Search clinical trials for: Distal myopathy with rimmed vacuoles (DMRV)

Total 159150 results

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

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
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
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
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
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
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)
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
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
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
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

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

Hereditary Inclusion Body Myopathy (HIBM)

United States
First Affiliated Hospital of Wenzhou Medical University
Shanghai First Song Therapeutics Co., Ltd

Not yet recruiting

The Safety and Efficacy of Anti-CD19 CAR-T Cells in Patients With Relapsed/Refractory Autoimmune Diseases

Systemic Sclerosis | Sjogren's Syndrome | Antiphospholipid Syndrome | ANCA Associated Vasculitis | Systemic Lupus Erythematosus (SLE) | Inflammatory Myopathy

China
Teeradon Waewworawit

Not yet recruiting

Efficacy of Intra-Articular Corticosteroid Injections on Clinical Outcomes in Patients With Operative Distal Radius Fractures

Wrist Fractures | Intraarticular Injection
Sohag University

Recruiting

Snodgrass Versus Grafted Snodgrass Repair in Narrow Urethral Plate in Distal Penile Hypospadias

Hypospadias

Egypt
Nordsjaellands Hospital

Recruiting

Ultrasound-guided Infraclavicular Block With Lidocaine or Ropivacaine for Closed Reduction of Distal Radius Fractures

Colles' Fracture | Fracture Dislocation | Distal Radius Fractures | Lidocaine | Ropivacaine | Closed Reduction of Fracture and Application of Plaster Cast

Denmark
China Immunotech (Beijing) Biotechnology Co., Ltd.
Shanghai Changzheng Hospital

Not yet recruiting

An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease

Systemic Lupus Erythematosus | Systemic Sclerosis | Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis | Sjogren's Syndrome | Antiphospholipid Syndrome | Inflammatory Myopathy
Ain Shams University

Recruiting

The Efficiency of Clear Aligners in Maxillary Molar Distalization: A Randomized Clinical Trial. (Aligner)

Orthodontic Aligners

Egypt
Cardenal Herrera University

Recruiting

Consequences on the Gait Pattern of Patients With Distal Femur Megaprostheses (IMU-DFM)

Distal Femoral Musculoskeletal Tumors

Spain
Ahi Evran University Education and Research Hospital

Recruiting

Fluidotherapy in Patients With Distal Radius Fractures

Pain | Distal Radius Fractures | Edema Arm

Turkey
Aarhus University Hospital
University of Aarhus; Central Denmark Region; Vejle Hospital; TrygFonden, Denmark; Aalborg University Hospital and other collaborators

Recruiting

Patient Education and Duloxetine, Alone and in Combination, for Patients With Multisystem Functional Somatic Disorder (EDULOX)

Functional Disorder

Denmark
Sichuan University

Not yet recruiting

Billroth II With Braun Anastomosis After Radical Distal Gastrectomy for Gastric Cancer

Gastric Cancer | Surgery | Gastrostomy | Anastomosis
Diskapi Teaching and Research Hospital

Recruiting

Anterior Cutaneus Nerve Block With USG for Total Knee Replacement

Knee Replacement Arthroplasty | Nerve Block | Pain Measurement

Turkey
Ahi Evran University Education and Research Hospital

Recruiting

Effectiveness of Instrument-Assısted Soft Tissue Mobılızatıon in Patients With Operated Distal Radius Fracture

Distal Radius Fractures | Instrument-assisted Soft Tissue Mobilization

Turkey
Al-Azhar University

Not yet recruiting

Results of Distal Clavicle Graft in Shoulder Dislocation Clavicle Auto Graft in Patient With Recurrent Anterior Shoulder Dislocation

Shoulder Injuries
Assiut University

Recruiting

The Use of a US-measured Styloid Process of the Distal Radius EET Diameter for Prediction of ETT Size (ETT/US)

Airway Management

Egypt
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
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
National Human Genome Research Institute (NHGRI)

Recruiting

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Metabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditions

United States
University of Kansas Medical Center

Not yet recruiting

Opioid-Free Pain Treatment in Trauma Patients

Femoral Neck Fractures | Distal Radius Fractures | Femoral Shaft Fracture | Intertrochanteric Fractures | Patella Fracture | Lisfranc Injuries | Distal Femur Fracture | Calcaneus Fractures | Clavicle Fractures | Proximal Humerus Fractures | Tibial Shaft Fracture With or Without Associated Fibula Fracture | Ankle... and other conditions

United States
UK Kidney Association

Recruiting

National Registry of Rare Kidney Diseases (RaDaR)

Vasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditions

United Kingdom
Children's Hospital of Philadelphia
National Institute of Neurological Disorders and Stroke (NINDS); North American...

