Clinical Trials on SEPN1-related Myopathy

Total 51432 results

Cure CMD
University of Chicago; Boston Children's Hospital; Valerion Therapeutics, LLC; Congenital...

Completed

Myotubular Myopathy Genetic Testing Study

Myotubular Myopathy

United States
Northwestern University

Terminated

Stem Cell Transplantation in Idiopathic Inflammatory Myopathy Diseases

MYOPATHY

United States
Hadassah Medical Organization

Unknown

Treatment of TNNT1-Myopathy With L-Tyrosine.

Nemaline Myopathy

Israel
Dynacure

Withdrawn

Early Phase Human Drug Trial to Investigate DYN101 in Participants 2 to 17 Years With Centronuclear Myopathies (DyNaMic)

Centronuclear Myopathy

Germany, France
Rigshospitalet, Denmark
University of Copenhagen

Completed

Biomechanical Properties in Intensive Care Unit (ICU) Survivors 12-month After Discharge

Intensive Care (ICU) Myopathy

Denmark
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
Mayo Clinic

Completed

Characterization of Skeletal Muscle Using Magnetic Resonance Elastography (MRE)

Stroke | Immobilization | Myofacial Pain | Hyperthyroid Myopathy

United States
University of Manchester
King's College Hospital NHS Trust; Northern Care Alliance NHS Foundation Trust and other collaborators

Active, not recruiting

MYOPROSP - a Prospective Cohort Study in Myositis (MYOPROSP)

Myositis | Idiopathic Inflammatory Myopathy

United Kingdom
Valerion Therapeutics, LLC
Institut de Myologie, France; Genethon

Completed

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM)

Myotubular Myopathy

France, Belgium, United States, Canada, Germany, Italy, Spain
Peking University People's Hospital

Unknown

Low-dose Interleukin-2 Treatment on Idiopathic Inflammatory Myopathy

Inflammatory Myopathy
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
Eduardo N. Chini

Recruiting

A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy

Mitochondrial Myopathy

United States
University of Helsinki
Helsinki University Central Hospital; University of Iowa; Institute for Molecular...

Completed

Niacin Supplementation in Healthy Controls and Mitochondrial Myopathy Patients (NiaMIT)

Mitochondrial Myopathies
Guy's and St Thomas' NHS Foundation Trust
University College, London; The Whittington Hospital NHS Trust

Completed

Intermittent Versus Continuous Feeding in ICU Patients

Intensive Care (ICU) Myopathy

United Kingdom
University of Sao Paulo

Recruiting

Acute Transcranial Direct Current Stimulation in Patients With Systemic Autoimmune Myopathies

Neurologic Manifestations | Myopathy | ElectroPhys: Myopathy

Brazil
Peking University People's Hospital

Unknown

Low-dose Interleukin-2 in Combination With Standard Therapy on Idiopathic Inflammatory Myopathy

Inflammatory Myopathy
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
Radboud University Medical Center

Completed

The Natural History of Patients With Mutations in SEPN1 (SELENON) or LAMA2

MDC1A | SELENON-related Myopathy

Netherlands
University of Oslo School of Pharmacy

Completed

Association Between Systemic Exposure of Atorvastatin and Metabolites and Atorvastatin-induced Myotoxicity

Myopathy
Reata, a wholly owned subsidiary of Biogen
AbbVie

Completed

RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

MItochondrial Myopathies

United States, Denmark
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
Centre Hospitalier Universitaire de Nice

Recruiting

Home Based Personalized Training and Video Consultation in Mitochondrial Myopathies: Study of Efficacy and Tolerance. (TELE-MIT)

Mitochondrial Myopathies

France
University of Helsinki
Helsinki University Central Hospital; Institute for Molecular Medicine

Completed

NiaMIT Continuation With Early-stage Mitochondrial Myopathy Patients

Mitochondrial Myopathies

Finland
National Institute of Arthritis and Musculoskeletal...

Unknown

The Effects of Exercise Versus Inactivity on People With Mitochondrial Muscle Disease

Mitochondrial Myopathy

United States
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
Guy's and St Thomas' NHS Foundation Trust

Completed

Rehabilitation Following Critical Illness

Critical Illness Myopathy

United Kingdom
Stealth BioTherapeutics Inc.

