Search clinical trials for: Lipid Metabolism, Inborn Errors

Total 24103 results

Laval University

Recruiting

Unravelling the Impact of Diet on Cardiovascular Health in Treated Heterozygous Familial Hypercholesterolemia.

Familial Hypercholesterolemia | Cholesterol, Elevated | Nutrition, Healthy

Canada
Fondazione SISA (Societa Italiana per lo Studio...

Recruiting

Lipid Transport Disorder Italian Genetic Record (LIPIGEN) (LIPIGEN)

Familial Hypercholesterolemia | Genetic Disorder

Italy
Hospital do Coracao
University of Sao Paulo; Instituto Dante Pazzanese de Cardiologia; National Institute...

Not yet recruiting

An Adapted Brazilian Cardioprotective Diet, Phytosterols and Krill Oil in Familial Hypercholesterolemia (DICA-FH) (DICA-FH)

Familial Hypercholesterolemia
Chiesi Farmaceutici S.p.A.
ICON plc

Not yet recruiting

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (FLY)

Fabry Disease
Caroline Michaela Kistorp
Sanofi

Not yet recruiting

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease (FORTIFY)

Chronic Kidney Diseases | Fabry Disease

Denmark
Forge Biologics, Inc

Not yet recruiting

Long-term Follow-up Study to Evaluate Safety and Efficacy of FBX-101 in Krabbe Patients

Krabbe Disease

United States
Suzhou Municipal Hospital

Not yet recruiting

Safety and Efficacy Study of NGGT006 in Refractory Hypercholesterolemia Patients

Familial Hypercholesterolemia | Refractory Hypercholesterolemia
Ondokuz Mayıs University

Recruiting

The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence

Quality of Life | Urinary Incontinence | Menopause | Wolman Disease | Psychological Well-Being

Turkey
Novartis Pharmaceuticals

Recruiting

Specified Drug-use Survey of Leqvio for s.c. Injection.

Hypercholesterolaemia | Familial Hypercholesterolaemia

Japan
Xinhua Hospital, Shanghai Jiao Tong University...
Shanghai Vitalgen BioPharma Co., Ltd.

Recruiting

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Type II Gaucher Disease

China
UniQure Biopharma B.V.

Recruiting

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

Fabry Disease

United States
Leadiant Biosciences, Inc.

Not yet recruiting

A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)

Cerebrotendinous Xanthomatoses
Brunel University
Royal Free Hospital NHS Foundation Trust

Recruiting

A Co-designed Physical Activity Intervention in Fabry Disease

Fabry Disease

United Kingdom
Chung-Hsing Wang
Sanofi

Not yet recruiting

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Gaucher Disease

Taiwan
University of Cambridge
Sanofi

Recruiting

Molecular Imaging in Fabry Disease of the Heart

Fabry Disease, Cardiac Variant

United Kingdom
University of Pennsylvania

Recruiting

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study (LCAT NHS)

LCAT Deficiency

United States
Children's Hospital of Fudan University

Not yet recruiting

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Fabry Disease

China
New York Stem Cell Foundation Research Institute

Recruiting

NYSCF Scientific Discovery Biobank

PTSD | Multiple Sclerosis | Cervical Cancer | Diabetes Mellitus, Type 2 | Diabetes Mellitus | Diabetes | Macular Degeneration | Diabetes Mellitus, Type 1 | Parkinson Disease | Amyotrophic Lateral Sclerosis | Dementia | Ovarian Cancer | Alzheimer Disease | ALS | Vulvar Cancer | Post Traumatic Stress Disorder | Lewy Body Disease and other conditions

United States
Sanofi
Pulse Infoframe Ltd.

Recruiting

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)

Niemann-Pick Diseases | Acid Sphingomyelinase Deficiency

United States
Qilu Hospital of Shandong University

Recruiting

Screening Patients With Fabry Disease in Patients With Hypertrophic Cardiomyopathy or Left Ventricular Hypertrophy (SEARCH)

Fabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular Hypertrophy

China
Verve Therapeutics, Inc.

Not yet recruiting

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

Heterozygous Familial Hypercholesterolemia | Premature Coronary Heart Disease
He Huang
Lingyi Biotech Co., Ltd.

Recruiting

A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I (LY-M001)

Gaucher Disease Type I

China
First Affiliated Hospital Xi'an Jiaotong University

Recruiting

NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia

China
Lysosomal and Rare Disorders Research and Treatment...

