Official Title: Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy, safety and tolerability of Givinostat in non-ambulant patients with Duchenne Muscular Dystrophy (DMD) (DSC/14/2357/50).

ULYSSES: Upper Limb performance in non-ambulatorY patientS on givinoStat and stEroidS.

Purpose: The main objective of this study is to demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant patients with Duchenne Muscular Dystrophy. Additional objectives are the evaluation of safety, tolerability of the drug and further exploration of efficacy of givinostat in non-ambulant DMD population.

Location: Europe (Belgium, France, Germany, Italy, Netherlands), UK and Canada.

For more information and enrollment status, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT05933057).

Official Title: A randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of Givinostat in ambulant patients with Duchenne Muscular Dystrophy (DMD) (DSC/14/2357/48).

EPIDYS: epigenetic rescue of dystrophin dysfunction.

Purpose: The main objective of this study was to evaluate the efficacy of Givinostat compared to placebo to slow the disease progression in Duchenne Muscular Dystrophy (DMD) ambulant patients. Additional objectives included evaluation of safety, and tolerability of the drug.

Location: Europe (Belgium, France, Germany, Italy, Netherlands, Spain), UK and North America (Canada and US).

For more information and enrollment status, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT02851797) or https://www.clinicaltrialsregister.eu/ctr-search/search.

Official Title: A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD (DSC/11/2357/43).

Purpose: The main objective of the study was to evaluate whether the beneficial histological effects observed with Givinostat in the mdx mouse could be extended to DMD boys.

Location: Europe (Italy).

For more information, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT01761292) or https://www.clinicaltrialsregister.eu/ctr-search/search.

See also the following link: Bettica et al. 2016.

Official Title: Open label, long-term safety, tolerability, and efficacy study of Givinostat in all DMD patients who have been previously treated in one of the Givinostat studies (DSC/14/2357/51).

Purpose: The main objective of the study is to assess the long-term safety and tolerability of Givinostat in patients with DMD following core protocols program and with naïve givinostat DMD subjects i.e. subjects screened in study DSC/14/2357/48 who met all inclusion criteria and none of the exclusion criteria, and never been randomized because the enrolment in the off-target group was completed.

Location: all countries where the previous trial have been conducted.

For more information and enrollment status, please visit www.clinicaltrials.gov (ClinicalTrials.gov Identifier: NCT03373968) or https://www.clinicaltrialsregister.eu/ctr-search/search.