Clinical Trial Finder

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.

Join The DMD Hub, to stay up-to-date with the latest DMD clinical trials updates.

IMPORTANT NOTICE REGARDING THE IMPACT OF COVID-19 ON CLINICAL TRIALS IN THE UK

The DMD Hub sites have re-started to screen patients for ongoing trials and are working together to move ahead with setting up new trials that were not open to recruitment before March 2020

They are also taking learning from the COVID-19 situation and applying it where they can, such as more home visits. 

Patients should contact sites directly for additional information on studies they believe they would be eligible to participate in. 

The DMD Hub is not promoting any particular trial or therapy.  You should always consult your neuromuscular consultant before joining a trial. Please note that the trial status may vary from site to site and you should contact the individual site for details.
Please remember that this information relies on the sponsors keeping the DMD Hub updated with accurate information.

Location Trial Status:

= Fully recruited

= Recruiting

= Not yet recruiting

= Enrolling by invitation


Showing 11

DMD Hub logo

Italfarmaco- Givinostat (EPIDYS)

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients with Duchenne Muscular Dystrophy (EPIDYS)

Hub Summary

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.

Study Number: NCT02851797

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
Italfarmaco
Age
6-17
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Wave Life Sciences Exon 51

A Multicentre, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy

Hub Summary

This phase 1 study is designed to determine the safety and tolerability of Wave Life Science’s Exon 51 skipping therapy.

Study Number: NCT03508947

Learn More
Overall Trial Status
Trial complete
Trial Sponsor
Wave Life Sciences
Age
5 to 18 years
Mutation Specific
Mutation specific therapies, must be amenable to exon 51 skipping
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Sarepta- ESSENCE

Study of SRP-4045 and SRP-4053 in DMD Patients

Hub Summary

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.

Study Number: NCT02500381

Learn More
Overall Trial Status
Recruiting
Trial Sponsor
Sarepta Therapeutics, Inc.
Age
7-13
Mutation Specific
Mutation specific therapies, Must be amenable to exon 45 or 53 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

FOR-DMD

Finding the optimum regimen for Duchenne Muscular Dystrophy

Hub Summary

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:

  • Prednisone 0.75mg/kg/day
  • Prednisone 0.75mg/kg/day with 10 days on/10 days off treatment
  • Deflazacort 0.9mg/kg/day

Study Number: NCT01603407

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
University of Rochester
Age
4-7
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

DYSTANCE 51 [TERMINATED]

A Randomised, Double-blind, Placebo-controlled, Efficacy and Safety Study of Suvodirsen (WVE-210201) With Open Label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy

Hub Summary

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.

Study Number: NCT03907072

Learn More
Overall Trial Status
Trial terminated
Trial Sponsor
Wave Life Sciences Ltd
Age
5-12 years old
Mutation Specific
Mutation specific therapies, Amenable to exon 51 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Roche- RO7239361 [TERMINATED]

Clinical trial to Evaluate the Efficacy, Safety and Tolerability of RO7239361 in Ambulatory Boys with Duchenne muscular dystrophy

Hub Summary

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.

Study Number: NCT03039686

Learn More
Overall Trial Status
Trial terminated
UK Locations
Trial Sponsor
Hoffmann-La Roche
Age
6-11
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Patient Registry Translarna (Ataluren)

Long-term observational study of Translarna safety and effectiveness in usual care

Hub Summary

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. 

Study Number: NCT02369731

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
PTC Therapeutics
Age
Child, adolescent and adult
Mutation Specific
Mutation specific therapies, Nonsense mutation
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Tamoxifen (TAMDMD)

Tamoxifen in Duchenne Muscular Dystrophy

Hub Summary

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.

Study Number: NCT03354039

Learn More
Overall Trial Status
Recruiting
UK Locations
Trial Sponsor
University Children's Hospital Basel
Age
78 months to 16 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant
DMD Hub logo

Vamorolone Phase 2b (VISION-DMD)

A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

Hub Summary

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. 

Study Number: NCT03439670

Learn More
Overall Trial Status
Fully recruited
Trial Sponsor
ReveraGen BioPharma
Age
4 to 7 years
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
Ambulant
DMD Hub logo

Disease translation in DMD: Neuromuscular rare disease translational research in patients with DMD.

Disease translation in DMD

Hub Summary

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. 

Study Number: Not on clinicaltrials.gov

Learn More
Overall Trial Status
Recruiting
Trial Sponsor
Great Ormond Street Hospital NHS Foundation Trust
Age
>5 years old
Mutation Specific
Non-mutation specific therapies
Muscle Biopsy
No Muscle Biopsy Required
Ambulation
DMD Hub logo

Sarepta - MOMENTUM

A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Hub Summary

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. 

The UK sites have not yet been finalised, we will provide an update once we have these details.

Study Number: NCT04004065

Learn More
Overall Trial Status
Recruiting
UK Locations
Trial Sponsor
Sarepta Therapeutics
Age
4-21 years old
Mutation Specific
Mutation specific therapies, must be amenable to exon 51 skipping
Muscle Biopsy
Muscle Biopsy Required
Ambulation
Ambulant and non-ambulant

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