What the trial was testing
The EPIDYS enrolled 179 patients with duchenne muscular dystrophy. The study was sponsored by Italfarmaco and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
14% slower decline in four-stair climb time vs. comparison.
The Lancet Neurology · 2024 · NCT02851797
These findings — that decline in four-stair climb time on givinostat vs. inactive comparison — were published in the The Lancet Neurology and represent the headline result of the study.
Researchers tracked outcomes across 179 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with duchenne muscular dystrophy, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Givinostat (Duvyzat) is FDA-approved and available now for Duchenne muscular dystrophy in patients 6 and older. It is given as a twice-daily liquid by mouth. The most common side effects are diarrhea and vomiting. Ask a pediatric neurologist familiar with DMD about access.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open duchenne muscular dystrophy trials
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
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.
CureDuchenne Link®: A Resource for Research
CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).