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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 Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

purpose:

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with non-ambulatory Duchenne muscular dystrophy (age 12 years and older). 

sponsor: FibroGen

A Registered Cohort Study on Duchenne Muscular Dystrophy

purpose:

Dystrophinopathy is a term of X-linked recessive genetic disease, including Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and the X-linked dilated cardiomyopathy. The aim of this study is to determine the clinical spectrum and natural progression of dystrophinopathy in a prospective multicenter natural history study, to assess the clinical, genetic of patients with dystrophinopathy to optimize clinical management.

sponsor: Ning Wang, MD, PhD

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, Inc.

Aerobic Exercise in Duchenne Muscular Dystrophy

purpose: Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births. The effect of aerobic training on muscle architectural properties and motor functions such as muscle activation is not clear in DMD. The aim of this study is to compare with children with DMD and healthy peers in terms of muscle architectural properties and motor functions, and investigate the eff...

sponsor: Hacettepe University

Biomarker for Duchenne Muscular Dystrophy (BioDuchenne)

purpose:

International, multicenter, observational, longitudinal study to identify biomarker/s for Duchenne Muscular Dystropy (DMD) and to explore the clinical robustness, specificity, and long-term variability of these biomarker/s.

sponsor: Centogene AG Rostock

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A Gene Delivery Study to Evaluate the Safety of and Expression From SRP-9001 in Duchenne Muscular Dystrophy (DMD)

purpose:

This is an open-label gene delivery study evaluating the safety of and expression from SRP-9001 in individuals with Duchenne Muscular Dystrophy over 260 weeks.

sponsor: Sarepta Therapeutics, Inc.

A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy (REACH-DMD)

purpose:

The purpose of this study is to evaluate the efficacy and safety of TAS-205 in patients with Duchenne muscular dystrophy.

sponsor: Taiho Pharmaceutical Co., Ltd.

A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

purpose:

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.

sponsor: Pfizer

A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)

purpose:

The primary purpose of this study is to evaluate the safety and tolerability of ASP0367.

This study will also evaluate the pharmacokinetics, pharmacodynamics and efficacy on muscle function of ASP0367.

sponsor: Astellas Pharma Inc

A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From >=6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

purpose:

This study is designed to evaluate safety, tolerability, physical and motor development, and pharmacokinetics (PK) in children aged >=6 months to <2 years treated daily for 52 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

sponsor: PTC Therapeutics

Brain Involvement in Dystrophinopathies (WP5) Part 1

purpose:

The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.

sponsor: University College London Hospitals

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A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications

purpose:

This is an 48-week open-label study to determine the efficacy and safety of casimersen, eteplirsen, or golodirsen for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.

sponsor: Kevin Flanigan

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, Inc.

AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications

purpose:

Open-label, single dose clinical trial of scAAV9.U7.ACCA via peripheral limb vein injection for Duchenne muscular dystrophy boys who have a duplication of exon 2.

sponsor: Megan Waldrop

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, Inc.

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, Inc.

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 4-12 years of age (up to 13th birthday) will be enrolled. Edasalonexent is an orally administered small mole...

sponsor: Catabasis Pharmaceuticals

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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

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 three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Patients who are randomized to placebo in Part 1 will have the opportunity for treatment with SRP-9001 in Part 2.

sponsor: Sarepta Therapeutics, Inc.

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 the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

purpose:

This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years with DMD.

sponsor: ReveraGen BioPharma, Inc.

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 and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function. A total of approximately 30 subjects wi...

sponsor: Pfizer

Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

purpose:

This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.

sponsor: University Children's Hospital, Zurich

location: Switzerland

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Safety and Efficacy of P-188 NF in DMD Patients

purpose:

This is an open-label study to evaluate the safety, tolerability and efficacy of daily, subcutaneous dosing with P-188 NF (Carmeseal-MD™) in non-ambulatory boys with Duchenne Muscular Dystrophy (DMD). This study will determine if continuous treatment with Carmeseal-MD™ can maintain or improve pulmonary function, and skeletal and cardiac muscle function, compared to baseline, in boys 12-25 years of age.

sponsor: Phrixus Pharmaceuticals, Inc.

location: United States

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

The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

purpose:

This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.

sponsor: NS Pharma, Inc.

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, Canada, Israel

Duchenne Muscular Dystrophy Heart Study

purpose: Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020. Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events. Objectives are to describe long-term nat...

sponsor: Hôpital Cochin

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

Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy

purpose: For ambulatory children with DMD, physiotherapy is aimed at protecting ambulation, improving motor performance to the best level and increasing quality of life. The investigators think that the treatment of children with Duchenne Muscular Dystrophy may become more effective with physiotherapy programs based on the comprehensive physiotherapy evaluation results, including the evaluation of fear of falling. This study investigates the fear of fa...

sponsor: Canan İpek

Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies

purpose: Duchenne muscular dystrophy (DMD), caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child. Although the development is rapidly progressive, there is variability in the severity of the disease between DMD patients that do not correlate with the type of mutations in the DMD gene. There are no easily measurable biomarkers for monitoring the DMD or moderate form of the disease, Becker muscular dy...

sponsor: University Hospital, Montpellier

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

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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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 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

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

purpose:

Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.

sponsor: PTC Therapeutics

location: United States

A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)

purpose:

The purpose of this study is to determine whether increasing doses of SMT C1100 are safe, well tolerated and achieve appropriate blood levels in patients with Duchenne Muscular Dystrophy (DMD).

sponsor: Summit Therapeutics

location: United Kingdom

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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

A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy

purpose: The purpose of this low interventional study is to collect data on everyday movement in boys with Duchenne muscular dystrophy (DMD) using wearable activity sensors. The activity sensors could provide useful information beyond what is currently collected by functional (movement, strength) assessments in clinic. This information can help with the understanding of the impact of DMD, and perhaps with how possible treatments can affect this impact....

sponsor: Pfizer

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

purpose: This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose levels will be investigated in a within subject dose escalating fashion. Subjects will be randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 we...

sponsor: Pfizer

location: United States, Australia, Bulgaria, Canada, Italy, Japan, Poland, United Kingdom

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 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).

sponsor: Mallinckrodt ARD LLC

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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