Clinical trials: Congenital Myopathy
Neurologic Diseases, Hemiparesis
NuroSleeve Powered Brace & Stimulation System to Restore Arm Function
The purpose of this study is to investigate if a person with weakness or paralysis in one or both arms, can use the NuroSleeve combined powered arm brace (orthosis) and muscle stimulation system to help restore movement in one…
- Conditions
- Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies
- Trial name
- NCT04798378
- Testing
- Neurosleeve
- Where
- 2 sites in United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Thomas Jefferson University
Duchenne Muscular Dystrophy
Aerobic Exercise in Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in childhood with an estimate incidence of 1 in 3500 to 5000 male births.
- Trial name
- NCT04173234
- Testing
- Aerobic Training, Home exercise program
- Where
- Turkey (Türkiye)
- Volunteers
- 19 people
- Sponsor
- Hacettepe University
Facioscapulohumeral Muscular Dystrophy
Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD)
On the basis of published data and the investigators' results indicating that oxidative stress may contribute to the peripheral skeletal muscle dysfunction in patients with FSHD, the investigators propose a study to test whether…
- Trial name
- FSHD (NCT01596803)
- Testing
- Taking of blood, needle biopsy of the vastus lateralis muscle, Vit C Vit E Zn Se
- Where
- France
- Volunteers
- 54 people
- Sponsor
- University Hospital, Montpellier
Chronic Fatigue Syndrome, Fibromyalgia
Compare the Medical Conditions of Gulf War Veterans to Non-Deployed Veterans
Primary Hypothesis: Gulf War veterans will have an equal prevalence or mean level of the following medical and psychological conditions frequently reported in the literature compared to a control group of nondeployed veterans…
- Conditions
- Chronic Fatigue Syndrome, Fibromyalgia, Post-Traumatic Stress Disorder, Neurologic Abnormalities
- Trial name
- NCT00032461
- Volunteers
- 2,000 people
- Sponsor
- US Department of Veterans Affairs
Hypokalemic Periodic Paralysis, Hyperkalemic Periodic Paralysis
Effect of Physical Training in Individuals With Hypokalemic and Hyperkalemic Periodic Paralysis
This study wishes to investigate the effects of strength exercise in patients with either HypoPP or HyperPP. The investigators wishes to include participants already diagnosed with either HypoPP or HyperPP in af 24 week…
- Trial name
- NCT07194174
- Testing
- Strength exercise
- Where
- Denmark
- Volunteers
- 36 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Duchenne Muscular Dystrophy, Gait Disorders in Children
Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
The aim of this study was to develop a reliable and valid gait classification scale for Duchenne Muscular Dystrophy (QIGS-DMD). The items of the QIGS-DMD were designed based on the literature review considering existing…
- Trial name
- NCT05564962
- Testing
- Quality and Independence of Gait Classification Scale for Duchenne Muscular Dystrophy (QIGS-DMD)
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people
- Sponsor
- Hacettepe University
Muscular Dystrophies
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy
- Trial name
- DEMAND I (NCT01128855)
- Testing
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968
- Where
- 2 sites in United States, France
- Volunteers
- 20 people
- Sponsor
- GlaxoSmithKline
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
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
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD.
- Trial name
- NCT03375164
- Testing
- delandistrogene moxeparvovec
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Glycogen Storage Disease Type III
Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies
Patients suffering from the metabolic myopathy Glycogen Storage Disease type IIIa (GSDIIIa) have a problem releasing sugar stored in cells that is needed for energy production.
- Trial name
- NCT02448667
- Testing
- FAXE Kondi, Faxe Kondi Free
- Where
- Denmark
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Myotonic Dystrophy Type 1
Cerebrospinal Fluid Biomarkers of Myotonic Dystrophy
Myotonic dystrophy is associated with central sleep apnea, excessive daytime sleepiness, diminished working memory, impaired visuospatial skills, and deficits in problem-solving skills.
- Trial name
- NCT06075693
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- Massachusetts General Hospital
Oculopharyngeal Muscular Dystrophy
Screening in Oculopharyngeal Muscular Dystrophy
Oculopharyngeal muscular dystrophy (OPMD) is a rare myopathic disease that results in progressive degeneration of the oral and pharyngeal muscular, resulting in severe dysphagia and dysarthria.
- Trial name
- NCT02877784
- Testing
- Videofluoroscopic swallowing study, Functional Oral Intake Scale, Eating Assessment Tool-10
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
This is a Phase 2 Open-label (Core Phase Plus Extension Phase) With 2 Cohorts Study to Assess the Pharmacokinetics and Safety of Givinostat in younger DMD Patients.
- Trial name
- NCT06769633
- Testing
- Givinostat Hydrochloride, Givinostat Hydrochloride - Cohort 2
- Where
- 9 sites in Belgium, Italy and 2 more
- Volunteers
- 18 people
- Sponsor
- Italfarmaco
Muscular Dystrophy, Duchenne
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an…
- Trial name
- NCT00016653
- Testing
- Creatine Monohydrate, Glutamine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Duchenne Muscular Dystrophy
Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy. II.
- Trial name
- NCT00004646
- Testing
- prednisone
- Volunteers
- 20 people
- Sponsor
- National Center for Research Resources (NCRR)
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Muscular Dystrophy, Duchenne
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will…
- Trial name
- MIS51ON (NCT03992430)
- Testing
- Eteplirsen
- Where
- 59 sites in United States, Colombia and 24 more
- Volunteers
- 160 people
- Sponsor
- Sarepta Therapeutics, Inc.
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