Duchenne Muscular Dystrophy
Conditions
Keywords
SGT-003, Duchenne Muscular Dystrophy (DMD), adeno-associated virus (AAV), IMPACT DUCHENNE
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.
Interventions
DRUGSGT-003
Adeno-associated virus (AAV)-based gene therapy that delivers a codon-optimized and CpG island-minimized human 5-repeat microdystrophin (h-μD5)
DRUGPlacebo
IV infusion
Sponsors
Solid Biosciences Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
MALE
Age
7 Years to 11 Years
Healthy volunteers
No
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 serotype 9 (AAV9). * 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 variant or variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69, inclusive of the DMD gene as documented by a genetic report. Other Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change From Baseline in Time to Rise (TTR) from Supine Velocity (rise/s) at Day 540 | Baseline, Day 540 |
Secondary
| Measure | Time frame |
|---|---|
| Change From Baseline in Stride Velocity 95th Centile (SV95C) (m/s) at Day 540 | Baseline, Day 540 |
| Change From Baseline in 4-Stair Climb (4SC) 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 |
| Change From Baseline in 10-meter Walk/Run (10MWR) Velocity (m/s) at Day 540 | Baseline, Day 540 |
| Cumulative Loss of Function in NSAA Items at Day 540 | At Day 540 |
| 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 Percent Predicted Forced Expiratory Volume in 1 second (FEV1) at Day 540 | Baseline, Day 540 |
| Change from Baseline in Left Ventricular Ejection Fraction (LVEF) at Day 540[ | Baseline, Day 540 |
| Change from Baseline in Global Longitudinal Strain (GLS) 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), Adverse Events of special interest (AESIs), and clinically significant changes in Electrocardiogram (ECG) and Echocardiography (ECHO) | From first dose up to Day 540 |
| Change From Baseline in Viral Shedding as Measured by Polymerase Chain Reaction (PCR) at Day 360 | Baseline, Day 360 |
| Change From Baseline in Anti-Adeno-Associated Virus (AAV) Antibody Titers at Day 540 | Baseline, Day 540 |
| Change from Baseline in Microdystrophin T-cell Reactivity by Enzyme-Linked Immunospot at Day 540 | Baseline, Day 540 |
Countries
Australia, Canada, United States
Contacts
CONTACTSolid Bio Clinical Trials
Outcome results
None listed