Tests SGT-003 gene therapy in boys with Duchenne muscular dystrophy
Tests SGT-003 gene therapy against placebo in boys with Duchenne muscular dystrophy.
Plain-language title generated from the registry record
Registry title: A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Key facts
- Where
- Leiden, NetherlandsSee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for boys aged 7 to 11 with Duchenne muscular dystrophy who can still walk, to test a one-time gene therapy infusion (SGT-003) against placebo.
- Age
- 7 to 11 years
- Sex
- Males only
- Disease stage
- Can still walk. Clinical diagnosis of Duchenne muscular dystrophy (DMD) with a documented DMD gene mutation. Must meet 10-meter walk/run time and time-to-rise-from-lying-down criteria.
- Earlier treatment
- Must have taken prednisone (at least 0.5 mg/kg/day) or deflazacort (0.75 mg/kg/day) by mouth every day for at least 6 months. Cannot have had gene transfer or gene editing therapy before.
- Biomarker or test result
- Negative for antibodies against adeno-associated virus serotype 9 (AAV9). DMD gene mutation documented.
Main reasons for not taking part
- Current or earlier gene transfer or gene editing therapy
- Vamorolone, givinostat, eteplirsen, golodirsen, casimersen, viltolarsen, ataluren or another investigational drug within 6 months or 5 half-lives, whichever is longer
- DMD variant predicted not to express exons 1 to 11, exons 42 to 45, or exons 57 to 69
- Body weight above 50 kg (110 lb)
Read by machine on 9 November 2026.
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?
Source: NCT07160634, who can take part
Where in Netherlands
- Leids Universitair Medisch Centrum (LUMC)Leiden, NetherlandsTaking 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
- 28 September 2026
- 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.