Completed

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

Mitochondrial Myopathy

United States
Cure CMD

Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)

Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions

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
argenx

Recruiting

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)

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

United States, Germany, Spain, Korea, Republic of, Austria, Turkey, Lithuania, Australia, Thailand, Serbia, Poland, Italy, France, Netherlands, Argentina, United Kingdom, Georgia, Belgium, Bulgaria, Canada, Cyprus, Denmark, Greece, Hu... and more
Cristina Domínguez González

Recruiting

Natural History in Primary Mitochondrial Myopathies (NHPMM)

Primary Mitochondrial Myopathies

Spain
argenx

Recruiting

A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy (ALKIVIA+)

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

Korea, Republic of, United States, Bulgaria, Belgium, Denmark, Georgia, Greece, Lithuania, Argentina
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
Myotonic Dystrophy Foundation

Recruiting

Myotonic Dystrophy Family Registry (MDFR)

Myotonic Dystrophy | Myotonic Dystrophy 1 | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Steinert Disease | Dystrophia Myotonica 1 | Myotonic Dystrophy 2 | Dystrophia Myotonica | Dystrophia Myotonica 2 | Myotonia Dystrophica | Myotonic Dystrophy, Congenital | Myotonic... and other conditions

United States
Technical University of Munich
Charite University, Berlin, Germany; Helmholtz Zentrum München

Recruiting

Metabolomics Study on Postoperative Intensive Care Acquired Muscle Weakness (MIRACLE I)

Critical Illness | Muscle Weakness | Critical Illness Myopathy | Critical Care | Critical Illness Polyneuropathy | Metabolomics | Intensive Care (ICU) Myopathy

Germany
National Heart Centre Singapore
National University Hospital, Singapore

Unknown

SAPhIRE Statin Adverse Drug Reaction (STATIN)

Myopathy

Singapore
Mads Peter Godtfeldt Stemmerik

Recruiting

Repeated Bout Effect i Neuromuscular Diseases

Myopathy

Denmark
Technical University of Munich
Helmholtz Zentrum München

Completed

Metabolomics Study on Intensive Care Acquired Muscle Weakness in Polytrauma (MIRACLE II)

Critical Illness | Muscle Weakness | Critical Illness Myopathy | Critical Care | Critical Illness Polyneuropathy | Metabolomics | Intensive Care (ICU) Myopathy

Germany
Università degli Studi di Brescia

Unknown

Validation of Simplified Electrophysiological Examination in the Diagnosis of Critical Illness Myopathy or Neuropathy (CRIMINE-3)

Critical Illness Myopathy | Critical Illness Polyneuropathy

Italy
The First Affiliated Hospital with Nanjing Medical...

Not yet recruiting

Adult Idiopathic Inflammatory Myopathy With Cardiac Injury

Cardiac Disease | Inflammatory Myopathy
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
Barts & The London NHS Trust
Queen Mary University of London

Completed

Muscle Recovery Following Aortic Surgery Induced ICUAW. (VARIANCE)

Surgery | Critical Illness Myopathy | Aortic Diseases | Intensive Care Unit Syndrome | Intensive Care (ICU) Myopathy | Intensive Care Neuropathy

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

Completed

Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Mitochondrial Myopathy
Eunice Kennedy Shriver National Institute of Child...

Completed

Investigations Into ISCU Myopathy or Iron Sulfur Scaffold U Protein Myopathy

Myopathy

United States
University Hospital, Montpellier

Completed

Effects of Statins on Pro- and Antioxidant Status : Link With Statin-Associated Myopathy.

Healthy

France
Institut National de la Santé Et de la Recherche...

Completed

IRM Cardiaque en Respiration Libre Pour Des Patients Atteints de Dystrophinopathie sévère

Duchenne or Severe Becker Myopathy

France
University of Aarhus

Recruiting

Muscular Deficits of Diabetic Myopathy

Obesity | Diabetes Mellitus | Weight Loss | Sarcopenia | Chronic Hyperglycaemia | Diabetic Myopathy

Denmark

Clinical Trials on SEPN1-related Myopathy

Sponsors and Collaborators

Medical Conditions

Drug Interventions

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CROs in South Korea

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Rare Diseases

Drug Interventions

Dietary Supplements

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