Clinical Trials on Myopathia, központi mag

Total 454 results

Vastra Gotaland Region

Enrolling by invitation

Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no Treatment (COMPIS)

Musculoskeletal Diseases | Neuromuscular Diseases | Centronuclear Myopathy | Nemaline Myopathy | Congenital Myopathy | Myosin Storage Myopathy

Sweden
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
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
Radboud University Medical Center
UMC Utrecht

Not yet recruiting

The Natural History and Muscle Fatigability of Patients With Congenital Myopathies. (READYCOM)

Centronuclear Myopathy | Nemaline Myopathy | Central Core Disease | Multi-Minicore Disease

Netherlands
Peking University People's Hospital

Unknown

Low-dose Interleukin-2 Treatment on Idiopathic Inflammatory Myopathy

Inflammatory Myopathy
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
Peking University People's Hospital

Unknown

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

Inflammatory Myopathy
Reata, a wholly owned subsidiary of Biogen
AbbVie

Completed

RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR

MItochondrial Myopathies

United States, Denmark
James Dowling
Canadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaborators

Recruiting

Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM)

X Linked Myotubular Myopathy

United Kingdom, Canada, United States
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Myotubular and Centronuclear Myopathy Patient Registry

X-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-Linked

United Kingdom
Cristina Domínguez González

Recruiting

Natural History in Primary Mitochondrial Myopathies (NHPMM)

Primary Mitochondrial Myopathies

Spain
Dynacure

Terminated

Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (Unite-CNM)

Centronuclear Myopathy

France, Netherlands, United Kingdom, Belgium, Denmark, Germany
Dynacure

Withdrawn

Natural History Study of Patients With Centronuclear Myopathies (NatHis-CNM)

Centronuclear Myopathy
Boston Children's Hospital
Muscular Dystrophy Association

Recruiting

Molecular and Genetic Studies of Congenital Myopathies

Centronuclear Myopathy | Myotubular Myopathy | Nemaline Myopathy | Central Core Disease | Congenital Fiber Type Disproportion | Multiminicore Disease | Rigid Spine Muscular Dystrophy | Undefined Congenital Myopathy

United States
Peking University People's Hospital

Recruiting

Clinical Characteristics and Mechanism Research of Inhibitors of Janus Kinase in the Idiopathic Inflammatory Myopathies

Idiopathic Inflammatory Myopathies

China
University Hospital, Brest

Recruiting

Adult Autoimmune Myopathies (MAIA) (MAIA)

Idiopathic Inflammatory Myopathies

France
Astellas Gene Therapies

Active, not recruiting

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy (ASPIRO)

X-Linked Myotubular Myopathy

United States, Canada, France, Germany
Institut de Myologie, France
Dynacure

Completed

Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies (NatHis-CNM)

Centronuclear Myopathy

France, Belgium, Germany, Italy, Spain
Cure CMD
University of Chicago; Boston Children's Hospital; Valerion Therapeutics, LLC; Congenital...

Completed

Myotubular Myopathy Genetic Testing Study

Myotubular Myopathy

United States
The First Affiliated Hospital with Nanjing Medical...

Not yet recruiting

Adult Idiopathic Inflammatory Myopathy With Cardiac Injury

Cardiac Disease | Inflammatory Myopathy
Astellas Gene Therapies

Completed

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

X-linked Myotubular Myopathy

United States, France, United Kingdom, Canada, Germany
University of Florida
Audentes Therapeutics

Completed

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

X-linked Myotubular Myopathy

United States
University of Manchester
Karolinska Institutet; Eli Lilly and Company; Clinical Trials Unit, Manchester

Completed

JAK 1/2 Inhibitor, Baricitinib, in the Treatment of Adult IIM (MYOJAK)

Idiopathic Inflammatory Myopathies

United Kingdom
Yale University

Completed

Application of Electrical Impedance Myography (EIM) as a Potential Biomarker of Idiopathic Inflammatory Myopathies

Idiopathic Inflammatory Myopathies

United States
Benha University

Unknown

Elastography in Patients With Idiopathic Inflammatory Myopathies

Idiopathic Inflammatory Myopathies

Egypt
Reneo Pharma Ltd

Terminated

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Primary Mitochondrial Myopathy

Spain, Australia, Belgium, Denmark, Canada, United Kingdom, France, Germany, Hungary, Italy, Netherlands, New Zealand
University of Sao Paulo

Completed

Physical Training in Patients With Idiopathic Inflammatory Myopathies

Physical Activity | Idiopathic Inflammatory Myopathies

Brazil
Cure CMD
University of Michigan; Congenital Muscle Disease International Registr

Completed

Myotubular Myopathy Event Study (MTMES)

X-linked Myotubular Myopathy

United States
The Emmes Company, LLC
United Mitochondrial Disease Foundation; PolG Foundation Mito Australia

Recruiting

Mitochondrial Video Assessment Source Material Study

PolG, Primary Mitochondrial Myopathy

United States
Dynacure

Withdrawn

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

Centronuclear Myopathy

Germany, France
Reneo Pharma Ltd

Completed

An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients (STRIDE)

Primary Mitochondrial Myopathy

United States, Spain, Australia, France, United Kingdom, New Zealand, Denmark, Norway, Czechia, Belgium, Canada, Germany, Hungary, Italy, Netherlands
Stealth BioTherapeutics Inc.

Terminated

A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension (MMPOWER-3)

Primary Mitochondrial Myopathy

United States, Germany, Canada, Italy, United Kingdom, Denmark, Hungary
Reneo Pharma Ltd

Terminated

A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy

Primary Mitochondrial Myopathy

United Kingdom
Astellas Gene Therapies

Completed

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Males With X-linked Myotubular Myopathy (XLMTM)

United States, 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
Centre Hospitalier Universitaire de Saint Etienne
Ministry of Health, France

Recruiting

Rituximab Therapy in Anti-Myelin Associated Glycoprotein Patients With Characteristics of Good Responders (THERAMAG)

Anti-MAG Neuropathy

France
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
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
Astellas Pharma Inc

Active, not recruiting

A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy (MOUNTAINSIDE)

Primary Mitochondrial Myopathy

United States
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, Thailand, United States, Bulgaria, Taiwan, Argentina, Belgium, Cyprus, Denmark, France, Georgia, Germany, Greece, Italy, Lithuania, Czechia, Sweden
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
Stealth BioTherapeutics Inc.

Completed

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

Mitochondrial Myopathy

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
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
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
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

Clinical Trials on Myopathia, központi mag

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