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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 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

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

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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

Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor (Es-Alert)

purpose: Spinal cord injuries and people with Duchenne Muscular Dystrophy or Infant Spinal Muscular Atrophy (ISA) are prone to pain and pressure sores associated with prolonged sitting. For this reason, it is recommended that people with spinal cord injuries release pressure every 15 to 30 minutes and motorized wheelchair users use the electric positioning functions at least 1 minute every hour. The aim is to prevent and/or reduce pain and pres...

sponsor: Assistance Publique - Hôpitaux de Paris

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

Regular Physical Exercise in Duchenne Muscular Dystrophy

purpose:

This study examine whether an evidence-based individual user-preferred exercise program will increase the physical activity level in boys with Duchenne muscular Dystrophy (DMD).

sponsor: Haukeland University Hospital

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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.

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 eac...

sponsor: Pfizer

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.

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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 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, 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

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

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

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Microdystrophin Gene Transfer Study in Adolescents and Children With DMD

purpose: This is a randomized, controlled, open-label, single-ascending dose study to evaluate the safety, tolerability and efficacy of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Eligible patients will be randomized to an active treatment group or an untreated control group. Patients in the active treatment group will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 2 years. P...

sponsor: Solid Biosciences, LLC

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 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

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

Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study

purpose:

Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding of DMD natural history. This study aim to assess the natural history of DMD through a composite assessment tool capable of capturing disease progression linking ambulant and non-ambulant phases of the disease.

sponsor: University College, London

location: France, Netherlands, United Kingdom

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

Prognostic Factors Affecting Duchenne Muscular Dystrophy

purpose:

Determination of prognostic factors affecting ambulation of duchenne muscular dystrophy

sponsor: Assiut University

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 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

An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

purpose: The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study....

sponsor: PTC Therapeutics

location: United States, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, Czechia, France, Germany, Israel, Italy, Republic of Korea, Poland, Spain, Sweden, Switzerland, Turkey, United Kingdom

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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