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.
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IMPORTANT NOTICE REGARDING THE IMPACT OF COVID-19 ON CLINICAL TRIALS IN THE UK
Due to the impact of COVID-19 all clinical trial sites in the UK have implemented some restrictions on screening, randomisation and study visits.
The situation is changing on a daily basis and it is difficult for the sites to provide us with updates proactively. Therefore, in order to ease the burden on sites, we will only update the clinical trail finder if a study is fully recruited or terminated. Patients should be aware that studies currently 'recruiting' on the clinical trail finder may be subject to some restrictions.
Patients should contact sites directly for additional information on studies they believe they would be eligible to participate in.
Patients currently enrolled into a clinical trail will be contacted directly by their site to discuss how the clinical trial is affected at that site.
= Fully recruited
= Not yet recruiting
= Enrolling by invitation
This phase 1 study is designed to determine the safety and tolerability of Wave Life Science’s Exon 51 skipping therapy.
Please note the development of this drug was stopped after this trial failed to reach its objectives.
This placebo-controlled study is designed to assess the efficacy, safety and tolerability of two different doses of RO7239361 in ambulatory males with DMD. RO7239361 is a subcutaneously delivered treatment which inhibits myostatin.
Myostatin is a protein in the body which inhibits muscle growth and it is required to stop muscles from growing too large. Myostatin production increases naturally with age. It is thought that inhibiting myostatin function could lead to increased muscle size and strength in patients with DMD.
This study is designed to study a number of genes considered to be modifiers for DMD. This translational research will identify and obtain DNA samples and clinical information from 400 cases with DMD. This data will then be grouped into clinically and genetically defined groups. The DNA of the participants will be analysed and correlated to motor performance, age at loss of ambulation, severity of respiratory failure and severity of cardiac impairment.
FOR-DMD study is designed to compare three different ways of giving corticosterioids to boys with DMD. The aim of this study is to see which method increases muscle strength the most and which produces the fewest side effects. The results of this study should provide patients and caregivers clearer information and guidelines about the best ways to take corticosteroids. The study will look at the following administration of corticosteroids:
PLEASE NOTE: Recruitment for Group A (ambulant DMD patients) has been completed but recruitment for Group B (non-ambulant, steroid-naive DMD patients) is still open. Please contact the sites if you would like to take part.
This placebo control, 48-week clinical trial will look at the treatment with Tamoxifen for both ambulant and non-ambulant patients with DMD. Tamoxifen has been used to treat breast cancer since the 1980s and is also used for hormonal disorders in pre-pubescent boys. Preliminary data in the DMD mouse model demonstrated that Tamoxifen reduced fibrosis, increased the thickness of muscle fibres, and resulted in a delay in disease progression.
This phase 4 clinical study is designed to assess the safety of Translarna, also known at Ataluren. This study will follow patients who are receiving Translarna as part of their usual care for 5 years. At the patients usual visits, data will be collected to determine the safety and effectiveness of Translarna.
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 Phase 2b study is designed to evaluate the efficacy, safety pharmacodynamics and pharmacokinetics of vamorolone in comparison to corticosteroids and placebo treatments over a 24 week period. The study will also evaluate the persistence of the effect of vamorolone over a period of 48 weeks.
The study is designed to compare 2 different doses of Vamorolone to a standard dose of corticosteroids (prednisone at 0.75 mg/kg/day) and to a placebo. Across all sites, this trial will be recruiting a total of 120 ambulant DMD patients ages 4 to <7 years.
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 study will compare the change in stair climb test and other functional tests in patients taking givinostat and patients taking a placebo. Givinostat has potential anti-inflammatory, antifibrotic and proregenerative effects.
Please note this protocol was amended early 2019.
Stay informed about the latest DMD clinical trials and keep up-to-date with the latest news from the DMD Hub.