Recruiting

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

Mitochondrial Diseases | Mitochondrial Myopathies | MELAS | Mitochondrial Encephalomyopathies | MERRF

United States
Saglik Bilimleri Universitesi Gazi Yasargil Training...

Recruiting

Increasing Security With End-zone Arterial Blood Pressure Monitoring During Surgery

Catheterization, Peripheral

Turkey
Hospital Costa del Sol

Recruiting

Inmobilization With Compression Bandage vs Antebraquial Splint in Distal Radius Fractures

Distal Radius Fractures | Internal Fixation; Complications, Mechanical

Spain
Grigore T. Popa University of Medicine and Pharmacy
Aristotle University Of Thessaloniki

Recruiting

Maxillary Molar Distalization Supported by Mini-implants With the Advanced Molar Distalization Appliance (Amda®)

Angle Class II

Romania
The First Affiliated Hospital with Nanjing Medical...

Not yet recruiting

Adult Idiopathic Inflammatory Myopathy With Cardiac Injury

Cardiac Disease | Inflammatory Myopathy
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
Bausch Health Americas, Inc.

Not yet recruiting

Dose Ranging Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Budesonide Rectal Foam in Pediatric Patients Aged 5 to 17 Years With Active, Mild to Moderate Distal Ulcerative Colitis

Ulcerative Colitis
Omeicos Therapeutics GmbH

Recruiting

OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) (PMD-OPTION)

Primary Mitochondrial Disease

Germany, Italy
Riphah International University

Recruiting

Neurodynamic Sequencing in Patients With Carpal Tunnel Syndrome

Carpal Tunnel Syndrome

Pakistan
Hangzhou Endonom Medtech Co., Ltd.

Not yet recruiting

A Real-World Study on Distal Aortic Remodeling for Stanford Type B Aortic Dissection With Fabulous Thoracic Aortic Stent System

Aortic Dissection

China
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
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
Aphaia Pharma US LLC

Recruiting

A Study to Evaluate Efficacy and Safety of Distal Jejunal-release Dextrose Beads Formulation (APHD-012) in Subjects With a Pathological Oral Glucose Tolerance Test (OGTT)

PreDiabetes | Glucose Tolerance Impaired

Slovakia
University of Florida

Recruiting

Distal Femur Fx: ORIF(Open Reduction and Internal Fixation) vs DFR (Distal Femur Replacement Total Knee Arthroplasty)

Distal Femur Fracture

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

Not yet recruiting

R-MINE+X in Patients With Relapsed/Refractory Diffuse Large B-cell Lymphoma

Diffuse Large B-cell Lymphoma
Birmingham Women's and Children's NHS Foundation...

Not yet recruiting

Use of Indocyanine Green During Primary Repair of Oesophageal Atresia and Distal Tracheo-oesophageal Fistula (iTOF)

Tracheo-Esophageal Fistula With Atresia of Esophagus

United Kingdom
Nova Scotia Health Authority

Recruiting

Latarjet vs Anatomic Glenoid Reconstruction

Bone Loss | Shoulder Dislocation | Arthroscopy

Canada
Centre Hospitalier Sud Francilien

Recruiting

Early Recanalization After Intravenous Thrombolysis With Tenecteplase Versus Alteplase in Distal Vessel Occlusion Strokes (DISTAL-IVT)

Acute Distal Vessel Occlusion Stroke

France
University Hospital of Patras

Not yet recruiting

Distal Versus Conventional Transradial Artery Access for Coronary Catheterization in Patients With STEMI (DR-STEMI)

Coronary Artery Disease | ST Elevation Myocardial Infarction | Percutaneous Coronary Intervention | Coronary Catheterization | Distal Transradial Artery | Transradial Artery | Primary PCI

Belgium, Greece, Switzerland

Search clinical trials for: Distal myopathy with rimmed vacuoles (DMRV)

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