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Clinical Trials

Givinostat for DMD

Phase III Study (ongoing, recruiting)

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.

Epigenetic Rescue Of Dystrophin Dysfunction Logo


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

Location: Europe (Belgium, France, Germany, Italy, Netherlands, Spain and 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.

Phase II Study (ongoing, recruiting closed)

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.

Long-term Study (ongoing, recruiting)

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.

Location:all countries where the previous trial have been conducted (already started in Italy).

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