Search clinical trials for: Nonaka disease

Total 381439 results

D'Or Institute for Research and Education
Oswaldo Cruz Foundation; Rio de Janeiro State Research Supporting Foundation... and other collaborators

Not yet recruiting

Extracellular Vesicles From Mesenchymal Cells in the Treatment of Acute Respiratory Failure

Pneumonia | Acute Respiratory Distress Syndrome (ARDS) | Severe Acute Respiratory Syndrome (SARS)

Brazil
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
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
Nobelpharma

Completed

Efficacy Confirmation Study of NPC-09

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

Japan
Kitasato University
Aetas Pharma Co. Ltd.

Unknown

First-in-Patient Study for Sing le Dose of M201-A Hydrochloride Injection in Japanese Patients With Paroxysmal Atrial Fibrillation

Paroxysmal Atrial Fibrillation

Japan
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
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
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
Tohoku University

Completed

Pharmacokinetic Study on N-acetylneuraminic Acid

Hereditary Inclusion Body Myopathy | Nonaka Myopathy

Japan
Faming Zhang

Not yet recruiting

Effect of Antibiotic Pretreatment on the Efficacy of WMT in the Treatment of Chronic Constipation

Chronic Constipation

China
Necmettin Erbakan University
Health Institutes of Türkiye (TUSEB)

Not yet recruiting

The Impact of Probiotic on Survival and Treatment Response in Metastatic Non-small Cell Lung Cancer Patients

Metastatic Non-small Cell Lung Cancer

Turkey
Zhejiang Provincial People's Hospital

Not yet recruiting

Accuracy of an Artificial Intelligence-assisted Diagnostic System for Caries Diagnosis: a Prospective Multicenter Clinical Study

Dental Caries | Machine Learning | Diagnosis | Artificial Intellegence
Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical...

Recruiting

Low-potassium Content Vegetables in Chronic Kidney Disease (LPV in CKD)

the Recommended Target of Daily Vegetables Intake | Risk of Hyperkalemia

Taiwan
National Yang Ming Chiao Tung University

Not yet recruiting

Effectiveness of a Combined Diet and Mindfulness Program on Weight Loss in Breast Cancer Survivors

Breast Cancer | Weight Loss

Taiwan
Etlik Zubeyde Hanım Women's Health Care, Training...

Not yet recruiting

The Effect of Polycystic Ovary Syndrome Phenotypes on Quality of Life and Sexual Function (PCOS)

Depression | Sexual Dysfunction | Polycystic Ovary
University of Miami
Medtronic

Not yet recruiting

STARLITE for Unresectable High-Grade Gliomas (STARLITE)

High Grade Glioma

United States
The First Affiliated Hospital of Soochow University

Recruiting

Study of A Venetoclax-based, Anthracycline-free Regimen in Newly Diagnosed CBFβ::MYH11(+) AML

Acute Myeloid Leukemia

China
Pamukkale University

Not yet recruiting

Evaluation of Supraclavicular Brachial Plexus Blocks at Various Volumes: Impact on Optic Nerve Sheath Diameter

Intracranial Pressure Increase | Upper Limb Injury

Turkey
Boston Children's Hospital

Not yet recruiting

A Safety and Feasibility Trial Protocol of Metformin in Infants After Perinatal Brain Injury

Hypoxic-Ischemic Encephalopathy | Infant Development | Neurodevelopment | HIE
Merck Sharp & Dohme LLC

Not yet recruiting

Vericiguat in Pediatric Participants With Heart Failure Due to Left Ventricular Systolic Dysfunction (MK-1242-043)

Systolic Dysfunction
University of Virginia

Recruiting

Role of TXA in Patients Undergoing Breast Free Flap Reconstruction

Breast Cancer | Blood Loss, Surgical

United States
Shirley Ryan AbilityLab

Not yet recruiting

Impact of Sensory Electrical Stimulation on Sensation and Tremor

Essential Tremor | Healthy Individuals

United States
Carl Zeiss Meditec AG

Not yet recruiting

Evaluation of a Monofocal Intraocular Lens (PRECISE)

Cataract
Grupo Lusófona
Centro Hospitalar Universitario do Algarve; Universidade Lusófona de Humanidades... and other collaborators

Not yet recruiting

Home-Combo: an Online Home-based Combined Exercise Intervention for Women With Breast Cancer

Breast Cancer | Chemotherapy Effect

Portugal
Omer Ayhan

Recruiting

Fully Immersive Virtual Reality Applications in Children With Cerebral Palsy

Cerebral Palsy

Turkey
Link Campus University

Recruiting

Damage Control Surgery Over the World in Acute Diverticulitis (DACOSAD) (DACOSAD)

Open Abdomen | Diverticular Perforation

Italy
Merck Sharp & Dohme LLC

Not yet recruiting

Study of Belzutifan (MK-6482) Plus Fulvestrant for ER+/HER2- Metastatic Breast Cancer (MK-6482-029/LITESPARK-029)

