Summary

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• This summary is based on the review of one randomized controlled trial(s). ^[1]
• In ambulant boys with Duchenne muscular dystrophy, the givinostat group showed a significantly smaller decline in the four-stair climb assessment compared to the placebo group, with a geometric least squares mean ratio of 0.86 (95% CI (Confidence Interval) 0.745-0.989, p=0.035).
• The study focused on male children aged 6 years or older with Duchenne muscular dystrophy, specifically targeting those stratified by baseline vastus lateralis fat fraction (VLFF) between 5% and 30% (Group A), where the primary endpoint demonstrated statistical significance favoring givinostat.
• The most common adverse events in the givinostat group were diarrhea (36%) and vomiting (29%), compared to 18% and 13%, respectively, in the placebo group. No treatment-related deaths were reported.
• The dose of givinostat was reduced following an interim safety analysis, and an ongoing extension study is evaluating the long-term safety and efficacy of the drug.
• The study focused on ambulant boys aged at least 6 years with a genetically confirmed diagnosis of Duchenne muscular dystrophy who had been on systemic corticosteroids for at least 6 months. Participants were stratified by baseline vastus lateralis fat fraction (VLFF), with the primary analysis showing a statistically significant difference favoring givinostat in group A (VLFF >5% but ≤30%). There were no specific findings for other subgroups beyond this stratification.