Clinical Trials on Miyoshi Myopathie

Total 294 results

First Affiliated Hospital Xi'an Jiaotong University

Recruiting

Clinical Study of MMF in Treatment of IIM-ILD and Its Effect on Peripheral Blood Treg Cells

Idiopathic Inflammatory Myopathy | Interstitial Lung Disease

China
Stealth BioTherapeutics Inc.

Completed

Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy (MMPOWER)

Mitochondrial Myopathy

United States
Cabaletta Bio

Recruiting

RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy

Idiopathic Inflammatory Myopathy | Dermatomyositis | Immune-Mediated Necrotizing Myopathy | Anti-Synthetase Syndrome

United States
Children's Hospital of Philadelphia
National Institute of Neurological Disorders and Stroke (NINDS)

Recruiting

Validation of Oxygen Nanosensor in Mitochondrial Myopathy

Mitochondrial Diseases | Mitochondrial Myopathies

United States
Rigshospitalet, Denmark

Completed

Diagnostic Screening Tests and Potential Biomarkers in Mitochondrial Myopathies

Mitochondrial Myopathies | Healthy Controls

Denmark
Radboud University Medical Center

Completed

Inspiratory Muscle Training in Nemaline Myopathy (NEMTRAIN)

Inspiratory Muscle Training | Nemaline Myopathy

Netherlands
Cooperative International Neuromuscular Research...
Carolinas Medical Center lead study site

Completed

Evaluation of Limb-Girdle Muscular Dystrophy

Becker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)

United States
Ultragenyx Pharmaceutical Inc

Completed

Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM) (GNEM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Ultragenyx Pharmaceutical Inc

Terminated

A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment

Hereditary Inclusion Body Myopathy | GNE Myopathy | Inclusion Body Myopathy 2 | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

Canada, United States, Bulgaria
University of Sao Paulo

Recruiting

Optimizing Treatment on Idiopathic Inflammatory Myopathies

Idiopathic Inflammatory Myopathies

Brazil
Ultragenyx Pharmaceutical Inc

Terminated

Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Distal Myopathy With Rimmed Vacuoles | Distal Myopathy, Nonaka Type | Quadriceps Sparing Myopathy

United States, Canada, United Kingdom, Israel, Italy, Bulgaria, France
Bristol-Myers Squibb

Completed

Trial to Evaluate the Efficacy and Safety of Abatacept in Combination With Standard Therapy Compared to Standard Therapy Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy

Dermatomyositis | Polymyositis | Autoimmune Necrotizing Myopathy | Overlap Myositis | Juvenile Myositis Above the Age of 18

France, United States, Australia, Brazil, Czechia, Germany, Italy, Japan, Korea, Republic of, Mexico, Sweden
Rigshospitalet, Denmark

Completed

Resveratrol Supplementation in Patients With Mitochondrial Myopathies and Skeletal Muscle Fatty Acid Oxidation Disorders

Mitochondrial Myopathies | Fatty Acid Oxidation Defects

Denmark
Children's Hospital of Philadelphia
University of Pennsylvania; United Mitochondrial Disease Foundation (UMDF)

Recruiting

Mitochondrial Myopathy Rating Scale

Primary Mitochondrial Disease

United States
Nobelpharma

Completed

Efficacy Confirmation Study of NPC-09

GNE Myopathy | Nonaka Disease | Distal Myopathy With Rimmed Vacuoles (DMRV) | Hereditary Inclusion Body Myopathy (hIBM)

Japan
Ultragenyx Pharmaceutical Inc

Completed

An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy

GNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)

United States, Israel
Ultragenyx Pharmaceutical Inc
Newcastle University

Completed

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy | GNE Myopathy | Nonaka Disease | Quadriceps Sparing Myopathy (QSM) | Distal Myopathy With Rimmed Vacuoles (DMRV)

United States, Bulgaria, Canada, France, United Kingdom
Newcastle University

Unknown

International GNE Myopathy Patient Registry

Hereditary Inclusion Body Myopathy | GNE Myopathy

United Kingdom
Tohoku University

Completed

Pharmacokinetic Study on N-acetylneuraminic Acid

Hereditary Inclusion Body Myopathy | Nonaka Myopathy

Japan
Immunoforge Co. Ltd.

Recruiting

Study Evaluating Efficacy and Safety of Froniglutide (PF1801) in Patients With Idiopathic Inflammatory Myopathy (FROG)

Dermatomyositis | Polymyositis | Idiopathic Inflammatory Myopathies

Korea, Republic of
National Human Genome Research Institute (NHGRI)
National Center for Advancing Translational Sciences (NCATS); Therapeutics...

Completed

Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

GNE Myopathy | Hereditary Inclusion Body Myopathy (HIBM)

United States
Ultragenyx Pharmaceutical Inc

Completed

A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (GNE Myopathy)

Hereditary Inclusion Body Myopathy | GNE Myopathy

United States, Israel
Ultragenyx Pharmaceutical Inc

Completed

Safety and Pharmacokinetics of Sialic Acid Tables in Patients With Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy (HIBM)

United States
National Center for Research Resources (NCRR)
University of Rochester

Completed

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Mitochondrial Myopathy
Maastricht University

Recruiting

Effect and Safety MABs Administration m.3243A>G Mutation Carriers

Mitochondrial Myopathies

Netherlands
Maastricht University

Recruiting

mtDNA Mutation Load Analysis in Mesoangioblasts (MABS05)

