The DMD Hub’s Clinical Trial Finder brings together trustworthy and reliable information on all existing and upcoming trials for Duchenne muscular dystrophy in the UK.
Our Clinical Trial Finder has been designed for patients and caregivers, to be as accessible and comprehensive as possible. Every trial has information on outcome measures, inclusion criteria and an easy to understand lay summary. You can use search filters to find trials that are relevant to you and download a fact sheet for each trial. The information on each trial has been sourced directly from industry and hospitals and is verified by Duchenne UK and the DMD Hub management team.
Please note that the DMD Hub is not responsible for the direct recruitment of patients to trials. Although we work closely with sites to ensure the recruitment status for every trial is accurate and up to date, there may be a delay in updating the Clinical Trial Finder while the patient screening process takes place.
We recommend that UK patients/parent and caregivers register with the Central Recruitment Pilot Project, which will enable trial sites to contact you directly if you are eligible for a study.
The DMD Hub is not promoting any particular trial or therapy. You should always consult your neuromuscular consultant before joining a trial.
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= Fully recruited
= Recruiting
= Not yet recruiting
= Enrolling by invitation
The SIDEROS trial is designed to determine the effect of idebenone at delaying the loss of lung function in patients with DMD, receiving glucocorticoid steroids. This is a placebo-controlled trial.
Please note that Wave have stopped the development of this drug after the Phase 1 Open-label extension failed to meet its primary endpoint.
DYSTANCE 51 is a phase 2/3 clinical trial designed to evaluate the efficacy and safety of WVE-210201 (suvodirsen) in ambulant boys with DMD mutations amenable to exon 51 skipping.
DYSTANCE 51 is comprised of two phases, a placebo-controlled phase and an open-label phase. In the placebo-controlled phase, patients will be randomized to receive suvodirsen 3 mg/kg, suvodirsen 4.5 mg/kg or placebo for 48 weeks. Dystrophin protein levels and functional outcomes will be assessed for each patient throughout the initial 48-week treatment period.
Each participant will have two biopsies in total, one at baseline, and one at either week 12, 22, or 46.
Following completion of the placebo-controlled phase of the study, patients will enter the open-label phase to receive ongoing treatment with suvodirsen. There will be no placebo or biopsies in the open label phase. However, functional assessments will continue.
This study is a stage 3 trial of Sarepta's exon 45 and exon 53 skipping drugs. Exon skipping drugs use a small piece of genetic material to skip over the part of the dystrophin gene with a mutation. The part of the dystrophin gene with a mutation varies between patients. Therefore, exon skipping trials are mutation specific. This trial requires you to be amenable to the skipping of exon 45 or 53.
The main objective of this study is to determine the efficacy of the drugs compared to a placebo in DMD patients.
This is a phase 1b/2a looking at the safety of a new exon skipping investigational therapy, WVE-N531. Wave will also be looking at the pharmacokinetics (how the drug is absorbed and metabolised in the body) and pharmacodynamics (how the drug affects the body).
Only patients who have a mutation amenable to exon 53 skipping are able to participate.
Over the course of the trial, they will look to find the best dose of the investigational therapy, so patients will receive up to 4 dose levels, 4 weeks apart over the course of 16 weeks, and then receive an additional 3 doses every other week.
This decentralised study aims to evaluate whether new video-based electronic clinical outcome assessments (eCOAs) captured with the Atom5TM platform DMDhome are able to detect disease progression from the late ambulatory to transfer stage and early non-ambulatory stage, compared with standard validated clinical assessments.
Learn MoreThe main objective of this study is to demonstrate the efficacy of givinostat in reducing muscle decline in non-ambulant patients aged 9-17 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.
This phase 2 study is designed to determine the maximum dose for Sarepta Therapeutics Exon 51 skipping therapy, as well as its safety and tolerability.
There will be two arms to the study - in Part A, patients will receive 1 of 5 doses of SRP-5051 monthly by intravenous infusion. Once the maximum dose has be has been determined, all patients will then roll over into Part B and will receive the maximum dose by intravenous infusion for 24 weeks. In Part B, an additional 15 patients will also be enrolled at the beginning of the study.
Part A recruitment has now been completed and Part B will be beginning soon, involving the original patients from Part A as well as some additional patients.
The UK sites have not yet been finalised, we will provide an update once we have these details.
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.
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.
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This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants (Aged up to 3 years of age) will be in the study for approximately 264 weeks.
The CONNECT2-EDO51 Phase 2 clinical trial is a multinational, randomized, double- blind, placebo-controlled, multiple ascending dose (MAD) study, that will enroll ambulatory and non-ambulatory boys and young men living with DMD amenable to exon 51-skipping, who are at least six years of age. Participants will receive seven doses of either PGN-EDO51 or placebo at approximately four-week intervals for 24 weeks. Participants will provide a muscle biopsy at baseline and then at week 25. The trial will evaluate the safety and tolerability of PGN-EDO51 and the levels of dystrophin in skeletal muscle following repeat dosing. All participants will have the opportunity to participate in an open-label extension for 108 weeks after completing the MAD period where all participants will receive only the investigational study drug PGN-EDO51.
This study will evaluate the safety and efficacy of gene transfer therapy in boys aged between 4 and 7 with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.
Entrada Therapeutics is developing an exon 44 skipping therapy (called ENTR-601-44) for people living with Duchenne. Its goal is to help the body make a shorter, but still potentially functional dystrophin protein. Dystrophin is important because it helps keep muscles strong and stable. The ENTR-601-44-201 study (also called ELEVATE-44) is a global, two-part, randomized, double-blind placebo-controlled, Phase 1/2b study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in people living with Duchenne who are amenable to exon 44 skipping. |
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