Givinostat is safe for long-term use and delays disease progression in patients with Duchenne muscular dystrophy (DMD), according to results of a clinical trial extension published in Annals of Clinical and Translational Neurology.
Givinostat is a drug that blocks the activity of histone deacetylases (HDACs), proteins that regulate the expression of several genes, including those that play a role in muscle health. By inhibiting HDACs, givinostat promotes muscle repair and reduces inflammation.
The study consisted of 194 individuals with DMD who previously participated in or were eligible for the Phase 2 and Phase 3 EPIDYS clinical trials, which investigated the efficacy of givinostat versus a placebo. In this open-label extension, all participants received givinostat, along with corticosteroids.
Participants, who had an average age of about 11 years, underwent comprehensive physical examinations every four months to monitor the effects of the drug. Patients received givinostat for an average of 615.6 days during the extension, although some who participated in the earlier trials had received the drug for over eight years.
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The authors compared 142 patients receiving givinostat to 142 individuals identified from datasets documenting the natural progression of disease. They found that across a number of functional measures, those who received givinostat had significantly delayed disease progression. For example, the ability to rise from the floor was extended by a median of two years and the ability to walk independently increased by about 2.9 years.
Findings revealed that 87% of participants reported one or more adverse events, though the majority were mild or moderate. The most common side effects were elevated triglyceride levels in the blood, an increase in falls and diarrhea.
While three participants ultimately withdrew from the study due to adverse drug effects, no life-threatening events were reported at the time of publication. These patients experienced moderate nausea, mild increases in triglyceride levels and atrial fibrillation (the only severe side effect reported), respectively.
“Patients continue ongoing follow-up in this open-label extension study,” the authors noted.
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