Clinical trials: Congenital Myopathy
Myotonic Dystrophy Type 1
Multicenter Observational Study of Myotonic Dystrophy Type 1
The purpose of the study is to determine the best ways to assess how people are affected by myotonic dystrophy type 1 (DM1). The study will assess walking speed, muscle strength, muscle size, myotonia, heart rhythm, mental…
- Trial name
- MOS-DM1/POP (NCT02308657)
- Where
- 6 sites in United States
- Volunteers
- 120 people
- Sponsor
- University of Rochester
Muscular Dystrophy, Duchenne
Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether long-term oral glutamine supplementation is effective in improving muscle mass and function in children with Duchenne muscular dystrophy (DMD).
- Trial name
- MDB-GLN (NCT00296621)
- Testing
- L-Glutamine
- Where
- 5 sites in France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hypokalemic Periodic Paralysis
Bumetanide in Hypokalaemic Periodic Paralysis
This is a randomised, double-blind, placebo-controlled phase II clinical trial with a cross-over design to investigate the efficacy of bumetanide in patients with hypokalemic periodic paralysis (HypoPP).
- Trial name
- NCT02582476
- Testing
- Bumetanide
- Where
- United Kingdom
- Volunteers
- 12 people
- Sponsor
- University College, London
Tobacco Use Disorder/Cigarette Smoking
Smoking Cessation Intervention for Young Adults Using MTM: A Randomized Controlled Trial
The goal of this clinical trial is to evaluate whether a Multi-Theory Model (MTM)-based behavioral intervention can effectively promote smoking cessation and support behavior change in young adult cigarette smokers aged 18 to 29…
- Trial name
- NCT07104006
- Testing
- Multi-Theory Model (MTM)-Based Smoking Cessation Program
- Where
- United States
- Volunteers
- 144 people
- Sponsor
- University of Nevada, Las Vegas
Myotonic Dystrophy Type 1
PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study)
PhenoDM1 will use patient reported outcomes to assess levels of pain, fatigue and quality of life in this cohort. Clinical and functional outcomes will look at muscle wasting and levels of myotonia.
- Trial name
- PhenoDM1 (NCT02831504)
- Where
- 2 sites in United Kingdom
- Volunteers
- 213 people
- Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18…
- Conditions
- Duchenne Muscular Dystrophy (DMD), Muscular Dystrophy, Duchenne, Muscular Dystrophy (DMD), DMD
- Trial name
- FORZETTO (NCT07608432)
- Testing
- Zeleciment Rostudirsen (DYNE-251)
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Dyne Therapeutics
Myotonic Dystrophy 1
Venous Thromboembolism in Myotonic Dystrophy Type 1
Investigators identified a high risk of deep vein thrombosis and pulmonary embolism in patients presenting myotonic dystrophy type 1 treated in our hospital, 10 times higher than general population matched on age and sex.
- Trial name
- DM1-VTE (NCT03424460)
- Testing
- Haemostasis tests, Monocytes and megacaryocytes culture and RNA extraction, RNA extraction
- Where
- France
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Myotonic Dystrophy 1
Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1
Eleven men with myotonic dystrophy type 1 (DM1) underwent a 12-week lower-limb strength training program. The training program consisted of 3 series of 6 to 8 maximal repetitions of 5 different exercises: Leg extension, leg…
- Trial name
- NCT04018820
- Testing
- Training program
- Where
- Canada
- Volunteers
- 11 people
- Sponsor
- Élise Duchesne
Centronuclear Myopathy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD)/preliminary efficacy of…
- Trial name
- Unite-CNM (NCT04033159)
- Testing
- DYN101
- Where
- 8 sites in Belgium, Denmark and 4 more
- Volunteers
- 14 people
- Sponsor
- Dynacure
Oculopharyngeal Muscular Dystrophy
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
BB-OPMD-202 is a randomized, double-blind, placebo-controlled study of IV trehalose for treatment of OPMD. The study includes a 4-week screening period, a 24-week blinded treatment period during which patients will receive weekly…
- Trial name
- NCT04226924
- Testing
- Trehalose
- Where
- 3 sites in Canada
- Sponsor
- Bioblast Pharma Ltd.
Duchenne Muscular Dystrophy (DMD)
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy…
- Trial name
- NCT07911995
- Testing
- Tegacorat
- Volunteers
- 36 people
- Sponsor
- Grünenthal GmbH
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
- Trial name
- NCT06641895
- Testing
- BBM-D101
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine
Duchenne Muscular Dystrophy
A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
This is a multicenter, open-label, non-randomized study to investigate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of SGT-003 in participants with Duchenne muscular dystrophy.
- Trial name
- NCT06138639
- Testing
- SGT-003
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 60 people
- Sponsor
- Solid Biosciences Inc.
Duchenne Muscular Dystrophy, Spinal Muscular Atrophy
Flu Vaccine Study in Neuromuscular Patients 2011
The purpose of the study is to compare the immune response of two different injection methods (Intramuscular V.S. Subcutaneous) of the 2011-2012 seasonal Influenza (Flu) vaccine among patients with neuromuscular conditions who…
- Conditions
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Congenital Muscular Dystrophy
- Trial name
- NCT01422200
- Testing
- 2011-2012 seasonal flu vaccine Subcutaneous, 2011-2012 seasonal flu vaccine Intramuscular
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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 (DMD)
A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin.
- Trial name
- TODDLER (NCT07423026)
- Where
- United Kingdom
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of Oxford
Muscular Dystrophy, Facioscapulohumeral
Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as the aberrant expression of a…
- Trial name
- ReSOLVE_France (NCT04038138)
- Testing
- Validation of new COA for FSHD patients
- Where
- 3 sites in France
- Volunteers
- 100 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Primary Familial Dilated Cardiomyopathy
Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities
The purpose of this Phase 2a study is to establish safety and preliminary efficacy of treatment with danicamtiv in patients with primary dilated cardiomyopathy (DCM) due to MYH7 or TTN variants or other causalities.
- Trial name
- NCT04572893
- Testing
- danicamtiv
- Where
- 19 sites in United States, Germany and 2 more
- Volunteers
- 41 people
- Sponsor
- Bristol-Myers Squibb
Muscular Dystrophy, Facioscapulohumeral
Study of Morphology and Functional Magnetic Resonance Imaging (MRI) Muscle Patients With Muscular Dystrophy Type FSHD Benefiting a Physical Training Introduced.
The safety of guided practice of physical activity in myopathies is increasingly accepted, including muscular dystrophies. In facioscapulohumeral dystrophy (FSHD), one of the most common muscular dystrophy, the aerobic training…
- Trial name
- FSHD3 (NCT01990976)
- Testing
- MRI, Biopsy
- Where
- 2 sites in France
- Volunteers
- 15 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Becker Muscular Dystrophy, Duchenne Muscular Dystrophy
Gentamicin Treatment of Muscular Dystrophy
This study will evaluate the antibiotic gentamicin for treating patients with muscular dystrophy caused by a specific genetic abnormality known as a nonsense mutation.
- Trial name
- NCT00005574
- Testing
- Gentamicin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
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