Mitochondrial Myopathies

Netherlands
Kwong Wah Hospital
Tung Wah Group of Hospitals

Unknown

Treating Idiopathic Inflammatory Myopathies Related Reduced Bone Mineral Density With Denosumab or Zoledronic Acid

Idiopathic Inflammatory Myopathies | Osteoporosis, Osteopenia

Hong Kong
Northern Care Alliance NHS Foundation Trust

Completed

A Case-control Study to Assess Risk of Coronary Heart Disease in Idiopathic Inflammatory Myopathy

Idiopathic Inflammatory Myopathy

United Kingdom
Academisch Medisch Centrum - Universiteit van Amsterdam...
Princess Beatrix Muscle Foundation

Recruiting

Add-on Intravenous Immunoglobulins in Early Myositis (TIMEISMUSCLE)

Myositis | Dermatomyositis | Polymyositis | Antisynthetase Syndrome | Immune-Mediated Necrotizing Myopathy | Inflammatory Myopathy, Idiopathic

Netherlands
National Human Genome Research Institute (NHGRI)
National Center for Advancing Translational Sciences (NCATS)

Completed

An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy

GNE Myopathy

United States
Maastricht University

Completed

MABs Therapy m.3243A>G Mutation Carriers (MABS01)

Mitochondrial Myopathies

Netherlands
Juno Therapeutics, Inc., a Bristol-Myers Squibb...
Bristol-Myers Squibb Services Unlimited Company

Recruiting

A Study of CC-97540, CD-19-Targeted Nex-T CAR T Cells, in Participants With Severe, Refractory Autoimmune Diseases

Systemic Lupus Erythematosus | Systemic Sclerosis | Idiopathic Inflammatory Myopathy

United States, Belgium, France, Spain, Germany, Italy
University Hospital, Basel, Switzerland
University of Minnesota

Completed

Muscle Force Assessment in the Intensive Care Unit and in Primary Myopathies

Sepsis | Primary Myopathies

Switzerland
Newcastle-upon-Tyne Hospitals NHS Trust
Newcastle University

Completed

A Study of Bezafibrate in Mitochondrial Myopathy

Mitochondrial Diseases

United Kingdom
Federal University of Espirito Santo

Not yet recruiting

Muscle Function and Effects of Repetitive Task Training in Patients With Inflammatory Myopaties

Idiopathic Inflammatory Myopathy | Dermatomyositis | Polymyositis

Brazil
National Institute of Environmental Health Sciences...

Completed

Studies of the Natural History and Pathogenesis of Autoimmune/Connective Tissue Diseases

Dermatomyositis | Polymyositis | Autoimmune/Connective Tissue Diseases | Idiopathic Inflammatory Myopathies

United States
Children's Hospital of Philadelphia
United States Department of Defense

Recruiting

Applying pGz in Mitochondrial Disease

Mitochondrial Diseases | Mitochondrial Myopathies

United States
Leadiant Biosciences, Inc.
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and other collaborators

Active, not recruiting

Multi-Center Study of ManNAc for GNE Myopathy (MAGiNE)

GNE Myopathy

United States
National Human Genome Research Institute (NHGRI)

Completed

Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy

Hereditary Inclusion Body Myopathy

United States
National Institute of Environmental Health Sciences...

Completed

Sodium Thiosulfate for Treatment of Calcinosis Associated With Juvenile and Adult Dermatomyositis

Dermatomyositis | Idiopathic Inflammatory Myopathies

United States
Massachusetts General Hospital

Completed

Defining 31Phosphorous Magnetic Resonance Spectroscopy Characteristics in Patients With Mitochondrial Myopathy

Mitochondrial Disease

United States
Stealth BioTherapeutics Inc.

Completed

Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study (MMPOWER-2)

Primary Mitochondrial Disease

United States
National Human Genome Research Institute (NHGRI)
Therapeutics for Rare and Neglected Diseases (TRND)

Recruiting

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

GNE Myopathy | GNE Related Diseases

United States
Radboud University Medical Center

Unknown

Muscle Relaxation in Myopathies With Positive Muscle Phenomena

Myotonic Dystrophy Type 2 | McArdle Disease | Nemaline Myopathy Type 6
Stealth BioTherapeutics Inc.

Terminated

Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

Primary Mitochondrial Disease

United States
Rigshospitalet, Denmark
University of Copenhagen

Recruiting

Human Mitochondrial Stress-driven Obesity Resistance (MITO-OB-RES)

Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Disorder

Denmark
Rigshospitalet, Denmark
University of Copenhagen

Recruiting

Mitochondrial Dysfunctions Driving Insulin Resistance (MITO-DYS-IR)

Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Disorders

Denmark
Rigshospitalet, Denmark
University of Copenhagen

Recruiting

Exercise-mediated Rescue of Mitochondrial Dysfunctions Driving Insulin Resistance (EX-MITO-DYS-IR)

Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Disorders

Denmark
University Hospital, Brest

Recruiting

Metabolic Exercise Testing (RANNOU)

Myalgia | Exercise Test | Metabolic Myopathies

France
Stealth BioTherapeutics Inc.

Active, not recruiting

Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) (NuPower)

Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Complex I Deficiency | Mitochondrial Pathology | Mitochondrial DNA Depletion | Mitochondrial DNA Mutation | Mitochondrial DNA Deletion | Mitochondrial Metabolism Defect

Spain, United States, Italy, Netherlands, Australia, Germany, Hungary, New Zealand, Norway, United Kingdom

Clinical Trials on Miyoshi Myopathie

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