Clinical trials: Myopathies
Selenoprotein N-related Myopathy
Pharmacological Treatment of a Rare Genetic Disease: N-acetylcysteine in Myopathy Associated Selenoprotein N-related Myopathy (SEPN1-RM)
The objective of this study is to determine whether the administration of N-acetylcysteine (NAC) improves oxidative stress. To determine this, the study will assess the impact of oral treatment on the balance between reduced and…
- Trial name
- SelNac (NCT02505087)
- Where
- France
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
SARS-CoV Infection, Systemic Disease
Serologic Profile of SARS CoV2 in COVID-19 Patients With Systemic Diseases
An observational study aiming to assess the serological profile of SARS-Cov2 patients with systemic diseases such as systemic lupus erythematosus, Sjogren syndrome, sarcoidosis, inflammatory myopathies, Behçet's disease…
- Conditions
- SARS-CoV Infection, Systemic Disease, Systemic Lupus Erythematosus, Sjogren's Syndrome
- Trial name
- NCT04530461
- Testing
- Serological test
- Where
- Tunisia
- Volunteers
- 200 people
- Sponsor
- faida agili
Electromyography, EMG: Myopathy
Electromyographic Study for the Help and Guidance of BoNTA Administration in the Treatment of Chronic Pelvic Floor Pain
Chronic pelvic pain (CPP) is a common presenting complaint affecting approximately 15-40 % of women aged 18-50 in western countries and 5-43% of women in most developing countries.
- Conditions
- Electromyography, EMG: Myopathy, EMG Syndrome, Pelvic Pain
- Trial name
- SEMG (NCT03715777)
- Testing
- Clostridium botulinum type A (BoNTA)
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Jose Alberola-Rubio
Cardiac Disease, Critical Illness
In-Bed Cycling in ICU Patients Post Cardiac Surgery
The purpose of this study is to determine if in-bed cycling is safe and feasible in critically-ill patients after open heart surgery.
- Conditions
- Cardiac Disease, Critical Illness, Critical Illness Polyneuropathy, Critical Illness Myopathy
- Trial name
- CardiO Cycle (NCT02976415)
- Testing
- In-Bed Cycling
- Where
- Canada
- Volunteers
- 23 people
- Sponsor
- Hamilton Health Sciences Corporation
Primary Mitochondrial Disease
Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
This randomized, double-blind, placebo-controlled, crossover study screened 32 subjects with primary mitochondrial myopathy (PMM) to evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of subcutaneous…
- Trial name
- MMPOWER-2 (NCT02805790)
- Testing
- Elamipretide
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Stealth BioTherapeutics Inc.
Primary Mitochondrial Myopathy
A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy
The purpose of this study is to assess REN001 safety in subjects with primary mitochondrial myopathy
- Trial name
- NCT03862846
- Testing
- REN001
- Where
- 2 sites in United Kingdom
- Volunteers
- 23 people
- Sponsor
- Reneo Pharma Ltd
Critical Illness, Respiratory Failure
Treatment of Muscle Weakness in Critically Ill Patients
Patients who are admitted to the intensive care unit and require mechanical ventilation frequently develop profound respiratory and limb muscle weakness.
- Conditions
- Critical Illness, Respiratory Failure, Myopathy, Weakness
- Trial name
- NCT02247895
- Testing
- Electrical stimulation
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Gerald Supinski
Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency
High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
The study also determines if eating a diet higher in protein alters body composition, energy balance and metabolic control among patients with a long-chain fatty acid oxidation disorder.
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Carnitine Palmitoyltransferase 2 Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT01494051
- Testing
- Diet counseling
- Volunteers
- 13 people
- Sponsor
- Oregon Health and Science University
Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis
European Home Mechanical Ventilation Registry
The European Home Mechanical Ventilation Registry (EHMVR) will enable a thorough evaluation of HMV by documenting the characteristics of HMV patients and their treatment.
- Conditions
- Pulmonary Disease, Chronic Obstructive, Amyotrophic Lateral Sclerosis, Spinal Cord Injury, Muscular Dystrophies
- Trial name
- EHMVR (NCT02315339)
- Where
- Germany
- Volunteers
- 37 people
- Sponsor
- ResMed
Immune-Mediated Necrotizing Myopathy
Juvenile Autoimmune Necrotizing Myopathies
Autoimmune necrotizing myopathies (AINM) in adult patients are characterized by severity of muscle damage, presence of necrosis with little inflammation on muscle biopsy and anti-HMGCR or anti-SRP auto-antibodies.
- Trial name
- MYONECPED (NCT04295785)
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Central Hospital, Nancy, France
Statin Adverse Reaction, Skeletal Myopathy
NIRS to Diagnose SAMS
This proposal seeks to determine whether near infrared spectroscopy (NIRS) can differentiate between patients with confirmed SAMS and those with non-specific muscle complaints.
- Trial name
- NCT03653663
- Testing
- Simvastatin 20 mg
- Where
- 2 sites in United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hartford Hospital
Critical Illness, Intensive Care (ICU) Myopathy
Muscle Dysfunction in Critical Illness
Patients surviving critical illness suffered significant skeletal muscle dysfunction and weakness that impacts their functionality and independence.
- Conditions
- Critical Illness, Intensive Care (ICU) Myopathy, PICS, Muscle Weakness
- Trial name
- NCT03717831
- Testing
- Observational study of standard of care
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- University of Kentucky
Inflammatory Myopathy
Low-dose Interleukin-2 in Combination With Standard Therapy on Idiopathic Inflammatory Myopathy
This study aims to explore the clinical and immunological efficacy of low-dose Interleukin-2 (IL-2) and cyclosporin a (CSA) on idiopathic inflammatory myopathy (IIM)
- Trial name
- NCT04237987
- Testing
- Interleukin-2, ciclosporin and corticosteroid
- Volunteers
- 20 people
- Sponsor
- Peking University People's Hospital
Metabolic Myopathies, Exercise Test
Metabolic Exercise Testing
Exertional symptoms are the hallmarks of metabolic myopathies, supporting the concept of using functional tests when this diagnosis is suspected.
- Conditions
- Metabolic Myopathies, Exercise Test, Myalgia
- Trial name
- RANNOU (NCT02362685)
- Testing
- functional test, matching genotype with phenotype
- Where
- France
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Mitochondrial Myopathy
Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Phase 1/2, multi-center, randomized, double-blind, multiple ascending dose, placebo-controlled study that enrolled 36 subjects with mitochondrial myopathy associated with genetically confirmed mitochondrial disease to evaluate…
- Trial name
- MMPOWER (NCT02367014)
- Testing
- elamipretide (low dose), elamipretide (intermediate dose), elamipretide (high dose)
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- Stealth BioTherapeutics Inc.
Critical Illness Polyneuropathy, Critical Illness Myopathy
Validation of Simplified Electrophysiological Examination in the Diagnosis of Critical Illness Myopathy or Neuropathy
Evaluate the accuracy, in the diagnosis of critical illness myopathy and / or neuropathy, of the simplified peroneal nerve test performed by a neurophysiopathology technician or by a neurophysiopathology doctor (as the gold…
- Trial name
- CRIMINE-3 (NCT04193943)
- Where
- Italy
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- Università degli Studi di Brescia
Hereditary Inclusion Body Myopathy
Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
This study will evaluate patients with Hereditary Inclusion Body Myopathy (HIBM) and examine the effects of immune globulin (IG) treatment on muscle and muscle function.
- Trial name
- NCT00195637
- Testing
- Immune Globulin
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Mitochondrial Myopathies, Fatty Acid Oxidation Defects
Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders
The purpose of this study is to investigate the potential beneficial effects of a daily supplement of Resveratrol (1000mg/day) on physical ability and on muscle metabolism in patients with verified mitochondrial myopathy and…
- Trial name
- NCT03728777
- Testing
- Resveratrol
- Where
- Denmark
- Volunteers
- 20 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors
Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy
This study aims to characterize the pathophysiological mechanisms of 21 different metabolic myopathies. The study will focus on exercise capacity and the metabolic derangement during exercise.
- Conditions
- Metabolism, Inborn Errors, Lipid Metabolism, Inborn Errors, Carbohydrate Metabolism, Inborn Errors, Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT02635269
- Testing
- Sugar
- Where
- Denmark
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Intensive Care Unit Syndrome, Intensive Care Neurological Disorder
Vanderbilt ICU Recovery Program Pilot Trial
Every year, millions of Americans are admitted to the intensive care unit. Due to advances in critical care, mortality rates are decreasing, increasing the number of ICU survivors.
- Conditions
- Intensive Care Unit Syndrome, Intensive Care Neurological Disorder, Intensive Care (ICU) Myopathy, Intensive Care Psychiatric Disorder (Diagnosis)
- Trial name
- VIP (NCT03124342)
- Testing
- VANDERBILT ICU RECOVERY PROGRAM
- Where
- United States
- Volunteers
- 232 people
- Sponsor
- Vanderbilt University Medical Center