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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, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom

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 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

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 Pe...

sponsor: PTC Therapeutics

location: United States

A Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

purpose: The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation an...

sponsor: Catabasis Pharmaceuticals

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, Spain

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A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

purpose: This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. Two dose cohorts are planned with up to 6 subjects for each. In or...

sponsor: Pfizer

location: United States

Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)

purpose: This research study wants to learn more about Duchenne Muscular Dystrophy (DMD) and exercise. Today it is unknown how exercising impacts boys with DMD. The investigators believe that increasing activity and aerobic exercise may help with heart, lung, and muscle function. The investigators are hoping to compare physical strength and blood samples of boys with DMD to see if there are any differences between kids who exercised more as a child ver...

sponsor: Ann & Robert H Lurie Children's Hospital of Chicago

location: United States

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 30 mg/kg of casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

sponsor: Sarepta Therapeutics

location: United States, Italy, United Kingdom

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 72 weeks to boys with DMD who complete Study NS-065/NCNP-01-201.

sponsor: NS Pharma, Inc.

location: United States, Canada

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, wit...

sponsor: PTC Therapeutics

location: United States

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:<br> • Group 1, subjects aged <6 years, 100 subjects; <br> • Group 2, subjects aged >=6 years and <12 years, 180 subjects; <br> • Group 3, subjects aged...

sponsor: Pfizer

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

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

An open-label extension study for patients with Duchenne muscular dystrophy who participated in studies of SRP-5051

purpose:

The purpose of this extension study is to evaluate the safety, tolerability, and pharmacokinetics of repeat administrations of SRP-5051 in patients with Duchenne muscular dystrophy (DMD) who participated in studies of SRP-5051.

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

Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy

purpose: Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cas...

sponsor: Cumberland Pharmaceuticals

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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

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

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

Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

purpose: The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant’s physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compa...

sponsor: Cooperative International Neuromuscular Research Group

location: United States, Argentina, Australia, Canada, India, Israel, Italy, Sweden

Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

purpose:

The objective is to determine whether nebivolol, a beta-blockade drug, can prevent the development of heart disease in patients with Duchenne muscular dystrophy aged 10 to 15 year-old.

sponsor: Assistance Publique - Hôpitaux de Paris

location: France

Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

purpose: The MoveDMD study is a 3-part, Phase 1/2, multi-site study to evaluate the safety, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of edasalonexent (also known as CAT-1004) in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from >=4 to <8 years of age will be enrolled. Edasalonexent is an orally administered small molecule targeted to inhibit activated NF-κB, a molecule that is activated from in...

sponsor: Catabasis Pharmaceuticals

location: United States

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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

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

Pediatric Radio Frequency Coils Generic

purpose:

The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).

sponsor: Children's Hospital Medical Center, Cincinnati

location: United States

Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy

purpose: Duchenne Muscular Dystrophy (DMD) is inherited neuromuscular disorders due to mutation in the gene that encodes critical muscle protein called dystrophin. Currently, there is no effective treatment option for the disease. A pharmacological approach by promoting mRNA translation regardless of the presence of premature stop codons by nonsense mutation, called the readthrough strategy, has been developing recently for DMD with nonsense mutation....

sponsor: Kobe University

location: Japan

(-)- 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

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

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

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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