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

purpose:

The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids

sponsor: Santhera Pharmaceuticals

location: United States, Austria, Belgium, Bulgaria, France, Germany, Hungary, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom

A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).

sponsor: Sarepta Therapeutics

location: United States

A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy

purpose: Current DMD therapies are aimed at increasing dystrophin levels and reducing inflammation. Improved anti-inflammatory therapies are needed to safely treat this pathology and delay disease progression. This study will be conducted in a single-centre and assess the safety, efficacy and PK of ATL1102 in non-ambulatory patients with DMD. Read more:...

sponsor: Antisense Therapeutics Limited

location: Australia

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

purpose:

This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and two validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.

sponsor: PTC Therapeutics

location: United States

A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren

purpose:

This study is designed to generate additional data on the effect of ataluren for producing dystrophin protein in nonsense mutation nmDMD participants. This study will evaluate dystrophin levels from participants with nmDMD who currently have been receiving ataluren for greater than or equal to (>=) 9 months.

The study will have a single visit (Visit 1).

sponsor: PTC Therapeutics

location: United States

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy

purpose:

This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD). Research: Anti-inflammatory

sponsor: Mallinckrodt

location: United States, Bulgaria, Israel, Italy, Mexico, Serbia, Spain, Turkey

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An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

purpose:

The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

sponsor: Sarepta Therapeutics

location: United States, Belgium, Italy, Spain, United Kingdom

An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (GalaxyDMD)

purpose: The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Patients who completed CAT-1004-201 or CAT-1004-301 or siblings of these boys from 4-10 years of age (up to 11th birthday) will be enrolled. Edasalonexent is an orally administered small mole...

sponsor: Catabasis Pharmaceuticals

location: United States

Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker

purpose: Duchenne’s Muscular Dystrophy and Becker Dystrophy, hereafter DMD and BMD, is a serious and progressive disease that affects 1 in 3,500-6,000 males born alive. Scale 6-minute walking test, is used for determine the inclusion of children with DMD in pharmacological studies. Furthemore, is used to verify a training effectiveness assessing muscular endurance and cardio-respiratory functions. This Research evaluates the feasibility and effectivene...

sponsor: University of Malaga

location: Spain

Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

purpose:

This long-term extension study is an open-label, multiple‑dose study to evaluate the long‑term safety, tolerability, efficacy and PD of vamorolone administered once daily by liquid oral suspension over a Treatment Period of 24 months to young boys with DMD who participated in the VBP15‑002 Phase IIa and VBP15-003 Phase IIa extension core studies.

sponsor: ReveraGen BioPharma, Inc.

location: United States, Australia, Canada, Israel, Sweden, United Kingdom

Study of ataluren in previously treated participants with nonsense mutation dystrophinopathy (nmDBMD)

purpose: The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan. The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, w...

sponsor: PTC Therapeutics

location: United States, Canada

A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy

purpose: This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients. A total of approximately 330 subjects will be enrolled with the target number of subjects in each group as below: • Group 1, subjects aged <6 years, 100 subjects; • Group 2, subjects aged >=6 years and <12 years, 180 subjects; • Group 3, subjects aged >=12 years, 50 subjects...

sponsor: Pfizer

A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 (Suvodirsen) in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

purpose:

This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping intervention (DYSTANCE 51)

sponsor: Wave Life Sciences Ltd.

A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients

purpose: This study will be comprised of 2 parts: Part 1 will be conducted to evaluate the safety and tolerability of two doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 8 patients; Part 2 will be conducted for the selection of a high dose (high dose level 1 vs high dose level 2) (dose finding phase), and its comparison with the 30 mg/kg dose of eteplirsen (dose comparison phase), in approximately 144 DMD patients with ge...

sponsor: Sarepta Therapeutics

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy

purpose:

The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)

sponsor: Sarepta Therapeutics

Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback

purpose:

Our study is a randomized controlled study and the subjects included in the study will be divided into three groups as virtual reality training, biofeedback training, and conventional rehabilitation.

sponsor: Merve Kurt

Natural History of Duchenne Muscular Dystrophy

purpose:

Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 6 Years over a period of 6 to 24 months using disease appropriate evaluations.

sponsor: Genethon

location: France, United Kingdom

view all (11)

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2)

purpose:

HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

sponsor: Capricor Inc.

location: United States

Becker Muscular Dystrophy – A Natural History Study to Predict Efficacy of Exon Skipping

purpose:

This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.

sponsor: Cooperative International Neuromuscular Research Group

location: United States, Canada, Italy, United Kingdom

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

purpose:

Longitudinal prospective observational study. This is a 24-month study with the possibility of extending the data time points. Initially baseline, then 12 and 24 months follow up studies will be completed.

sponsor: Nationwide Children's Hospital

location: United States

Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

purpose: This is a pilot clinical trial to assess the ability of a new ultrasound-based imaging method, Double-Push Acoustic Radiation Force (DP ARF) ultrasound, to monitor the progression of Duchenne muscular dystrophy. The hypothesis being tested is that DP ARF ultrasound delineates changes in muscle composition and function in individual dystrophic muscles, from early through late stages of disease development, that correlate to time to loss of ambu...

sponsor: University of North Carolina, Chapel Hill

location: United States

Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

purpose:

This is an open-label, extension study of NS-065/NCNP-01 administered intravenously once weekly for an additional 144 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

sponsor: NS Pharma, Inc.

location: United States, Canada

Finding the Optimum Regimen for Duchenne Muscular Dystrophy

purpose: The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to determine which of the three ways increases muscle strength the most, and which causes the fewest side effects. Using the results of this study, the investigators aim to provide patients and families with clearer information about the best way to take these drugs....

sponsor: University of Rochester

location: United States, Canada, Germany, Italy, United Kingdom

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Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

purpose:

The expanded access program will provide access to treatment with deflazacort in children, adolescent, and adult patients with DMD in the U.S. who are ineligible, unable, or otherwise unwilling to enroll in a clinical study examining the efficacy of deflazacort while a new drug application is under preparation and review. Enrollment is open to all eligible patients.

sponsor: PTC Therapeutics

location: United States

Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)

purpose:

The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD.

sponsor: Santhera Pharmaceuticals

location: United States

Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

purpose:

The intent of this protocol is to provide continued access to vamorolone for subjects in the United States who Have Completed the VBP15-LTE or VBP15- 004 protocols (and are thereby ineligible to enroll in another trial of vamorolone therapy), during the time a new drug application for vamorolone is under preparation and review.

sponsor: ReveraGen BioPharma, Inc.

location: United States

Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

purpose:

This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Both of these properties could lead to potential benefits for a DMD patient.

sponsor: Allergy and Asthma Consultants, Wichita, Kansas

location: United States

Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

purpose:

We will utilize the Cooperative International Neuromuscular Research Group (CINRG) network to collect and store tissue and blood from patients with Duchenne muscular dystrophy (DMD) with specific genetic mutations within the dystrophin gene that could be treated by antisense oligonucleotide (AO) drugs.

sponsor: Cooperative International Neuromuscular Research Group

location: United States, Canada

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

purpose:

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28 week eteplirsen study: Study 4658-us-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.

sponsor: Sarepta Therapeutics

location: United States

Exploratory Study of NS-065/NCNP-01 in DMD

purpose:

This study is designed to assess the safety, tolerability, efficacy and pharmacokinetics (PK) of NS-065/NCNP-01 in subjects diagnosed with Duchenne muscular dystrophy (DMD).

sponsor: National Center of Neurology and Psychiatry, Japan

location: Japan

Muscle Oxygenation in Effort in Neuromuscular Diseases

purpose: Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin. Our study aim at assessing muscle oxygenation during effort in different neuromuscular diseases (muscular dystrophies related and not related to dystrophin, non dystrophic myopathies and motor neuron diseases) compared to a group of healthy controls. Patients and controls are invited to perform an infr...

sponsor: University Hospital, Lille

location: France

Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

purpose:

PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.

