Clinical Trials on Inherited Metabolic Storage Diseases in Belgium

Total 1256 results

Cliniques universitaires Saint-Luc- Université...

Unknown

Safety, Efficacy Evaluation of Empagliflozin Administration for Neutropenia in Glycogenosis Type 1b and G6PC3 Deficiency (GLYCO-1B)

Glucose 6 Phosphatase Deficiency | Glycogen Storage Disease Type I

Belgium
Universitaire Ziekenhuizen KU Leuven

Completed

The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients

Cystinosis

Belgium
Universiteit Antwerpen

Completed

The Effect of a Red Rice and Olive Extract Nutrition Supplement on Cholesterol Levels in Patients With Metabolic Syndrome

Hypercholesterolaemia

Belgium
Sanofi

Recruiting

A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

Glycogen Storage Disease Type II

Belgium, United States, Italy, Spain, Taiwan, United Kingdom, Netherlands, France, Germany
Sanofi

Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

Glycogen Storage Disease Type II

Belgium, United States, China, Germany, Italy, Netherlands, Spain, Taiwan, United Kingdom
Genzyme, a Sanofi Company

Completed

Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.

Pompe Disease | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Genzyme, a Sanofi Company

Completed

Avalglucosidase Alfa Extension Study (NEO-EXT)

Glycogen Storage Disease Type II Pompe Disease

United States, Belgium, Denmark, France, Germany, Netherlands, United Kingdom
Genzyme, a Sanofi Company

Completed

Alglucosidase Alfa Pompe Safety Sub-Registry

Pompe Disease

Belgium, United States, Czechia, Germany, Italy, Taiwan
BioMarin Pharmaceutical

Terminated

BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)

Late-onset Pompe Disease

United States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands
Recordati Rare Diseases

Completed

Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) (SCOB2)

Cystinosis

Belgium, France, United Kingdom, Germany, Italy
Amicus Therapeutics

Terminated

STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT

Late-onset Pompe Disease

United States, Canada, Australia, Belgium
Zymenex A/S
European Commission

Completed

Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis

Alpha-Mannosidosis

Belgium, Denmark, Spain, United Kingdom
Genzyme, a Sanofi Company

Recruiting

Pompe Pregnancy Sub-Registry

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase Deficiency

Croatia, United States, Italy, Belgium, Czechia
Shire

Completed

Sophisticated Assessment of Disease Burden in Patients With Fabry Disease (SOPHIA)

Fabry Disease

Finland, Sweden, Germany, Belgium, Czechia, Denmark
Genzyme, a Sanofi Company

Completed

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

Glycogen Storage Disease Type II;Pompe's Disease

Czechia, Taiwan, Belgium, Germany, Korea, Republic of, United States, Argentina, Australia, Austria, Brazil, Canada, Denmark, France, Hungary, Italy, Japan, Mexico, Netherlands, Poland, Portugal, Russian Federation, Spain, Switzerland, Turk... and more
Alexion Pharmaceuticals

Completed

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

Spain, Germany, Italy, United States, Croatia, Canada, Russian Federation, Denmark, United Kingdom, Belgium, Mexico, Australia, Netherlands, Brazil, Turkey
CENTOGENE GmbH Rostock

Completed

The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease (SWITCH)

Fabry Disease | Anderson-Fabry Disease | Fabry´s Disease

Argentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
Amicus Therapeutics

Active, not recruiting

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more
Amicus Therapeutics

Completed

A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PROPEL)

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
Amicus Therapeutics

Completed

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

Fabry Disease

Canada, Belgium, United States, Australia, Netherlands
University Hospital, Ghent

Completed

Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy

Left Ventricular Hypertrophy

Belgium
Imelda Hospital, Bonheiden
Université Catholique de Louvain

Recruiting

Metabolic Profiling of Hematopoietic Stem Cells in Clonal Hematopoiesis (CHIP)

Aging | Hip Fractures | Osteoporosis | Osteoarthritis, Hip | Clonal Hematopoiesis of Indeterminate Potential

Belgium
Genzyme, a Sanofi Company

Recruiting

Pompe Disease Registry Protocol

Glycogen Storage Disease Type II | Pompe Disease

Hungary, Croatia, Serbia, United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, India, Indonesia, Israel, Italy, Japan, Jordan, Korea, Republic of, Kuwait and more
Chiesi Farmaceutici S.p.A.
Iqvia Pty Ltd; Protalix

Completed

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion (PEOPLE)

Fabry Disease

United States, Belgium, Denmark, Italy, United Kingdom
Hasselt University

Recruiting

Preventing Exercise Resistance With Sedentary Interruptions (PERSI)

Exercise | Sedentary Behavior | Lipid Metabolism Disorder

Belgium
Patrice D. Cani
Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Completed

Evaluation of the Effects Associated With the Administration of Akkermansia Muciniphila on Parameters of Metabolic Syndrome (Microbes4U)

