Clinical trials: Congenital Myopathy
Neck; Anomaly, Muscle Weakness
Attentional Focus Influence During Cranio-Cervical Flexion Test
The purpose of this study is to termine if a different attentional focus would alter the motor control in cervical muscles during the cranio-cervical test.
- Conditions
- Neck; Anomaly, Muscle Weakness, Muscle Weakness Condition
- Trial name
- NCT03567122
- Testing
- Internal Focus of Attention, External Focus of Attention, Control
- Where
- Brazil
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Federal University of Health Science of Porto Alegre
Spina Bifida, Neuromuscular Diseases
Metformin in Children With Motor Deficit
Obesity with insulin resistance in the paediatric population provides an increasing challenge. Children with neurological or neuromuscular diseases are even more prone to obesity: their locomotor impairment leads to an…
- Trial name
- NCT00720161
- Testing
- Metformin
- Where
- Belgium
- Volunteers
- 42 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Dysphagia
Dysphagia in Oculopharyngeal Muscular Dystrophy (OPMD)- Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up
The investigators aimed to review the natural history of dysphagia and dysphonia in OPMD in order to identify the best candidates and the proper timing to perform dysphagia alleviating procedures in both heterozygote and…
- Trial name
- OPMD (NCT01167439)
- Testing
- Upper esophageal sphincter myotomy
- Where
- Israel
- Volunteers
- 40 people
- Sponsor
- Hillel Yaffe Medical Center
Duchenne Muscular Dystrophy
A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
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…
- Trial name
- NCT02340923
- Where
- 5 sites in United States
- Volunteers
- 327 people, healthy volunteers welcome
- Sponsor
- Skulpt, Inc.
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
Brief Summary: 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…
- Trial name
- NCT03439670
- Testing
- Vamorolone, Prednisone
- Where
- 33 sites in United States, Australia and 9 more
- Volunteers
- 121 people
- Sponsor
- ReveraGen BioPharma, Inc.
Pompe Disease (Late-onset), Inclusion Body Myositis
Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease
The primary aim of this nationwide, explorative, cross-sectional study in Germany is to characterize the prevalence, severity and quality of musculoskeletal pain in adult patients with late-onset Pompe disease (LOPD).
- Conditions
- Pompe Disease (Late-onset), Inclusion Body Myositis, Spinal Muscular Atrophy Type 3, FSHD
- Trial name
- NCT05272969
- Testing
- Beck depression inventory fast screen (Questionnaire), Brief Pain Inventory (BPI) (Questionnaire), German Pain Inventory (Questionnaire)
- Where
- Germany
- Volunteers
- 95 people
- Sponsor
- LMU Klinikum
MELAS Syndrome
Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
The purpose of this study is to compare the efficacy of two (2) different doses of idebenone with that of a placebo over a one month period on cerebral lactate concentration as measured by magnetic resonance spectroscopy.
- Trial name
- MELAS (NCT00887562)
- Testing
- Idebenone
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Michio Hirano
Facioscapulohumeral Muscular Dystrophy
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in patients with Facioscapulohumeral muscular dystrophy…
- Trial name
- NCT02927080
- Testing
- ACE-083
- Where
- 24 sites in United States, Canada and 1 more
- Volunteers
- 95 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
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
Muscular Dystrophy, Facioscapulohumeral
Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation
Facioscapulohumeral dystrophy (FSHD) is one of the most common hereditary neuromuscular disorders (NMD), with an estimated prevalence of 2000 patients in the Netherlands.
- Trial name
- MUSCLE+ (NCT06227182)
- Testing
- Muscle ultrasound with surface electromyography and dynamometry, MRI scan
- Where
- Netherlands
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Radboud University Medical Center
Duchenne Muscular Dystrophy, Cardiomyopathies
The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
Purpose This cardiac dysfunction in patients with Duchenne muscular dystrophy is associated with minor cardiac damage as indicated by elevation of plasma cardiac troponin I (cTnI).
- Trial name
- NCT00606775
- Testing
- Carvedilol
- Where
- Japan
- Volunteers
- 60 people
- Sponsor
- Suzuka Hospital
Duchenne Muscular Dystrophy, Cardiomyopathy
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
The objective is to determine whether nebivolol, a beta-blockade drug, can prevent the development of heart disease in patients with Duchenne muscular dystrophy aged 10 to 15 year-old.
- Conditions
- Duchenne Muscular Dystrophy, Cardiomyopathy, Heart Failure
- Trial name
- NEBIDYS (NCT01648634)
- Testing
- Nebivolol
- Where
- France
- Volunteers
- 51 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Juvenile Dermatomyositis, Rheumatic Diseases
Comparison Of The Effects Of Two Different Exercise Training Based On Internal And External Focus In Children and Adolescents With Rheumatic Diseases
The study aims to compare the effects of two different exercise programs, based on distinct focus strategies, on physical fitness and fatigue in children and adolescents with rheumatic diseases.
- Conditions
- Juvenile Dermatomyositis, Rheumatic Diseases, Juvenile Idiopathic Arthritis (JIA), Familial Mediterranean Fever (FMF)
- Trial name
- NCT07138157
- Testing
- External Focus Exercises, Internal Focus Exercises
- Where
- Turkey (Türkiye)
- Volunteers
- 28 people
- Sponsor
- Istanbul University - Cerrahpasa
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
Facioscapulohumeral Muscular Dystrophy, FSHD
Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD
A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD
- Trial name
- NCT07435129
- Testing
- Apitegromab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Scholar Rock, Inc.
Duchenne Muscular Dystrophy
The U.K. NorthStar Clinical Network
The goal of this natural history study is to capture the natural history of Duchenne Muscular Dystrophy (DMD) in children and adults in the United Kingdom. Children and adults with DMD will be invited to join.
- Trial name
- NS (NCT06711692)
- Where
- 26 sites in United Kingdom
- Volunteers
- 300 people
- Sponsor
- University College, London
Duchenne Muscular Dystrophy
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531…
- Trial name
- NCT07209332
- Testing
- WVE-N531
- Where
- 3 sites in Jordan, United Kingdom
- Volunteers
- 175 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy (DMD)
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
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
- Trial name
- SIDEROS (NCT02814019)
- Testing
- Idebenone 150 mg film-coated tablets
- Where
- 63 sites in United States, Austria and 13 more
- Volunteers
- 255 people
- Sponsor
- Santhera Pharmaceuticals
Muscular Dystrophy, Facioscapulohumeral
Clenbuterol to Target DUX4 in FSHD
The purpose of this study is to determine if Clenbuterol is a therapeutic option for FSHD by determining the safety and tolerability of the medication at three different dose levels.
- Trial name
- Target FSHD (NCT06721299)
- Testing
- Clenbuterol
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Jeffrey Statland
Muscular Dystrophies
Diaphragm Ultrasound in Neuromuscular Disorders
Muscular dystrophies are neurologic disease with higher morbidity and mortality. Cardiac function and respiratory muscles can be affected, in addition with skeletal muscle deficiency .
- Trial name
- NCT04045158
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
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