Recruiting

Biomarkers Related to Bone in Pediatric Gaucher Disease

Bone Diseases | Gaucher Disease

United States
AceLink Therapeutics, Inc.
Tigermed Consulting Co., Ltd

Recruiting

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

Fabry Disease

China
Verve Therapeutics, Inc.

Not yet recruiting

Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease

Hypercholesterolemia | Atherosclerotic Cardiovascular Disease | Heterozygous Familial Hypercholesterolemia

New Zealand, United Kingdom
Centre Hospitalier Universitaire de la Réunion

Recruiting

Impact of Lipodystrophy on the Inflammatory State of Non-diabetic Dunnigan Reunion Island Subjects (LIPOKINES)

Lipodystrophy | Dunnigan Syndrome

Réunion
Westfälische Wilhelms-Universität Münster
Chiesi GmbH

Recruiting

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa (GoPEG)

Fabry Disease

Germany
ISU Abxis Co., Ltd.

Recruiting

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

Fabry Disease

Korea, Republic of
Jerry Vockley, MD, PhD
Ultragenyx Pharmaceutical Inc

Recruiting

Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Medium-chain Acyl-CoA Dehydrogenase Deficiency

United States
Jerry Vockley, MD, PhD
Acer Therapeutics Inc.

Recruiting

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Medium-chain Acyl-CoA Dehydrogenase Deficiency

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
Lysosomal and Rare Disorders Research and Treatment...

Recruiting

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Fabry Disease

United States
Boston Children's Hospital
Children's Hospital Medical Center, Cincinnati

Recruiting

Shwachman Diamond Syndrome Registry and Study (SDSR)

Shwachman-Diamond Syndrome | Shwachman-Diamond Syndrome-Like

United States
Sanofi

Recruiting

Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan

Fabry Disease

Taiwan
Institut National de la Santé Et de la Recherche...

Recruiting

MPS (RaDiCo Cohort) (RaDiCo-MPS)

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Mucopolysaccharidosis III | Mucopolysaccharidosis IV | Mucopolysaccharidosis VII | Multiple Sulfatase Deficiency Disease | Mucopolysaccharidosis IX

France
Sanofi

Recruiting

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme (SHORTEN)

Fabry's Disease

United States
Minovia Therapeutics Ltd.

Recruiting

Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-201 in Pediatric Patients With Pearson Syndrome

Mitochondrial Diseases | Pearson Syndrome

Israel
Beijing Suncadia Pharmaceuticals Co., Ltd

Not yet recruiting

Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia
Fundacion para la Investigacion Biomedica del Hospital...
Fundación Mutua Madrileña

Recruiting

Autoimmune and Inflammatory Response Biomarkers in Fabry Disease (Bio-FAIR)

Fabry Disease

Spain
NewAmsterdam Pharma

Recruiting

Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies (TANDEM)

Dyslipidemias | Hypercholesterolemia | Familial Hypercholesterolemia | High Cholesterol | ASCVD

United States
National Institute of Diabetes and Digestive and...

Recruiting

Feasibility of Adipose Tissue Triglyceride (TG) Labelling in Familial Partial Lipodystrophy (FPLD)

Lipodystrophy

United States
Fundación Española de Hematología y Hemoterapía

Recruiting

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Gaucher Disease | Acid SphingoMyelinase Deficiency

Spain
Hospices Civils de Lyon

Recruiting

T1 Mapping in Fabry Disease (MAP-FAB)

Fabry Disease

France
Eline C. B. Eskes

Recruiting

Biomarkers Of Lung InVolvement In ASMD (BOLIVIA)

Asmd, Visceral Type

Netherlands
Sanofi

Recruiting

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Gaucher Disease

United States
Visirna Therapeutics HK Limited

Not yet recruiting

A Phase 3 Study of VSA001 in Chinese Adults With Familial Chylomicronemia Syndrome

Familial Chylomicronemia Syndrome
Eline C. B. Eskes

Recruiting

Magnetic Resonance Spectroscopy in Acid Sphingomyelinase Deficiency (MONACO)

Asmd, Visceral Type

Netherlands
GeneCradle Inc

Not yet recruiting

Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreatitis

Hypertriglyceridemia, Familial

China
Visirna Therapeutics HK Limited

Not yet recruiting

A VSA003 Phase 1 Study in Chinese Adult Healthy Volunteers

Dyslipidemias | Hypertriglyceridemia | Familial Hypercholesterolemia

Search clinical trials for: Lipid Metabolism, Inborn Errors

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