Glucose Metabolism Disorders | Obesity | Dyslipidemias | Metabolic Syndrome x

Belgium
Genzyme, a Sanofi Company

Completed

A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Sphingomyelin Lipidosis

United States, Belgium, Brazil, France, Germany, Italy, United Kingdom
OptiScan Biomedical Corporation
Erasme University Hospital

Completed

OptiScanner Versus Standard Blood Glucose Monitoring

Hyperglycemia

Belgium
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks (BRIGHT)

Fabry Disease

United States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
University Hospital, Ghent
Astellas Pharma Inc

Completed

The Impact of Magnesium Supplementation on Insulin Resistance and Secretion in Renal Transplant Recipients

Glucose Metabolism | Renal Transplantation

Belgium
Liaquat University of Medical & Health Sciences
KU Leuven

Completed

The Antihypercholestrolemic Effect of a Combined Nutritional Supplementation of Standardized Extracts of Amla, Walnut, Red Yeast Rice and Olive

Hyperlipidemia

Belgium
Université Catholique de Louvain
Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Completed

Lipid-lowering Therapy Individualization

Hypercholesterolemia

Belgium
Pfizer

Terminated

Single Dose Study of PF-06815345 in Healthy Subjects

Hypercholesterolemia

Belgium
Horizon Pharma USA, Inc.

Completed

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Cystinosis

France, United States, United Kingdom, Belgium, Italy, Netherlands
Pfizer
Halozyme Therapeutics

Terminated

Pharmacokinetic and Pharmacodynamic Study of Bococizumab Alone and When Combined With Recombinant Human Hyaluronidase

Healthy | Hypercholesterolemia

Belgium
University Ghent

Completed

Validation of a Diagnostic Tool for Postprandial Hypoglycaemia in Patients After Roux en Y Gastric Bypass Surgery (EDS)

Post Prandial Hypoglycemia

Belgium
Cliniques universitaires Saint-Luc- Université...

Unknown

Non-invasive Evaluation of Hepatic Fibrosis in Patients With the Metabolic Syndrome

Metabolic Syndrome

Belgium
Shire

Terminated

Natural History Study of Children With Metachromatic Leukodystrophy

Metabolic Diseases | Brain Diseases | Central Nervous System Diseases | Nervous System Diseases | Demyelinating Diseases | Genetic Diseases, Inborn | Metabolism, Inborn Errors | Lysosomal Storage Diseases | Lipid Metabolism Disorders | Sphingolipidoses | Hereditary Central Nervous System Demyelinating Diseases and other conditions

United States, Denmark, France, Argentina, Belgium, Brazil, Canada, Germany, Japan, Turkey
Onze Lieve Vrouw Hospital

Completed

Effect on Vascular Calcification of Replacing Warfarin by Rivaroxaban With or Without VitK2 in Hemodialysis Patients

Vascular Calcification

Belgium
Idorsia Pharmaceuticals Ltd.

Completed

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 (RETRIEVE)

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2

United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
Genzyme, a Sanofi Company

Terminated

A Study of Renal Function in Treatment-naïve, Young Male Patients With Fabry Disease

Fabry Disease

Austria, Brazil, France, United States, Argentina, Belgium, Canada, Finland, Hungary, Norway, Poland, Spain, Taiwan, United Kingdom
Amicus Therapeutics

Completed

Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease

Fabry Disease

France, Austria, United States, Belgium, Japan, United Kingdom, Denmark, Brazil, Australia, Italy
Chiesi SA/NV

Enrolling by invitation

Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients (PROREAL)

Nephropathic Cystinosis

Belgium
University of Lausanne
University of Liege

Completed

Fructose Supplementation in Carriers for Hereditary Fructose Intolerance

Hereditary Fructose Intolerance

Belgium
Chiesi Farmaceutici S.p.A.

Active, not recruiting

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (Bright51)

Fabry Disease

Belgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
Khondrion BV
Drug Research Unit Ghent, Belgium

Completed

A Dose-escalating Clinical Trial With KH176

Mitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Belgium
Amicus Therapeutics

Terminated

Open-Label Phase 3 Long-Term Safety Study of Migalastat (AT1001-041)

Fabry Disease

Australia, United States, Italy, Spain, Belgium, Turkey, Brazil, Argentina, United Kingdom, Denmark, France, Canada, Egypt
Universitaire Ziekenhuizen KU Leuven

Unknown

Anti-oxLDL IgM Antibodies as a Novel Therapy for Metabolic Lipid Diseases

Lipid Metabolism, Inborn Errors

Belgium
University of Liege

Recruiting

Bone Density and Vascular Calcifications Evolution After Renal Transplant

Bone Loss | Kidney Transplant; Complications

Belgium
Amicus Therapeutics

Completed

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Fabry Disease

United States, Austria, United Kingdom, Argentina, Australia, Belgium, Brazil, Canada, Denmark, Egypt, France, Italy, Japan, Spain, Turkey

Clinical Trials on Inherited Metabolic Storage Diseases in Belgium

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