Clinical trials: Congenital Myopathy
Oculopharyngeal Muscular Dystrophy
Natural History Study of Oculopharyngeal Muscular Dystrophy
The objective of this study is to test a set of clinical outcome measures longitudinally in a cohort of OPMD patients to identify ones that show quantifiable change over time as the disease progresses.
- Trial name
- NH-OPMD (NCT03161847)
- Testing
- Non-interventional study
- Sponsor
- University of New Mexico
Duchenne Muscular Dystrophy
Evaluation of Home Based Assessments on Participants With DMD
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
- Trial name
- NCT05657938
- Testing
- Wearable Device, Duchenne Video Assessment (DVA)
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Solid Biosciences Inc.
Neuromuscular Diseases in Children, Respiratory Function Impaired
Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases
The clinical trial titled "Investigation of Respiratory Functions, Thoracoabdominal Movements, and Exercise Capacity in Neuromuscular Diseases" aims to evaluate the respiratory functions, thoracoabdominal movements, and exercise…
- Conditions
- Neuromuscular Diseases in Children, Respiratory Function Impaired, Respiratory Insufficiency, Duchenne Muscular Dystrophy
- Trial name
- NCT06756633
- Testing
- Respiratory Function Test, Structured light plethysmography, 6 Minute Walking Test
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Lokman Hekim University
Muscular Dystrophies, Intensive Care (ICU) Myopathy
Prognostic Factors , Morbidity and Mortality in Patients With Neuromuscular Disorders Admitted in ICU
Muscular dystrophies are neuromuscular disorders with disability. Restrictive pulmonary failure and cardiomyopathy affect prognosis.The investigators aim to establish predictive factors for mortality and morbidity in Intensive…
- Trial name
- NCT02685215
- Where
- France
- Volunteers
- 800 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Duchenne Muscular Dystrophy (DMD)
Study of Eteplirsen in DMD Patients
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51.
- Trial name
- PROMOVI (NCT02255552)
- Testing
- eteplirsen
- Where
- 37 sites in United States
- Volunteers
- 109 people
- Sponsor
- Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
- Trial name
- NCT06328725
- Testing
- EN001
- Where
- 2 sites in South Korea
- Volunteers
- 88 people
- Sponsor
- ENCell
Myotonic Dystrophy 1
DM-IMT - Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1
For some diseases, regular respiratory muscle training could delay the start of ventilation. For DM1, however, there are no clinically high-quality studies.
- Trial name
- DM-IMT (NCT04052958)
- Testing
- IMT - inspiratory muscle training
- Where
- Germany
- Volunteers
- 45 people
- Sponsor
- LMU Klinikum
Duchenne Muscular Dystrophy
Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
The purpose of the study is to show that the intake of L-arginine and metformin improves muscle function and delays disease progression in patients with Duchenne's muscular dystrophy.
- Trial name
- NCT02516085
- Testing
- Metformin, L-Arginine
- Volunteers
- 5 people
- Sponsor
- University Hospital, Basel, Switzerland
Spinal Muscular Atrophy, Duchenne Muscular Dystrophy
Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices
The addition of SMA and DMD muscle diseases to newborn screening and premarital carrier screening has been controversial. In this study, researchers aim to measure the awareness level of SMA and DMD muscle diseases of individuals…
- Conditions
- Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Healthy Individuals
- Trial name
- NCT05110885
- Testing
- Questionnaire
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Bahçeşehir University
Myotonic Dystrophy 1
Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe]
The rationale of the study is to collect structured data in the neuropsychological, clinical neuroradiologic and neurorehabilitation fields in children/young people affected by congenital and juvenile myotonic dystrophy.
- Trial name
- MDCJ-NeuBeRe (NCT06378216)
- Testing
- neurocognitive evaluations
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Eugenio Medea
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
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
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
FSH Muscular Dystrophy, Muscular Dystrophy
Amino Acids and Exercise in FSHD
This study evaluates whether amino acid supplementation, combined with a structured diet and exercise program, improves body composition (increased muscle mass and reduced fat mass) and physical performance in individuals with…
- Trial name
- NCT07543016
- Testing
- Essential Amino Acids Supplementation intervention, Exercise, Diet
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- University of Pavia
Myopathy, Myositis
Ultrasound in Muscle Biopsy
The objective of the proposed study is to evaluate the clinical utility of muscle ultrasonography for improving the diagnostic yield and safety of core muscle biopsy.
- Conditions
- Myopathy, Myositis, Muscular Dystrophy
- Trial name
- NCT01790178
- Testing
- Ultrasound
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Duke University
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
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Duchenne Muscular Dystrophy (DMD)
Peabody Developmental Motor Scaling In Children With Period Duchenne Muscular Dystrophy
To perform the reliability and validity study of the Peabody Developmental Motor Scale (Peabody Developmental Motor Scales-2) in children with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT05990608
- Testing
- Peabody Developmental Motor Scales-2, PDMS
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Ondokuz Mayıs University
Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT)
Personalized Training for People With Rare Neuromuscular Disorders
The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders…
- Conditions
- Neuromuscular Diseases (NMD), Charcot Marie Tooth Disease (CMT), Facioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy Type 1 (DM1)
- Trial name
- PETRA-NMD (NCT06708468)
- Testing
- Exercise, Usual Care
- Where
- 5 sites in Norway
- Volunteers
- 120 people
- Sponsor
- Oslo University Hospital
Muscular Dystrophy, Duchenne
An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
This study will help to determine the safety and efficacy of the nutritional supplement Coenzyme Q10 when added to steroids as a treatment for Duchenne muscular dystrophy (DMD).
- Trial name
- NCT00033189
- Testing
- Coenzyme Q10
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Cooperative International Neuromuscular Research Group
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