Search clinical trials for: Nonaka Disease

Total 381571 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
Nanjing First Hospital, Nanjing Medical University

Recruiting

Safety and Efficacy of Recombinant Human Thyroid Stimulating Hormone for Adjuvant Diagnosis in Patients With Locally Advanced/Metastatic Differentiated Thyroid Cancer

Recombinant Human Thyroid Stimulating Hormone

China
Chia Tai Tianqing Pharmaceutical Group Nanjing...

Not yet recruiting

A Study of TQB2102 for Injection in the Treatment of HER2-positive Biliary Tract Cancer

Biliary Tract Cancer

China
Cairo University

Recruiting

Effects of Different Doses of Intravenous Lidocaine Infusion on Peri-operative Pain and Incidence of Postoperative Chronic Pain Within ERAS Protocols, a Dose Finding Study.

Perioperative Pain Management

Egypt
Ariel University

Not yet recruiting

Nutritional Therapy to Incretin-based Anti-obesity Medications in the Management of Gastrointestinal Adverse Events

Obesity; Drug

Israel
Universidad de los Andes, Chile

Recruiting

Intra-articular Injection of UC-MSC Exosome in Knee Osteoarthritis (EXO-OA01)

Osteo Arthritis Knee

Chile
University Hospital, Montpellier

Not yet recruiting

Feasibility and Efficiency of Screening for Neurodevelopmental Disorders by an Advanced Practice Nurse in Children With Congenital Heart Disease (NEURODEV-IPA)

Neurodevelopmental Disorders | Congenital Heart Disease in Children

France
Christian Riberholt

Not yet recruiting

Intensive Physical Exercise Versus Standard Exercise During Rehabilitation of Patients With Traumatic Brain Injury (INSPIRE)

Traumatic Brain Injury
Fundación GECP

Not yet recruiting

Study of Treatment With Sacituzumab and Zimberelimab for Patients With Lung Cancer Confined to the Chest and Previously Operated on Who Were Not Disease-free. (ARIAN)

Lung Diseases | Carcinoma, Non-Small-Cell Lung | Resectable Lung Non-Small Cell Carcinoma

Spain
Chong Kun Dang Pharmaceutical

Recruiting

Efficacy and Safety of Telmisartan Compared With Losartan

Hypertension | Diabetic Nephropathies

Korea, Republic of
Tanta University

Recruiting

General Anesthesia Versus Sedation By Dexmedetomidine and Ketamine With Local Infiltration for Percutaneous Transcatheter Closure of Atrial Septal Defect in Pediatric Patients

Atrial Septal Defect | Ketamine | General Anesthesia | Dexmedetomidine | Local Infiltration

Egypt
Priovant Therapeutics, Inc.

Not yet recruiting

A Phase 3 Study of Brepocitinib in Adults With Active, Non-Infectious, Non-Anterior Uveitis (CLARITY) (CLARITY)

Uveitis | Uveitis, Posterior | Uveitis, Intermediate
UMC Utrecht
Radboud University Medical Center; University Medical Center Groningen; Amsterdam...

Recruiting

The Electronic Cardiovascular Genetics (eCG) Clinic for Presymptomatic Genetic Counselling (eCG)

Inherited Cardiac Disease

Netherlands
IRCCS Policlinico S. Matteo
Federico II University; University of Roma La Sapienza

Recruiting

Effect of Fixed Combination Citicoline Homotaurine and Pyrroloquinoline Quinone on PERG in Glaucomas

Glaucoma | Neuroprotection

Italy
GlaxoSmithKline

Not yet recruiting

A Study to Find the Dose and Assess the Immune Response and Safety of a Vaccine Against Influenza in Healthy Younger and Older Adults

Influenza, Human

United States
Zhongshan Hospital Xiamen University

Not yet recruiting

Efficacy of Self vs. Physician Application of Triamcinolone-Econazole in Otomycosis

Otomycosis

China
Ironshore Pharmaceuticals and Development, Inc

Not yet recruiting

Ph3, Multicenter, 3wk Fixed-dose, Randomized, DBL, Placebo-controlled, Parallel-grp Efficacy, Safety & PK Study of Evening Dosed MPH HCl ER Capsules (HLD200) in Children 4-5 Yr With ADHD

Attention Deficit Hyperactivity Disorder
Sichuan University

Recruiting

Safety and Efficacy of Whole Brain LDRT+ICI+Intrathecal Chemotherapy in Refractory Meningeal Metastasis of Lung Cancer

NSCLC | PD-1 Inhibitor | Leptomeningeal Metastasis | Low Dose Radiotherapy

China
University Hospital, Caen

Recruiting

CORRECTION OF EAR DEFORMITIES IN NEWBORNS BY MODELING, COMPARISON OF TWO PROTOCOLS (MOLDEAR)

Ear Malformation

France
First Affiliated Hospital, Sun Yat-Sen University

Not yet recruiting

The Correlation Between Ripretinib Exposure and the Efficacy and Safety in Patients With Advanced GISTs

