The clinical evidence base for Givinostat comprises 4 peer-reviewed publications across 4 journals, 1 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Muscular Dystrophy, Duchenne. Most recent publication: Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons., Ann Clin Transl Neurol, 2025.
Top peer-reviewed publications
Curated set of pivotal-trial result papers and recent publications in high-tier journals.
Histological effects of givinostat in boys with Duchenne muscular dystrophy.
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. · Lancet Neurol · 2024
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
For individuals with Duchenne Muscular Dystrophy, the clinical evidence for Givinostat includes results from a pivotal trial. In NCT02851797, a study investigating Duchenne Muscular Dystrophy, the primary outcome measured was the mean change from baseline in the 4 Standard Stairs Climb (4SC) after 18 months of treatment. In the Givinostat arm of NCT02851797, the mean change from baseline in 4SC climb was 1.27 seconds (least squares mean) after 18 months of treatment. In the placebo arm of NCT02851797, the mean change from baseline in 4SC climb was 1.48 seconds (least squares mean) after 18 months of treatment.
All values are sourced from primary registry reporting; individual papers should be consulted for clinical decisions.
All Givinostat publications (4)
2025 (1 paper)
Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons.
McDonald CM, Guglieri M, Vučinić D, et al. · Ann Clin Transl Neurol · 2025 · Derived
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Mercuri E, Vilchez JJ, Boespflug-Tanguy O, et al. · Lancet Neurol · 2024 · Derived
Publications: ctgov.study_references (PubMed PMIDs auto-attached by ClinicalTrials.gov to each trial), reference types RESULT, DERIVED, and BACKGROUND.
Per-arm outcome values: ctgov.outcome_measurements joined to ctgov.design_outcomes where outcome_type = 'PRIMARY', restricted to phase PHASE3 and PHASE2/PHASE3.
This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).