Clinical trials: Metabolic Myopathies
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
Cancer, Diabetes
Identifying Therapeutic Factors in an 8-Week (30 Hour) Mindfulness-Based Stress Reduction (MBSR) Program
Since it's introduction in 1979 at the University of Massachusetts Medical Center by Dr. Jon Kabat-Zinn, Mindfulness-Based Stress Reduction (MBSR) has become a respected adjunct to conventional treatment for patients suffering…
- Conditions
- Cancer, Diabetes, Hypertension
- Trial name
- mbsr (NCT01599143)
- Testing
- Teaching Mindfulness-based Stress Reduction strategies
- Volunteers
- 135 people
- Sponsor
- North York General Hospital
Stroke, Ischemic, Stroke Hemorrhagic
Exploring Accessible Beauty for Individuals With Upper Extremity Deficits
The purpose of this study is to investigate the accessibility of beauty products for individuals with upper extremity disabilities.
- Conditions
- Stroke, Ischemic, Stroke Hemorrhagic, Stroke (CVA) or TIA, Stroke, Acute
- Trial name
- NCT05945784
- Testing
- Use of Rare Beauty makeup products
- Where
- United States
- Volunteers
- 57 people, healthy volunteers welcome
- Sponsor
- Casa Colina Hospital and Centers for Healthcare
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
Fibrodysplasia Ossificans Progressiva (FOP)
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Primary Objective(s): To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) To describe the prevalence of clinical characteristics of…
- Trial name
- NCT06064656
- Testing
- Non-Interventional
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Regeneron Pharmaceuticals
Paralysis, Hyperkalemic Periodic, Hypokalemic Periodic Paralysis
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders
OBJECTIVES: I. Assess the efficacy of dichlorphenamide in the treatment of episodic weakness attacks in patients with hyperkalemic periodic paralysis, paramyotonia congenita with periodic paralysis, and hypokalemic periodic…
- Conditions
- Paralysis, Hyperkalemic Periodic, Hypokalemic Periodic Paralysis, Paramyotonia Congenita
- Trial name
- NCT00004802
- Testing
- dichlorphenamide
- Volunteers
- 64 people
- Sponsor
- National Center for Research Resources (NCRR)
Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy
UTSW HP [13-C] Pyruvate Injection in HCM
The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal…
- Conditions
- Cardiomyopathy, Hypertrophic, Dilated Cardiomyopathy, Duchenne Muscular Dystrophy, Cardiac Sarcoidosis
- Trial name
- HPHCM (NCT03057002)
- Testing
- Hyperpolarized 13C-Pyruvate
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Muscle Weakness, Muscle Loss
A Study on the Effects on Glucose Regulation, Inflammation and Serum Lipids After Fish Protein Supplementation in Elderly (GRIPE)
The elderly population have and increased risk of loss of both muscle mass and function and is therefore recommended a higher protein intake than the healthy adult population.
- Conditions
- Muscle Weakness, Muscle Loss, Blood Sugar; High, Inflammation
- Trial name
- GRIPE (NCT03529344)
- Testing
- Blue whiting protein hydrolysate
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- University of Bergen
Idiopathic Inflammatory Myopathies, Osteoporosis, Osteopenia
Treating Idiopathic Inflammatory Myopathies Related Reduced Bone Mineral Density With Denosumab or Zoledronic Acid
Idiopathic inflammatory myopathies (IIM) patients are at high risk of development of reduced bone mineral density due to impairment of functional status due to the disease and a relatively high dose of glucocorticoid use for the…
- Trial name
- NCT04034199
- Testing
- Denosumab, Zoledronic Acid
- Where
- Hong Kong
- Volunteers
- 40 people
- Sponsor
- Kwong Wah Hospital
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are…
- Conditions
- Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
- Trial name
- Nemucough (NCT00839033)
- Testing
- mechanical insufflation - exsufflation, Standard respiratory physiotherapy
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sarcopenia, Muscle Weakness
HORMA: Hormonal Regulators of Muscle and Metabolism in Aging
The purpose of this study is to determine the relationship of deficiencies in testosterone and growth hormone to loss of muscle mass (sarcopenia) and functional impairment during aging and whether there is an interaction of these…
- Conditions
- Sarcopenia, Muscle Weakness, Frailty
- Trial name
- NCT00183040
- Testing
- Topical testosterone, Recombinant human growth hormone
- Where
- 2 sites in United States
- Volunteers
- 108 people
- Sponsor
- National Institute on Aging (NIA)
Respiratory Failure
Respiratory Care in Intensive Care Unit for Neuromuscular Patients
Retrospective multicentric observational study exploring the population of adult neuromuscular patients presenting a respiratory distress requiring their admission in an Intensive Care Unit (ICU) for ventilatory support.
