Search clinical trials for: Hereditary inclusion body myopathy (hIBM)

Total 20407 results

Institut de Myologie, France

Completed

Clinical, Biological and NMR Outcome Measures Study for Hereditary Inclusion Body Myopathy Due to Mutation of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine Kinase Gene (GNE) (ClinBio-GNE)

HIBM

France
Tohoku University

Completed

Pharmacokinetic Study on N-acetylneuraminic Acid

Hereditary Inclusion Body Myopathy | Nonaka Myopathy

Japan
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

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

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

Hereditary Inclusion Body Myopathy (HIBM)

United States
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
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)

Completed

Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy

Hereditary Inclusion Body Myopathy

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
Nobelpharma

Completed

Efficacy Confirmation Study of NPC-09

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

Japan
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
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
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
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
Newcastle University

Unknown

International GNE Myopathy Patient Registry

Hereditary Inclusion Body Myopathy | GNE Myopathy

United Kingdom
University of California, Irvine

Recruiting

Characterization of Inclusion Body Myopathy Associated With Paget's Disease of Bone and Frontotemporal Dementia (IBMPFD)

Frontotemporal Dementia | Myopathy | Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia | Paget Disease of Bone

United States
Abcuro, Inc.
Syneos Health

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of ABC008 for Inclusion Body Myositis

Inclusion Body Myositis

United States, Australia, Belgium, France, Canada, Germany, United Kingdom
Novartis Pharmaceuticals

Terminated

Efficacy and Tolerability of BAF312 in Patients With Polymyositis

Polymyositis

Hungary, Taiwan, United States, Canada, Czechia, Poland
Novartis Pharmaceuticals

Terminated

Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

Dermatomyositis | Polymyositis

Hungary, United States, Czechia, Sweden, United Kingdom
National Institute of Neurological Disorders and...

Completed

Immune Abnormalities in Sporadic Inclusion Body Myositis

Myositis | Sporadic | Inclusion Body

United States
Ludwig-Maximilians - University of Munich

Completed

Natural History in CCFDN and IBM Syndromes

Inclusion Body Myositis, Sporadic | Inclusion Body Myopathy, Autosomal-recessive | Inclusion Body Myopathy, Autosomal-dominant | Congenital Cataracts, Facial Dysmorphism, And Neuropathy

Germany
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
Chugai Pharmaceutical

Not yet recruiting

Phase 1b Trial of RAY121 in Immunological Diseases (RAINBOW Trial)

Antiphospholipid Syndrome (APS) | Immune Thrombocytopenia (ITP) | Dermatomyositis (DM) | Bullous Pemphigoid (BP) | Behçet's Syndrome (BS) | Immune-mediated Necrotizing Myopathy (IMNM)

United States, Japan
Johns Hopkins University
American Heart Association; Morgan State University

Not yet recruiting

Developing a Learning COmmunity to Increase eNgagemeNt and Enrollment in Cardiovascular Clinical Trials (CONNECT) (CONNECT)

Cardiovascular Diseases | Stroke | Hypertension | Hyperlipidemias | Recruitment | Overweight and Obesity | Diabetes Type 2
Medical University of Vienna
University of Vienna; Griffith University

Recruiting

Brown Adipose Tissue Activity in Gilbert's Syndrome

Gilbert Syndrome

Austria
University of Illinois at Urbana-Champaign
Kenneth Dallmier Consulting, LLC

Recruiting

The Effect of the Inclusion of Soybean Flour on Glycemic Responses

Overweight and Obesity

United States
Duke University
National Human Genome Research Institute (NHGRI)

Not yet recruiting

Genomic Medicine Risk Assessment Care for Everyone - Implementation Phase (GRACE)

Liver Diseases | Cardiovascular Diseases | Cancer | Hyperthermia

United States
Instituto de Investigación Hospital Universitario...
Instituto de Salud Carlos III

Not yet recruiting

Efficacy, Safety and Cost-efficacy of a Pre-emptive Genotyping Strategy in Patients Receiving Statins (PREVESTATGx)

Cardiovascular Diseases | Dyslipidemias | Statin Adverse Reaction | Pharmacogenic Myopathy

Spain
Rolfs Consulting und Verwaltungs-GmbH (RCV)
Rhythm Pharmaceuticals, Inc.

