Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients (NCT07287189) | Clinical Trial Compass
RecruitingPhase 2
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
United States51 participantsStarted 2025-12-08
Plain-language summary
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial will study two doses of SAT-3247 in a randomized, double-blind, placebo-controlled weekday regimen for 12 weeks to determine the optimal dose, safety, tolerability, and preliminary efficacy.
Who can participate
Age range7 Years – 9 Years
SexMALE
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Key Inclusion Criteria:
* Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene.
* Male DMD patients who are ambulatory and aged ≥ 7 to \< 10 years at the time of screening.
* Stable dose of systemic glucocorticoids (i.e., prednisolone, deflazacort, or vamorolone) according to the standard of care for ≥ 3 months prior to the Screening Visit and for the duration of the trial. Patients who are not receiving glucocorticosteroids are also eligible if stopped ≥ 3 months prior to the Screening Visit.
* Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter medicines and/or herbal supplements for supportive care ≥ 1 month prior to the Screening Visit and for the duration of the trial.
* Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial setting \> 18 months prior to screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible.
* Participants that have previously received an exon skipper \> 6 months prior to Screening whose muscle function tests have stabilized or demonstrated decline ≥ 3 months prior to Screening, as determined by investigator and documented in chart notes, will be eligible…