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New treatment option for Duchenne Muscular Dystrophy
Official title Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
ClinicalTrials.gov ID: NCT07287189
What this study is testing
What is SAT-3247?
SAT-3247 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 to 9, men only
You may be able to join if
- 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...
- Stable doses of prescription medicines including ACE inhibitors, β-blockers, and diuretics (excluding glucocorticosteroids) and over-the-counter...
- Participants that have previously received delandistrogene moxeparvovec (brand name Elevidys) either in a prior clinical trial or in the commercial...
You likely can't join if
- 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...
- 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
See the full eligibility 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...
- 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...
- 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...
- 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...
- 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...
- 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
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Aurora, Colorado, United States
- Chicago, Illinois, United States
- Worcester, Massachusetts, United States
- St Louis, Missouri, United States
- Columbus, Ohio, United States
- Seattle, Washington, United States
- Westmead, New South Wales, Australia
- Melbourne, Victoria, Australia
- Liège, Liège, Belgium
- Ghent, Oost-Vlaanderen, Belgium
- Ottawa, Ontario, Canada
- Gdansk, Pomeranian Voivodeship, Poland
- Lodz, Łódź Voivodeship, Poland
- Belgrade, Serbia, Serbia
- Donostia / San Sebastian, Basque Country, Spain
- Valencia, Valencia, Spain
- Barcelona, Spain
- London, UK, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 7 years to 9 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Aurora, Colorado, United States; Chicago, Illinois, United States; Worcester, Massachusetts, United States; St Louis, Missouri, United States; Columbus, Ohio, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.