Metastatic Breast Cancer
Central South University

Not yet recruiting

Investigating the Insulin Resistance in Individuals With Type 2 Diabetes

Type 2 Diabetes

China
Wright State University

Recruiting

The Preventive Role of Fractionated Laser Resurfacing Against Actinic Neoplasia in an At-Risk Geriatric Population

Aging | Actinic Keratoses | Non-Melanoma Skin Cancer

United States
Halic University

Not yet recruiting

The Relationship Between Body Perception and Self-Esteem Level and Quality of Life in Adolescent Idiopathic Scoliosis

Quality of Life | Scoliosis; Adolescence | Self Esteem

Turkey
AbbVie

Not yet recruiting

A Study to Evaluate the Risk of Tumor Lysis Syndrome (TLS) in Adult Participants Receiving Oral Venetoclax in Combination With Intravenously Infused Obinutuzumab or Oral Acalabrutinib for Previously Untreated Chronic Lymphocytic Leukemia (CLL)

Chronic Lymphocytic Leukemia
Blokhin's Russian Cancer Research Center

Recruiting

Neoadjuvant Prolgolimab Monotherapy in Locally Advanced MMR-deficient Colorectal Cancer

Colorectal Cancer

Russian Federation
Universidad de Murcia

Recruiting

Three Types of Papilla Incision in Periodontal Surgery

Periodontitis | Periodontal Diseases | Periodontal Pocket

Spain
Merck Sharp & Dohme LLC

Not yet recruiting

A Clinical Study of MK-2870 Alone or With Chemotherapy to Treat Gastrointestinal Cancers (MK-9999-02A)

Colorectal Cancer | Biliary Tract Cancer | Pancreatic Ductal Carcinoma
I.R.C.C.S. Fondazione Santa Lucia

Not yet recruiting

REducing the Risk of COgnitive DEcline ad Dementia in Patients With Subjective Cognitive Decline Through an Immersive Virtual Reality and Telemedicine-based Multi-component Intervention: the SCD-ReCODED Study (SCD-ReCODED)

Subjective Cognitive Decline
Novartis Pharmaceuticals

Not yet recruiting

Clinical Study of Asciminib in Previously Treated Indian Patients With Ph+ CML-CP Without T315I Mutation and in Patients With Ph+ CML-CP With T315I Mutation (ASC4INDIA)

Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Massachusetts General Hospital
American Heart Association

Recruiting

Impact of Cognitive Behavioral Therapy on PTSD-CVD Link

Posttraumatic Stress Disorder

United States
Massachusetts General Hospital
United States Department of Defense

Not yet recruiting

Mobile Application to Improve Health-Related Outcomes in Patients With Advanced Lung Cancer (THRIVE)

Lung Cancer
The First Affiliated Hospital with Nanjing Medical...

Not yet recruiting

Study on the Mechanism of ZhenQiFuZheng in the Treatment of Allergic Rhinitis Based on Intestinal Flora and Metabolites

Rhinitis, Allergic
Seoul National University Hospital

Not yet recruiting

Investigator-initiated Clinical Trial to Observe Conjunctival Goblet Cell Using an Anterior Segment Imaging Device

Ocular Surface Disease
Assiut University

Not yet recruiting

Incidence of Early Infection After Bipolar Hemiarthroplasty and Its Associated Factors in Fracture Neck of Femur

Prosthetic-joint Infection
Luminopia

Recruiting

Clinical Data Registry of Amblyopia Patients on Luminopia Treatment

Amblyopia

United States
Malmö University

Not yet recruiting

Treatment Effects of Two Pharmaceutical Skin Care Creams for Xerotic Feet Among Persons With Diabetes

Diabetes Mellitus | Skin and Connective Tissue Diseases
Tandem Diabetes Care, Inc.

Recruiting

Tandem Freedom - Feasibility Trial 1

Diabetes Mellitus, Type 1

New Zealand
Dr George Medvedev
H. Lundbeck A/S; Royal Columbian Hospital; Centre for Neurology Studies, Surrey... and other collaborators

Not yet recruiting

Eptinezumab as an Adjunct to Standard of Care for Migraine in an Acute Emergency Context

Migraine | Chronic Migraine

Canada
Ankara City Hospital Bilkent

Not yet recruiting

Relationship Between Preoperative Subcutaneous Trochanteric Fat Thickness and Postoperative Infection Risk

Hip Infection

Turkey
SpliSense Ltd.

Recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of SPL84 in Patients With Cystic Fibrosis

Cystic Fibrosis

United States
Tufts University

Not yet recruiting

Systemic Absorption of Dexamethasone Oral Rinse in Patients With Oral Lichen Planus

Oral Lichen Planus | Oral Lichenoid Reaction

United States
Bayer

Not yet recruiting

A Study to Learn About the Safety of BAY3283142 in People With Mild to Moderate High Blood Pressure

Chronic Kidney Disease | Arterial Hypertension | Non-proliferative Diabetic Retinopathy

Germany

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