- Trial name
- NCT02936908
- Testing
- Ventilatory support
- Where
- 4 sites in France
- Volunteers
- 300 people
- Sponsor
- Hospices Civils de Lyon
Mitochondrial Myopathies, Mitochondrial Diseases
Validation of Oxygen Nanosensor in Mitochondrial Myopathy
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM).
- Trial name
- NCT04086329
- Testing
- Nanosensor
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
Nuclear Magnetic Spectroscopy Imaging to Evaluate Primary Lateral Sclerosis, Hereditary Spastic Paraplegia and Amyotrophic Lateral Sclerosis
This study will use a magnetic resonance imaging technique called nuclear magnetic spectroscopy (H-MRS) to define the pathology and progression of primary lateral sclerosis, hereditary spastic paraplegia and amyotrophic lateral…
- Conditions
- Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Amyotrophic Lateral Sclerosis
- Trial name
- NCT00023075
- Where
- United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cystinosis, Myopathy
Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
Background: -(Degree)ystinosis is an inherited disease. If not treated correctly, it can cause muscle wasting and weakness and kidney damage. Researchers want to learn if growth hormone (GH) can help people with cystinosis.
- Trial name
- NCT02124070
- Testing
- rh Growth Hormone
- Sponsor
- National Human Genome Research Institute (NHGRI)
Tetraplegia, Functional Tetraplegia
Study of Home Use of the Jaco Robotic Manipulation Device by Individuals With Tetraplegia
For all individuals deprived of active and functional motor control of the upper limbs to perform grasping tasks, the restoration of a grasping function becomes a priority.
- Conditions
- Tetraplegia, Functional Tetraplegia, Spinal Cord Injury, Muscular Dystrophy
- Trial name
- TETRAJACO (NCT07680361)
- Testing
- Use of the JACO robotic arm for a period of 2 months
- Where
- France
- Volunteers
- 18 people
- Sponsor
- Association APPROCHE
Healthy Volunteer, Muscular Dystrophies
Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
This is a Phase 1 2-part, single-center, open-label study in healthy male volunteers. Part A will assess the absorption, metabolism, excretion, and pharmacokinetics of one oral dose of radiolabeled EDG-5506.
- Conditions
- Healthy Volunteer, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT05730842
- Testing
- EDG-5506 Tablet, Radiolabeled EDG-5506 Suspension, Radiolabeled EDG-5506 Intravenous
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- Edgewise Therapeutics, Inc.
Mitochondrial Myopathies, Mitochondrial Diseases
Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin Resistance
The overarching aim of this intervention study is to interrogate the interconnection between the muscle mitochondrial adaptations and the changes in muscle insulin sensitivity elicited by exercise training in individuals…
- Conditions
- Mitochondrial Myopathies, Mitochondrial Diseases, Mitochondrial Disorders
- Trial name
- EX-MITO-DYS-IR (NCT06080594)
- Testing
- High-intensity exercise training
- Where
- Denmark
- Volunteers
- 11 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
MItochondrial Myopathies, Mitochondrial Disease
Validation of Nanosensor Oxygen Measurement
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM).
- Trial name
- NCT06819683
- Testing
- Nanosensor
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Duchenne Muscular Dystrophy
Wearable Technology to Evaluate Hyperglycemia and HRV in DMD
Duchenne muscular dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death.
- Trial name
- NCT06124196
- Testing
- wearable technology
- Where
- United States
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center