Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
7 – 9
Sex
Male
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
51 (estimated)
Sponsor
Satellos Bioscience, Inc. · Industry
Who this trial is looking for
This trial is looking for boys aged 7 to under 10 who can walk and have Duchenne Muscular Dystrophy. Participants will take a drug called SAT-3247 for 12 weeks, followed by 9 more months, to see how it works and if it's safe.
Are You a Good Fit for This Trial?
Must be takingGlucocorticoidsGivinostat
Rules you outHeart issuesLiver problems
You may be able to join if
I have Duchenne Muscular Dystrophy confirmed by genetic testing.
I am a boy aged 7 to under 10 years old.
I have been taking glucocorticoids for at least 3 months.
I am currently stable on my prescription medications.
I have not taken certain gene therapies in the last 18 months.
I have not taken an exon skipper in the last 6 months.
I have been stable on givinostat for at least 18 months.
I have been stable in physical therapy for at least 2 months.
You may not be able to join if
I am expected to lose my ability to walk in the next 12 months.
I can't have an MRI or muscle biopsy.
I have significant liver problems.
I have heart issues with a poor ejection fraction.
My lung capacity is below 60% of what's expected.
I am currently in another clinical trial.
I consume grapefruit or grapefruit products.
I have severe behavioral or cognitive problems.
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Phase 2a trial of SAT-3247 in ambulatory DMD patients aged ≥ 7 and \< 10 years. The trial has two parts. In Part 1, 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. In Part 2, the trial will study two doses of SAT-3247 for an additional 9 months.
Trial Locations
Loading…
Loading trial locations…
Facility
City
State
Country
Status
Eligibility 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 glucocorticoi…
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.
Picking one helps us show the most relevant trials first.
Your results are loading in the background — you can
change this any time from the results page.
Set your location to continue
Find My Trials uses your location to surface clinical trials near you.
Add your city or zip code to your profile and try again.