Gastrointestinal Stromal Tumors
Mahidol University

Not yet recruiting

Obstetric Comorbidity Index in Postpartum Hemorrhage

Cesarean Section Complications | Postpartum Hemorrhage | Morbidity;Perinatal

Thailand
Beijing Chao Yang Hospital

Recruiting

Magnetic Resonance Lung Function Imaging Study of Pulmonary Fibrosis Combined With Emphysema Syndrome

Using Functional Lung MRI to Explore Pulmonary Perfusion Changes in Patients With Pulmonary Fibrosis and Emphysema Syndrome

China
Nanjing First Hospital, Nanjing Medical University

Recruiting

Effect of Strontium-90 Brachytherapy Combined With Hyperthermia in the Treatment of Keloid

Keloid

China
Andrea Ravida
Association for Dental Infection Control

Recruiting

Efficacy of Non-surgical and Surgical Surface Decontamination Methods on Peri-implantitis-affected Implants

Peri-Implantitis | Dental Implant Failed

United States
Colgate Palmolive

Not yet recruiting

The Clinical Investigation of Colgate Dual Zinc Toothpaste as Compared to Colgate Cavity Protection Toothpaste in Controlling Established Plaque and Gingivitis

Gingivitis | Plaque, Dental

China
Centre Hospitalier Universitaire Dijon

Not yet recruiting

Study of the Value of hPG80 (Circulating Progastrin) for the Diagnosis of Neuroendocrine Tumours in Patients With an MEN1 Mutation (PRO-NEM1)

Neuroendocrine Tumors | MEN1 Mutation

France
China National Center for Cardiovascular Diseases

Recruiting

Potts-shunt for the Treatment of Pediatric Patients With Severe Pulmonary Hypertension

Pulmonary Artery Hypertension

China
University of Colorado, Denver
Usona Institute

Not yet recruiting

A Phase 1b Study of Psilocybin Assisted Psychotherapy to Address Fear of Recurrence

Breast Cancer | Ovarian Cancer

United States
Hager Abdo Mohamed

Not yet recruiting

Rate of Blood Pressure Control Among Hypertensive Patients With Hemodialysis in Sohag University Hospital

Hypertension | Hemodialysis

Egypt
Octapharma

Not yet recruiting

Post Marketing Observational Study on Safety of BALFAXAR vs. KCENTRA for Reversal of Vitamin K Antagonist Induced Anticoagulation in Adults Undergoing Urgent Surgery or Invasive Procedure

Significant Bleeding Risk | Vitamin K-Dependent Coagulation Defect
Children's National Research Institute

Not yet recruiting

Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

Rhabdomyolysis

United States
Renmin Hospital of Wuhan University

Recruiting

Metabolomic Profile of Vitreoretinal Diseases: an NMR-Based Approach Using Vitreous. (NMR)

Vitreoretinal Disease

China
Firat University

Not yet recruiting

The Effect of Sujok Therapy on Patient Comfort, Pain Intensity, and Anxiety Level in Cancer Patients With Port Catheter Placement: A Randomized, Placebo and Controlled Study

Cancer | Chemotherapy Effect

Turkey
Erzincan Military Hospital

Not yet recruiting

Evaluating the Efficacy and Safety of Preoperative Administration of Duloxetine for Pain Management in Women Undergoing Vaginal Hysterectomy.

Postoperative Pain | Vaginal Hysterectomy
Ionis Pharmaceuticals, Inc.

Not yet recruiting

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Baylor Research Institute
National Institute on Disability, Independent Living, and Rehabilitation...

Recruiting

Early Robotic Gait Training After Stroke (ERA Stroke)

Stroke

United States
Mansoura University

Recruiting

Prevalence Of Significant Endoscopic and Histopathologic Findings in Patients Presenting With Unexplained Iron Deficiency Anemia

Gastrointestinal Diseases

Egypt
Hookipa Biotech GmbH

Not yet recruiting

Safety, Reactogenicity and Immunogenicity of HB-502 and HB-501 Versus Placebo in People With HIV on Suppressive ART

Human Immunodeficiency Virus (HIV) Infection

United States
Istituto Clinico Humanitas
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Recruiting

A Novel Multiomic AI Approach for Early Preeclampsia Prediction in Pregnancy (PREMEM)

Preeclampsia

Italy
Medical University of Vienna
Lund University; Ziekenhuis Netwerk Antwerpen (ZNA); St. Antonius Hospital; Azienda... and other collaborators

Not yet recruiting

Outcomes of Local Treatment for Oligometastatic Prostate Cancer Diagnosed Using PSMA PET Imaging: OLIGOMET Study (OLIGOMET)

Prostate Cancer | Prostate Cancer Metastatic | Oligometastasis | Prostate Neoplasm | Oligometastatic Disease | Prostate Cancer Metastatic to Bone
Dr Alistair Mackett

Recruiting

Useability and Acceptability of the CUE1 Device in Older People With Parkinson's Disease

Parkinson Disease

United Kingdom

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