sponsor: Genethon

location: France, United Kingdom

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(-)- Epicatechin Becker Muscular Dystrophy

purpose: This is a 48-week open-label extension of our initial proof-of-concept study (UCD0113) in patients with Becker muscular dystrophy who participated in the earlier trial. This single center study will enroll up to 10 adults who will receive the purified nutritional extract (-)-epicatechin 100mg/day orally for 8 weeks. After screening visits, participants will be enrolled in the study if they meet all inclusion criteria. They will be evaluated at...

sponsor: Craig McDonald, MD

location: United States

2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children

purpose:

Compare systolic function of left ventricle (LV) and right ventricle (VD) by 2D strain evaluation in Duchenne muscular dystrophy children versus a control group.

sponsor: University Hospital, Montpellier

location: France

A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD

purpose:

This is a 2-part, phase 2 study to assess the effects of Givinostat on muscle histologic parameters and on clinical parameters in ambulant children with DMD. The safety, tolerability, and pharmacokinetics of Givinostat will also be assessed. All children treated in part 2 continue in the extension phase, for a maximum of an additional 12 months.

sponsor: Italfarmaco

location: Italy

A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

purpose:

The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

sponsor: GlaxoSmithKline

location: Argentina, Belgium, Brazil, Canada, Chile, Czechia, Denmark, France, Germany, Hungary, Italy, Japan, Republic of Korea, Netherlands, Norway, Poland, Russian Federation, Spain, Taiwan, Turkey

A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study.

sponsor: GlaxoSmithKline

location: United States

A Device for Rapid, Painless, Bedside Muscle Evaluation of Children

purpose:

The purpose of this protocol is to perform Electrical Impedance Myography (EIM) testing on healthy children and children with duchenne muscular dystrophy so as to develop a new, convenient tool for the office based assessment of children with a wide variety of neuromuscular conditions.

sponsor: Skulpt, Inc.

location: United States

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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of the study is to see whether BMN053 is safe and effective to use as medication for Duchenne muscular dystrophy (DMD) patients with a mutation around location 53 in the DNA for the dystrophin protein.

sponsor: BioMarin Pharmaceutical

location: Belgium, France, Italy, Netherlands, United Kingdom

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy

purpose:

To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.

sponsor: BioMarin Pharmaceutical

location: United States, Argentina, Belgium, Brazil, France, Germany, Italy, Netherlands, Sweden, Turkey

A Study of Tadalafil for Duchenne Muscular Dystrophy

purpose: The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD). The study will also assess the safety of tadalafil and any side effects that might be associated with it in boys who have DMD. Participants will receive study treatment (tadalafil or placebo) for the first 48 weeks of the study, and can then continue into an open label extension (OLE) that cons...

sponsor: Eli Lilly and Company

location: United States, Argentina, Belgium, Canada, France, Germany, Italy, Japan, Republic of Korea, Netherlands, Puerto Rico, Russian Federation, Spain, Taiwan, Turkey, United Kingdom

A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

purpose: This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876, DMD114044 and DMD114349. Subjects will receive 6mg/kg subcutaneous drisapersen on a weekly basis. For subjects who have previously experienced significant safety or tolerability issues or who experience these during the stud...

sponsor: BioMarin Pharmaceutical

location: United States, Canada

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

purpose: This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo...

sponsor: Pfizer

location: United States, Canada, Italy, Japan, United Kingdom

CoQ10 and Prednisone in Non-Ambulatory DMD

purpose: This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD. Participants who are enrolled in this study should not have taken any corticosteroids within the last six months. This is a 13-month, prospective, randomized study comparing a daily prednisone arm (0.75mg/kg/day), a CoQ10 arm (serum of greate...

sponsor: Cooperative International Neuromuscular Research Group

location: United States

view all (22)

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) (PTCEMF)

purpose: The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males with DMD aged greater than or equal to (>=) 2 to lesser than (<) 5 years. The study will comprise of 2 periods (Period 1: 52-week safety and pharmacokinetics [PK], and...

sponsor: PTC Therapeutics

Stem Cell Therapy in Duchenne Muscular Dystrophy

purpose:

The purpose of this study was to study the effect of stem cell therapy in patients with Duchenne Muscular Dystrophy.

sponsor: Neurogen Brain and Spine Institute

location: India