Early Phase Trial a first for children in Ireland

Children’s Health Ireland (CHI), in partnership with St. James’s Hospital have successfully opened Ireland’s first early phase clinical trial, enrolling eligible children with Duchenne muscular dystrophy (DMD). The global trial, called DELIVER, which started in August 2022 as the first-in-human (phase 1/2) trial of a therapeutic called DYNE-251 at sites around the world targets male children with a specific type of DMD (children amenable to exon 51 skipping). The trial is being delivered by the HRB CHI Clinical Research Centre and the Wellcome HRB Clinical Research Facility at St James’s Hospital and involves delivery of a next generation molecular therapy developed by Dyne Therapeutics, a Massachusetts based biotechnology company, specialising in the development of therapies for rare muscle diseases. The study is progressing through at sites around the world but is the first time such an early phase trial has been conducted in children in Ireland.

Duchene muscular dystrophy (DMD) is a rare neuromuscular disease that results in progressive deterioration in muscle strength and premature death. Symptoms are often first detected as early as age two, but diagnosis often occurs between four and five years of age. The disease is almost exclusively seen in boys. It is caused by gene mutations that affect a protein called dystrophin, which plays a critical role in muscle cell structure and function. As they grow, children with DMD become gradually weaker, losing the ability to walk and ultimately being confined to a wheelchair. Weakness of the muscles involved in swallowing, breathing and heart function are particularly serious and ultimately life threatening. Currently, very few treatments are available for this devastating disease, and children and their families are anxiously awaiting treatments that can target the basic defect in DMD, aimed at slowing or stopping the inevitable decline in muscle function.

Clinical trials can be extremely complex to undertake and are governed by very strict and detailed safety codes. They involve years of careful planning by a large team of experts within the hospitals, regulators and industry. Global healthcare regulatory agencies comprehensively review data on investigational medicines before the clinical trial can start in humans.

The development of clinical trials in Neurology in CHI has been supported by a research grant from Children’s Health Foundation, CHI’s fundraising partner.

CHI’s Director of Research & Innovation, Paul McNally welcomed CHI’s participation in the ongoing study saying:

“This is an exciting day for everyone involved in clinical trials in CHI. These early phase trials are an enormous undertaking but a vitally important thing for us to be able to do if we really want to become a leading children’s hospital and offer our patients the very latest in cutting edge treatments in the world. Our partnership with St James’s Hospital has been critical here and is an example of the type of benefits that we can expect when working together on the same campus when we move to the new children’s Hospital.”

Having previously supported the CHI team to deliver gene therapy to many infants with a rare neurological disorder, the Wellcome HRB Clinical Research Facility at St James’s Hospital is no stranger to advanced paediatric trials.