A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Key facts
- Where
- Calgary, Toronto és Vancouver, KanadaSee the 3 sites
- Phase
- 3. fázis
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 7 and 11 years old?
Forrás: NCT07160634: ki vehet részt
Where in Kanada
- Alberta Children's HospitalCalgary, KanadaTaking people
- The Hospital for Sick Children (SickKids)Toronto, KanadaTaking people
- BC Children ' s HospitalVancouver, KanadaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Solid Biosciences Inc.
- Trial code
- NCT07160634EU 2025-522949-22-00
- Record updated
- 2026. szeptember 28.
- Original description
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.Read full original descriptionShow less
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
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.
Inclusion: 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: 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/Exclusion criteria to be applied as per protocol.