SAT-3247 for Duchenne Muscular Dystrophy

Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients

Recruiting Phase 2 Interventional Study
Duchenne Muscular Dystrophy Duchenne DMD Neuromuscular Diseases Muscular Dystrophies
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 taking GlucocorticoidsGivinostat
Rules you out Heart 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.

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About This Trial

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
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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…
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