Clinical Trial Finder

Santhera (SIDEROS) [TERMINATED]

A Phase III Double-blind Study with Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) taking Glucocorticoid Steroids

Hub Summary

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.

Study Number: NCT02814019

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

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

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

Interactive Virtual Reality System on Physiotherapy

Investigating the viability and acceptability of an Interactive Virtual Reality (IVR) System on physiotherapy rehabilitation in paediatric Duchenne Muscular Dystrophy (DMD) patients

Hub Summary

This study is looking at the use of Immersive Virtual Reality (IVR) to improve the uptake of physiotherapy amongst young people with DMD. The first phase will involve a design workshop to explore different scenarios on the IVR to mirror current DMD physiotherapy recommendations, with the second phase looking at testing of these scenarios by young people with DMD. The study is currently open to those patients currently being seen at Leeds Teaching Hospital and Sheffield Children's NHS Foundation Trust, and is being funded by The Children's Hospital Charity.

Study Number: Not on clinicaltrials.gov

PepGen - CONNECT2-EDO51

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)

Hub Summary

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.

Study Number: EU Clinical Trial Number: 2023-508383-29

Santhera SIDEROS Open Label Extension [TERMINATED]

Phase III Study with Idebenone in Patients with Duchenne Muscular Dystrophy

Hub Summary

This study is a stage 3 trial of Sanethera's Idebenone drug and it's long term effects in delaying the loss of lung function in patients with DMD, receiving glucocorticoid steroids. This trial is only open to those patients who were part of the SIDEROS trial and are currently taking steroids. 

Study Number: NCT03603288

FibroGen - LELANTOS 2

A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

Hub Summary

This phase 3 study is looking at the efficacy and safety of pamrevlumab versus a placebo, in combination with corticosteroids (deflazacourt or prednisone). The trial is open to patients who are able to complete the 6 Minute Walk test (6MWD) with a distance of at least 270m but no more than 450m on two occasions 3 months before starting on the trial, as well as being able to rise from the floor (TTSTAND) in less than 10 seconds at the screening visit. Patients must also be on a stable dose of corticosteroids for a minimum of 6 months.

There will be a placebo arm of the trial and 50% of the patients will be randomly allocated to each arm. Once all patients have completed the 52-week study, they may be eligible for rollover into an open-label extension (OLE) with pamrevlumab + corticosteroids. 

Pamrevlumab targets connective tissue growth factor (CFGF), which leads to muscle fibrosis. Stopping CTGF can improve muscle function. Data from the open-label Phase 2 clinical trial has shown that lung, heart and upper arm function was better preserved than usually expected in the normal progression of DMD.

Study Number: NCT04632940

Tamoxifen (TAMDMD)

Tamoxifen in Duchenne Muscular Dystrophy

Hub Summary

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

Sarepta - MIS51ON

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients WITH DUCHENNE MUSCULAR DYSTROPHY With Deletion Mutations Amenable to Exon 51 Skip

Hub Summary

This phase 3 study is designed to evaluate the safety and tolerability of two doses of eteplirsen. Part 1 (now closed for recruitment) will investigate two doses with Part 2 comparing the most effective dose from Part 1 with a 30mg/kg dose of eteplirsen. 

Study Number: NCT03992430

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

Antisense - ATL1102

A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of ATL1102 in Non-ambulatory Participants With Duchenne Muscular Dystrophy

Hub Summary

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to 17 years old. The study includes a randomised, double-blind, placebo-controlled treatment period (Part A), followed by an open labelled treatment period (Part B).

Study Number: NCT05938023

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

FibroGen - LELANTOS

A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

Hub Summary

This phase 3 study is looking at the efficacy and safety of pamrevlumab versus a placebo, in combination with corticosteroids (deflazacourt or prednisone). It is only open to non-ambulant patients. There will be a placebo arm of the trial and 50% of the patients will be randomly allocated to each arm. Once all patients have completed the 52-week study, they may be eligible for rollover into an open-label extension (OLE) with pamrevlumab + corticosteroids. 

Pamrevlumab targets connective tissue growth factor (CFGF), which leads to muscle fibrosis. Stopping CTGF can improve muscle function. Data from the open-label Phase 2 clinical trial has shown that lung, heart and upper arm function was better preserved than usually expected in the normal progression of DMD. 

Study Number: NCT04371666

Dyne Therapeutics - DYNE-251

Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Hub Summary

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy, aged 4 to 16 years, Amenable to Exon 51 Skipping.

Study Number: NCT05524883

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