Trial of givinostat to treat DMD patients between 2 to 6 years old
Research type
Research Study
Full title
A Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Than 6 Years Old
IRAS ID
1010236
Contact name
Serena Franciosi
Contact email
Sponsor organisation
Italfarmaco SpA
Eudract number
2024-511823-32
Clinicaltrials.gov Identifier
Research summary
The purpose of the study is to find out about the safety and tolerability of givinostat for the treatment of Duchenne muscular dystrophy (DMD) in patients aged from at least 2 years old to less than 6 years old. The study will also evaluate how the body absorbs, distributes, breaks down and eliminates givinostat.
Givinostat is experimental, which means that it is not approved by health authorities/competent authorities including the UK Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of DMD in patients that are 2 to less than 6 years old.
DMD is a genetic disorder which causes muscle degeneration and weakness due to the changes of a protein called dystrophin. Dystrophin helps keep muscle cells intact. Lack of dystrophin causes repetitive muscle damage and can lead to inflammation and the breakdown of muscle fibers which then get replaced by fat and connective tissue (tissue that supports, protects, and gives structure to other tissues and organs in the body). The regular treatment for DMD usually includes a corticosteroid. Givinostat has been developed to treat DMD by helping to protect the muscle fibers and promote muscle regeneration.
Approximately 18 males aged from at least 2 years old to less than 6 years old will take part in this study at several different locations internationally.REC name
West of Scotland REC 1
REC reference
24/WS/0086
Date of REC Opinion
8 Aug 2024
REC opinion
Further Information Favourable Opinion