Clinical trials: Congenital Myopathy
Duchenne Muscular Dystrophy
High-dose Prednisone in Duchenne Muscular Dystrophy
This study will help to determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00110669
- Testing
- Prednisone
- Where
- United States
- Volunteers
- 64 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Myotonic Dystrophy
Extracellular RNA Biomarkers of Myotonic Dystrophy
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies.
- Trial name
- NCT05020002
- Where
- 3 sites in United States
- Volunteers
- 215 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Myotonic Dystrophy 1
Study of ATX-01 in Participants With DM1
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated.
- Trial name
- ArthemiR (NCT06300307)
- Testing
- ATX-01
- Where
- 12 sites in United States, Canada and 5 more
- Volunteers
- 56 people
- Sponsor
- ARTHEx Biotech S.L.
Quality of Life, Muscle Weakness
Effects of Progressive Resistance Training Compared With Aerobic Training in Patients With Hemophilia
This study aims to assess the effects of progressive resistance training compared with aerobic training, among those with hemophilia. Participants will be divided into two groups.
- Trial name
- NCT04892628
- Testing
- Progressive resistance training, Aerobic training
- Where
- Pakistan
- Volunteers
- 60 people
- Sponsor
- Dow University of Health Sciences
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Nemaline Myopathy
Nemaline Myopathy Clinical Research Network (NM-CTRN)
The goal of this study is to establish a research network to help define the natural disease history and clinical outcome measures for Nemaline Myopathy (NM).
- Trial name
- NCT06774703
- Where
- 6 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Stanford University
Fear of Falling, Duchenne Muscular Dystrophy (DMD)
Fear of Falling in Muscular Dystrophy
Primary objectives WP1: Evaluate the prevalence of FOF in the study population and how this varies over time. Evaluate whether there are relationships between the variables investigated (clinical, motor, cognitive, psychological)…
- Conditions
- Fear of Falling, Duchenne Muscular Dystrophy (DMD), Muscular Dystrophies, Rehabilitation
- Trial name
- NCT07129954
- Testing
- Motor rehabilitation, Motor Rehabilitation and Cognitive Behavioral Therapy
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Universita di Verona
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
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).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Duchenne Muscular Dystrophy (DMD)
To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
This study will evaluate the pharmacokinetics plasma profile of 3 treatments: ISO 20, IBU 200 and IBU and ISO combinations (200 +20) given in single dose.
- Trial name
- ISOFEN1 (NCT01478022)
- Testing
- Isosorbide Dinitrate, Ibuprofen, Ibuprofen and Isosorbide Dinitrate combination
- Where
- Italy
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Parent Project, Italy
Duchenne Muscular Dystrophy (DMD)
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients.
- Trial name
- CHERISH (NCT07172971)
- Testing
- SGLT-2 inhibitor, SGLT2 inhibitor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Vanderbilt University Medical Center
Duchenne Muscular Dystrophy
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
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…
- Trial name
- GalaxyDMD (NCT03917719)
- Testing
- Edasalonexent
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 130 people
- Sponsor
- Catabasis Pharmaceuticals
Duchenne Muscular Dystrophy, Muscular Dystrophies
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
This pilot study aims to assess subcellular muscle structure in patients with Duchenne X-linked progressive Duchenne muscular dystrophy (DMD) in comparison to healthy volunteers using multispectral optoacoustic tomography (MSOT).
- Conditions
- Duchenne Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy, Duchenne
- Trial name
- MSOT_DMD (NCT03490214)
- Testing
- Multispectral Optoacoustic Tomography
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- University of Erlangen-Nürnberg Medical School
Duchenne Muscular Dystrophy
Upper Extremity Muscle Strength, Balance and Functional Skills in DMD
This study aims to examine the relationship between upper extremity muscle strength, balance and functional skills of children with DMD.
- Trial name
- NCT06379906
- Testing
- Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 32 people
- Sponsor
- Halic University
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Conditions
- Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Dystrophinopathy
- Trial name
- NCT01557400
- Testing
- Ataluren
- Where
- 21 sites in Australia, Belgium and 8 more
- Volunteers
- 94 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
NSAA NON-Interventional Study Protocol
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT06054971
- Testing
- Remote Administration of NSAA
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Red Nucleus Enterprise Solutions, LLC
Duchenne Muscular Dystrophy (DMD)
A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease for which no curative treatment has yet been identified, making it important to slow progression and improve the quality of life among affected boys and…
- Trial name
- NCT02036463
- Testing
- Prednisone
- Where
- United States
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENERGYC Study
This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of…
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, MELAS, Subacute Necrotizing Encephalomyelopathy
- Trial name
- NCT04846036
- Testing
- Sonlicromanol
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Khondrion BV
Dystrophinopathy, Duchenne Muscular Dystrophy
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids.
- Conditions
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- Trial name
- NCT01388764
- Testing
- L-arginine
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Massachusetts General Hospital
Myotonic Dystrophy 1, Myotonic Dystrophy 2
An Ambispective Natural History Study in Myotonic Dystrophy Patients Linking Retrospective Data Captured From the DM-Scope Registry With a Prospective 24-month Follow-up Period
This natural history observational study is being conducted to follow patients with DM1 or DM2 over a 2 year period to study the presence of myotonia, how it's perceived and its impact on patients quality of life.
- Trial name
- Track-DM (NCT07732439)
- Where
- 6 sites in France
- Volunteers
- 100 people
- Sponsor
- Lupin Ltd.
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