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SAT-3247 treatment in ambulatory children aged 7–9 with Duchenne muscular dystrophy

A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients

Phase 2 Interventional Satellos Bioscience, Inc. · NCT07287189

This trial will test whether daily SAT-3247 is safe and helps boys aged 7 to 9 with Duchenne muscular dystrophy who can still walk.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment51 (estimated)
Ages7 Years to 9 Years
SexMale
SponsorSatellos Bioscience, Inc. Industry-sponsored
Locations25 sites (Los Angeles, California and 24 other locations)
Trial IDNCT07287189 on ClinicalTrials.gov

What this trial studies

This randomized, double-blind, placebo-controlled phase 2a trial gives once-daily SAT-3247 or matched placebo on weekdays for 12 weeks in ambulatory boys aged ≥7 to <10 with genetically confirmed DMD. Up to 51 participants will be randomized, with stratification by baseline corticosteroid regimen and prior DMD medications; dosing arms differ by region (one dose in US/Canada, two doses in UK/EU/Serbia/Australia). Participants undergo screening, a baseline visit, a Week 1 phone check, and in-person visits at Weeks 4, 8, and 12 to monitor safety, tolerability, and preliminary functional outcomes. The primary aims are to identify an optimal dose and gather safety and early efficacy data.

Who should consider this trial

Good fit: Ambulatory male patients aged 7 to <10 years with a genetically confirmed DMD diagnosis and stable background medications (including stable corticosteroid use or cessation ≥3 months) are ideal candidates.

Not a fit: Children who are non-ambulatory, outside the 7 to <10 age window, have unstable cardiac or respiratory disease, or do not meet medication stability requirements are unlikely to benefit from this trial.

Why it matters

Potential benefit: If successful, SAT-3247 could improve muscle regeneration and help preserve walking ability in young boys with DMD.

How similar studies have performed: Strategies to boost satellite cell–mediated muscle regeneration are relatively novel for DMD with encouraging preclinical results but limited clinical proof-of-concept so far.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.
* Participants receiving a stable dose of givinostat (brand name Duvyzat) for at least 18 months or longer prior to the Screening Visit will be eligible. Participants unable to tolerate givinostat who discontinued treatment before 18 months are eligible to enroll if date of last dose is ≥ 30 days from the Screening date. Givinostat should not be discontinued, if tolerated, to meet study entry criteria.
* Participants that have received prior treatment with an investigational gene therapy product (other than delandistrogene moxeparvovec) ≥ 24 months prior to the Screening Visit.
* If participating in a physical therapy/strength training regimen, must be stable for ≥ 2 months prior to the Screening Visit and for the duration of the trial.

Key Exclusion Criteria:

* Ambulatory patients expected to experience loss of ambulation within ≤ 12 months.
* Participants for whom MRI or open muscle biopsy are contraindicated.
* Evidence of significant hepatic dysfunction, defined as GLDH \> 2X upper limit of normal (ULN) at the Screening Visit.
* Impaired cardiac function defined as a left ventricular ejection fraction of \< 50% on screening cardiac assessments (echocardiogram or MRI) or evidence of symptomatic cardiomyopathy.
* A forced vital capacity \< 60% predicted at the Screening Visit.
* Ongoing participation in any other therapeutic clinical trial or follow-up study for a therapeutic intervention
* Consumption of grapefruit juice or grapefruit containing products
* Severe behavioural or cognitive problems that preclude participation in the study, in the opinion of the investigator.

Additional entry criteria will be reviewed with the clinical site investigator.

Where this trial is running

Los Angeles, California and 24 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Duchenne Muscular DystrophyDuchenneDMDNeuromuscular DiseasesMuscular Dystrophiesmuscle regenerationsatellite cellasymmetric division
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.