Active Brain & Nervous System NIHR-supported project Digestion, Kidneys & Other Organs

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

Funder NIHR (part of DHSC)
Recipient organisation NIHR Leeds Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Dec 2028

In plain English

AI plain-English summary

A drug already tested in older boys with Duchenne muscular dystrophy is now being given to toddlers as young as two to see if it is safe and how their bodies process it. Duchenne muscular dystrophy is a genetic condition that destroys muscle tissue, leaving boys unable to walk by their early teens and often causing death in their twenties. Current treatment relies on steroids, which slow damage but come with severe side effects. The experimental drug givinostat aims to protect muscle fibres and promote repair by targeting the inflammation that follows the loss of the dystrophin protein. This study is the first to test it in the youngest patients—18 boys aged two to under six—where early intervention might offer the best chance to preserve muscle function before irreversible damage sets in. If givinostat proves safe and well-tolerated in this age group, it could open the door to starting treatment years earlier than currently possible. That shift might slow disease progression from the earliest stages, potentially extending the time boys can walk independently and reducing the severity of later complications. The study also provides crucial data on how the drug behaves in a developing child’s body, information that does not yet exist.

View original technical description
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.

Researchers

Anne-Marie Childs (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

Randomised, double-blind, placebo-controlled, multicentre study to evaluate the efficacy, safety and tolerability of givinostat in non-ambulant patients with Duchenne Muscular Dystrophy
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
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.
Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy
PGN-EDO51-103 - A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple-Ascending Dose Study of PGN-EDO51 with a Long-Term Extension in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)

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