New pill for duchenne MD enters Mid-Stage trial in young boys
NCT ID NCT07287189
First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 8 times
Summary
This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy who can still walk. The goal is to find the best dose, check safety, and see if it helps muscle strength. Participants take the drug or a placebo for 12 weeks, and all continue their usual steroid treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 51 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2025
- Expected to finish
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Feb 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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7 to 9 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The places running it
21 sites in 8 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Show contact details
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Children's Hospital Eastern Ontario
RECRUITINGOttawa, Ontario, K1H8L1, Canada
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Children's Hospital at Westmead
RECRUITINGWestmead, New South Wales, 2145, Australia
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Clinic of Neurology and Psychiatry for Children and Youth
NOT_YET_RECRUITINGBelgrade, Serbia, 11000, Serbia
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Colorado Children's
RECRUITINGAurora, Colorado, 80045, United States
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Great Ormond Street
RECRUITINGLondon, UK, WC1N 3JH, United Kingdom
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Hospital Infantil i Hospital de la Dona
RECRUITINGBarcelona, 08035, Spain
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Hospital Universitario Donostia
NOT_YET_RECRUITINGDonostia / San Sebastian, Basque Country, 20014, Spain
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Hospital Universitario y Politécnico La Fe
RECRUITINGValencia, Valencia, 46026, Spain
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Hôpital De La Citadelle (CHR)
RECRUITINGLiège, Liège, 4000, Belgium
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Instytut Centrum Zdrowia Matki Polki
RECRUITINGLodz, Łódź Voivodeship, 93-338, Poland
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Klinika Neurologii Rozwojowej Uniwersyteckie
RECRUITINGGdansk, Pomeranian Voivodeship, 80-952, Poland
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Lurie Children's
RECRUITINGChicago, Illinois, 60611, United States
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Mother and Child Health Care Institute
RECRUITINGBelgrade, Serbia, 11070, Serbia
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
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Royal Children's Hospital Melbourne
RECRUITINGMelbourne, Victoria, 3052, Australia
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Seattle Children's
RECRUITINGSeattle, Washington, 98105, United States
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UMass Memorial Medical Center
RECRUITINGWorcester, Massachusetts, 01655, United States
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UZ Gent
RECRUITINGGhent, Oost-Vlaanderen, 9000, Belgium
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University Children's Clinic Tirsova
RECRUITINGBelgrade, Serbia, 11000, Serbia
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University of California Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
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Washington University
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Email: •••••@•••••
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