Recruiting

Early Genetic Identification of Obesity (WEGIO)

Cognitive Impairment | Hyperphagia | Obesity, Childhood | Bardet-Biedl Syndrome | Polydactyly | Retinopathy | Syndactyly | POMC Deficiency

Germany
University of Kansas Medical Center
BioSensics

Recruiting

Monitoring Biomarker for Detecting Change in Physical Activity and Limb Function in Inclusion Body Myositis Over Time

Inclusion Body Myositis

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
Aristotle University Of Thessaloniki
Papageorgiou General Hospital

Recruiting

Evaluation of Bone Metabolism in Children and Adolescents With Familial Mediterranean Fever (FMF)

Familial Mediterranean Fever

Greece
University Hospital, Montpellier

Not yet recruiting

Assessment of microRNAs Role in Familial Mediterranean Fever FMF Pathophysiology (miRinFMF)

Familial Mediterranean Fever

France
University Hospital, Toulouse

Not yet recruiting

Evaluate the Evolution of Body and Scalp Skin Discomfort in Patients With Hereditary Ichthyosis After Wraps (EnvelopIchtyose)

Ichthyosis Inherited

France
LMU Klinikum

Recruiting

The Risk of Falls Index for Patients With Neuromuscular Disorders

Myasthenia Gravis | Myotonic Dystrophy | Inclusion Body Myositis | Facioscapulohumeral Muscular Dystrophies | The Falls Efficacy Scale International | The Morse Fall Scale

Germany
University of Aarhus

Recruiting

Muscular Deficits of Diabetic Myopathy

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

Denmark
Johns Hopkins University

Recruiting

Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1

Neurofibromatosis 1 | Atypical Neurofibroma | Malignant Peripheral Nerve Sheath Tumor (MPNST) of Soft Tissue (Diagnosis)

United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Evaluating the Genetics and Immunology of Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome and Other Tonsil Disorders

Obstructive Sleep Apnea | Sleep Disordered Breathing | Tonsillitis | Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa) | Tonsil Disorder

United States
Marmara University

Recruiting

Investigation of Autonomic Dysfunction in Patients With Familial Mediterranean Fever

Familial Mediterranean Fever | Autonomic Dysfunction

Turkey
Sohag University

Not yet recruiting

Clinical and Molecular Evaluation of Childern With Familial Meditterranean Fever and Their Siblings

Familial Mediterranean Fever

Egypt
LMU Klinikum

Recruiting

Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease

Pompe Disease (Late-onset) | FSHD | Spinal Muscular Atrophy Type 3 | Inclusion Body Myositis

Germany
R-Pharm International, LLC
Atlant Clinical LLC; Center for Pharmaceutical Analytics LLC; Unimed Laboratories... and other collaborators

Recruiting

Safety and Efficacy of RPH-104 Used to Prevent Recurrent Fever Attacks in Adult Patients With Colchicine Resistant or Colchicine Intolerant Familial Mediterranean Fever

Familial Mediterranean Fever | FMF

Armenia, Georgia, Russian Federation
Tasly GeneNet Pharmaceuticals Co., Ltd

Recruiting

A Study to Assess the Efficacy and Safety of PXT3003 in Charcot-Marie-Tooth Type 1A

Charcot-Marie-Tooth Type 1A

China
R-Pharm International, LLC
R-Pharm; Atlant Clinical LLC; Unimed Laboratories CJSC; Data Management 365; Center...

Recruiting

Efficacy and Safety of RPH-104 for Resolution and Prevention of Recurring Attacks in Adult Subjects With Familial Mediterranean Fever With Resistance to or Intolerance of Colchicine

Familial Mediterranean Fever | FMF

Armenia, Georgia, Russian Federation
Assistance Publique - Hôpitaux de Paris

Recruiting

Cell Therapy for IBM by Muscle Injection of ADSVF (ADSVF-in-IBM)

Inclusion Body Myositis

France
University of Kansas Medical Center
The Perron Institute

Recruiting

Phase III Trial of Sirolimus in IBM

Inclusion Body Myositis

United States, Australia
Nicole Baca

Recruiting

Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence

Neurofibromatosis Type 1

United States
University of Greenwich
Ingesport Health Spa Consulting

Not yet recruiting

Long-term Intervention Effects of a Personalized, Home-based Exercise Program and Nutritional Advice, Along With the Inclusion of a Pre-workout Multi-ingredient Supplement on the Body Composition, Functional Capacity and Quality of Life of Middle-aged and Older Adults (40-70 Years)

Quality of Life | Obesity | Stress, Psychological | Self-Perception | Weight Loss | Happiness | Obesity, Abdominal | Muscle Loss

United Kingdom

Search clinical trials for: Hereditary inclusion body myopathy (hIBM)

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Zambia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations