Clinical trials: Metabolic Myopathy
Cardiac Diseases, Muscular Weakness
Biomarker for Pompe Disease (BioPompe)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Pompe disease from blood (plasma)
- Conditions
- Cardiac Diseases, Muscular Weakness, Hepato-splenomegaly, Lung Disease
- Trial name
- BioPompe (NCT01457443)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
MELAS Syndrome
Glutamine Supplement in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes) Syndrome
The purpose of this study is to assesses the efficacy of oral supplementation with glutamine over three months on several amino acids and lactate concentration measured in cerebrospinal fluid and cerebral lactate measured by…
- Trial name
- NCT04948138
- Testing
- Glutamine oral supplementation
- Where
- Spain
- Volunteers
- 9 people
- Sponsor
- Jesús González de la Aleja Tejera
Duchenne Muscular Dystrophy (DMD)
Glucagon-Like Peptide-1 Receptor Agonists to Attenuate Metabolic Risk in Individuals With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a rare, genetic disease that leads to muscle weakness, breathing difficulties, heart disease, and early death.
- Trial name
- NCT07642635
- Testing
- Semaglutide (Rybelsus®)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Vanderbilt University Medical Center
Systemic Lupus Erthematosus, Sjogren's Syndrome
Mass Spectrometry-based Immune Profiling in Autoimmune Diseases
Based on mass spectrometry flow method, this study analyzed the typing of new T, B, NK and DC cell subsets in peripheral blood of common autoimmune diseases and their correlation with disease activity, aiming at establishing an…
- Conditions
- Systemic Lupus Erthematosus, Sjogren's Syndrome, Inflammatory Myopathies, Systemic Sclerosis (SSc)
- Trial name
- NCT07188285
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Peking University People's Hospital
Urea Cycle Disorder, Organic Acidemia
Systemic Biomarkers of Brain Injury From Hyperammonemia
Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxic brain edema and vascular compromise leading to intellectual and…
- Conditions
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I
- Trial name
- NCT04602325
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Children's National Research Institute
Sleep Disturbance, Fatigue Syndrome, Chronic
Bao Yuan Tang Extract and Galactooligosaccharides for Sleep Regulation
Certain classical formulas in traditional Chinese medicine and dietary prebiotics have demonstrated beneficial effects on various aspects of suboptimal health status in previous studies.
- Conditions
- Sleep Disturbance, Fatigue Syndrome, Chronic, Endocrine Disorder, Glucose Metabolism Disorder
- Trial name
- NCT07774897
- Testing
- Combination product (Bao Yuan Tang full-component extract 2.0g and galactooligosaccharides 2.0g), Galactooligosaccharides 2.0g
- Where
- 6 sites in China
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Hepatopancreatobiliary Surgery Institute of Gansu Province
Amyotrophic Lateral Sclerosis
Clinical Trial of Vitamin E to Treat Muscular Cramps in Patients With ALS
Muscular cramps are a common and uncomfortable symptom of amyotrophic lateral sclerosis (ALS). This clinical trial will compare the response of high dose vitamin E supplementation to placebo for treatment of muscular cramps in…
- Trial name
- NCT00372879
- Testing
- Vitamin E
- Where
- Canada
- Volunteers
- 32 people
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Autosomal Dominant Hypocalcemia (ADH)
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
- Trial name
- CALIBRATE (NCT05680818)
- Testing
- Encaleret, Standard of Care
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 67 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Neuromuscular Diseases
Muscle Oxygenation in Effort in Neuromuscular Diseases
Previous studies showed modifications of muscle oxygenation parameters in muscular dystrophies du to an impairment or an absence of dystrophin.
- Trial name
- OXYNEMU (NCT02789059)
- Testing
- muscle oxygenation
- Where
- 3 sites in France
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University Hospital, Lille
Fibromyalgia (FM)
Effects of Curcumin-Piperine Supplementation in Fibromyalgia
The goal of this clinical trail is to evaluate the effectiveness and safety of curcumin-piperine supplementation in patients with fibromyalgia over 3 months compared to placebo.
- Trial name
- NCT07753018
- Testing
- Curcumin - Piperine
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS)
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility…
- Conditions
- Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke
- Trial name
- INTENT (NCT07521930)
- Testing
- INTENT Neural Interface System
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Johns Hopkins University
Diabetes Mellitus, Type 2, Musculoskeletal Pain
"Shoulder Musculoskeletal Disorders in Type 2 Diabetes Mellitus: Contributing Factors and Rehabilitation Protocol"
Goals: to compare the effects of two distinct rehabilitation protocols (conventional shoulder musculoskeletal rehabilitation combined with aerobic exercises versus solely conventional shoulder musculoskeletal rehabilitation) on…
- Conditions
- Diabetes Mellitus, Type 2, Musculoskeletal Pain, Shoulder Pain
- Trial name
- NCT04817514
- Testing
- Shoulder rehabilitation exercises and aerobic exercises
- Volunteers
- 36 people
- Sponsor
- Universidad Católica del Maule
Osteoporosis
Calcium Metabolism in Mexican American Adolescents
The purpose of this study is to identify calcium metabolism characteristics in Mexican American adolescents. The investigators predict that calcium requirements to maximize bone health in this population will be lower than for…
- Trial name
- NCT01277185
- Testing
- Low Calcium Diet, High Calcium Diet
- Where
- United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Purdue University
Dermatomyositis, Idiopathic Inflammatory Myopathies
Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis
Background: Dermatomyositis (DM) and juvenile dermatomyositis (JDM) cause inflammation in the muscles. People with DM and JDM can develop calcium deposits in places they should not, known as calcinosis.
- Trial name
- NCT03267277
- Testing
- Sodium Thiosulfate
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Fibromyalgia, D Vitamin Deficiency
Valuation of Efficacy and Safety of Vitamin D3 Use in 80 Women Diagnosed With Fibromyalgia. Using FIQ Score and VAS.
Fibromyalgia is a disease that significantly decreases the quality of life of people who suffer from it, has great repercussions at the socio-economic level due to the incapacity it produces.
- Trial name
- NCT03369379
- Testing
- D3 Vitamin
- Where
- Mexico
- Volunteers
- 80 people
- Sponsor
- Universidad Autonoma de Nuevo Leon
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics
Cerebral Palsy, Spastic
Muscle-tendon Pathology and Metabolic Dysregulation in CP
The main aim of this project is to gain knowledge about the muscle-tendon pathology of moderately to severely affected young people with cerebral palsy and their risk for cardiometabolic diseases and chronic inflammation.
- Trial name
- NCT06330922
- Testing
- Cardiorespiratory fitness training
- Where
- Austria
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Graz
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Gut Microbiome, Gastrointestinal Microbiome
A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease
This study seeks to correlate microbiome sequencing data with information provided by patients and their medical records.
- Conditions
- Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder
- Trial name
- NCT04031469
- Testing
- There is no intervention in this study
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- ProgenaBiome
Mitochondrial Diseases
A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease
A long-term extension study to assess the safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) in children with inherited mitochondrial diseases who previously enrolled into study…
- Trial name
- NCT02473445
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 22 people
- Sponsor
- Amgen