PEF) at Day 540 Baseline, Day 540 Change From Baseline in Microdystrophin Tissue Distribution by Immunofluorescence (% positive fibers) at Day 90 Baseline, Day 90 Change from baseline in Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540 Baseline, Day 540 Change from baseline in the Pediatric Outcomes Data Collection Instrument (PODCI) Global score at Day 540 Baseline, Day 540 Number of Participants with Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of special interest (AESIs) From first dose up to Day 540