Clinical Trials on Amino Acid Metabolism, Inborn Errors in Belgium

Total 177 results

PTC Therapeutics
Genzyme, a Sanofi Company

Terminated

Ataluren for Nonsense Mutation Methylmalonic Acidemia

Amino Acid Metabolism, Inborn Errors

France, United Kingdom, Italy, Belgium, Germany, Switzerland
Nutricia Research

Completed

PANDA: PKU Amino Acid Evaluation (PANDA)

Phenylketonuria (PKU)

United Kingdom, Belgium
Nutricia Research

Completed

SNAP: Study Nutrients in Adult PKU (SNAP)

Phenylketonuria

Spain, Belgium, Denmark
Swedish Orphan Biovitrum

Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 (HT-1)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Sweden
Promethera Therapeutics

Completed

Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

Urea Cycle Disorders

Belgium, Spain, France, Poland
BioMarin Pharmaceutical

Completed

Kuvan® in Phenylketonuria Patients Less Than 4 Years Old (SPARK)

Phenylketonuria

Italy, Turkey, United Kingdom, Germany, Netherlands, Slovakia, Austria, Belgium, Czechia
Promethera Therapeutics

Completed

Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)

Urea Cycle Disorders | Crigler Najjar Syndrome

Belgium
Laboratoires Genévrier

Completed

Evaluation of Non-cultured Epidermal Cellular Grafting vs Hyaluronic Acid for Repigmenting Vitiligo and Piebaldism

Vitiligo | Piebaldism

Belgium, France, Italy
Arcturus Therapeutics, Inc.

Recruiting

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

OTC Deficiency | Ornithine Transcarbamylase Deficiency | OTCD

Spain, Belgium, United Kingdom, Sweden
Swedish Orphan Biovitrum

Completed

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care (OPAL)

Hereditary Tyrosinemia, Type I

Belgium, Denmark, France, Germany, Austria, Czechia, Finland, Hungary, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, United Kingdom
Kaleido Biosciences

Terminated

UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder (UNLOCKED)

Urea Cycle Disorder

Belgium, United States, Spain, United Kingdom, Germany, Switzerland, Turkey
Promethera Therapeutics

Completed

Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) (HEP001)

Urea Cycle Disorders | Crigler Najjar Syndrome

Israel, United Kingdom, Belgium, France, Italy
University of Lausanne
University of Liege

Completed

Fructose Supplementation in Carriers for Hereditary Fructose Intolerance

Hereditary Fructose Intolerance

Belgium
Universitaire Ziekenhuizen KU Leuven

Unknown

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

Lipid Metabolism, Inborn Errors

Belgium
Alexion

Recruiting

Lysosomal Acid Lipase (LAL) Deficiency Registry (ALX-LALD-501)

Wolman Disease | Cholesterol Ester Storage Disease | Lysosomal Acid Lipase Deficiency | Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 | Acid Lipase Deficiency | LIPA Deficiency | LAL-Deficiency

France, Belgium, United States, Spain, Germany, Greece, Israel, Italy, Slovenia, United Kingdom, Brazil, Canada, Denmark, Australia, Croatia, Czechia, Ireland, Mexico, Netherlands, Poland, Portugal, Saudi Arabia
Cellaion SA

Enrolling by invitation

HepaStem Long-Term Safety Registry (PROLONGSTEM)

Urea Cycle Disorder | Crigler-Najjar Syndrome | Acute on Chronic Liver Failure

Belgium, Spain, France, Bulgaria, Poland
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
Maastricht University Medical Center
Annadal stichting

Unknown

Proton Pump Inhibitors in the Prevention of Iron Reaccumulation in Patient With Hereditary Hemochromatosis (He-ppi)

Hemochromatosis

Netherlands, Belgium
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
KU Leuven

Completed

Comparison of the Colonic Metabolism in Patients With Lactose Intolerance and Healthy Controls

Lactose Intolerance

Belgium
Universitaire Ziekenhuizen KU Leuven

Withdrawn

Implications for Quality of Life and Quality of Care in Patients With Hereditary Haemochromatosis

Hereditary Haemochromatosis

Belgium
Cliniques universitaires Saint-Luc- Université...

Recruiting

Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases (BUSARD)

Rare Diseases | Inborn Errors of Metabolism

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
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

Unknown

Renal Tubular Acidosis in Incident Renal Transplant Recipients

Renal Transplant Disorder

Belgium
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
Universitair Ziekenhuis Brussel

Recruiting

Efficacy of a Low FODMAP Diet in the Absence of Lactose Malabsorption in Moderate to Severe ROME IV IBS.

Irritable Bowel Syndrome

Belgium
Novartis Pharmaceuticals

Completed

Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis (HH)

Hereditary Hemochromatosis

Belgium, France, Russian Federation, Romania, Slovakia, Spain, Switzerland
Universitaire Ziekenhuizen KU Leuven

Completed

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

Cystinosis

Belgium
AstraZeneca

Completed

A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia

Homozygous Familial Hypercholesterolemia (HoFH)

Malaysia, Canada, Taiwan, Belgium, Denmark, Israel
AstraZeneca

Completed

An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

Familial Hypercholesterolaemia

United States, Netherlands, Norway, Canada, Belgium
Alnylam Pharmaceuticals

Approved for marketing

Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

Primary Hyperoxaluria

Belgium
Cerenis Therapeutics, SA

Terminated

CER-001 Therapy as a Novel Approach to Treat Genetic Orphan Diseases (TANGO)

Familial Hypoalphalipoproteinemia

France, United States, Belgium, Canada, Israel, Italy, Netherlands
AstraZeneca

Completed

A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia (HYDRA)

Homozygous Familial Hypercholesterolemia (HoFH)

Malaysia, Canada, Israel, Netherlands, Taiwan, Belgium, Denmark
Nestlé

Completed

Althera® Versus Nutramigen / Cow's Milk Intolerance

Allergy

Belgium
OxThera

Terminated

An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria (ePHex-OLE)

Hyperoxaluria, Primary

United Kingdom, Spain, Belgium, United States, Germany, Tunisia
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
OxThera

Completed

A Study to Evaluate the Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria

Primary Hyperoxaluria

France, United Kingdom, Spain, Belgium, United States, Germany, Tunisia
Alexion Pharmaceuticals, Inc.

Not yet recruiting

Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP (MULBERRY)

Hypophosphatasia

United States, Taiwan, Thailand, Vietnam, United Kingdom, Korea, Republic of, Israel, Italy, Australia, Turkey, France, Poland, Germany, Spain, Belgium, China, Canada, Brazil, Argentina, Mexico
Alexion Pharmaceuticals, Inc.

Recruiting

Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa (HICKORY)

Hypophosphatasia

Italy, Spain, Israel, Korea, Republic of, Belgium, Germany, United States, China, France, Taiwan, Austria, Japan, India, Australia, Argentina, Poland, Turkey, Brazil, Canada, Thailand, United Kingdom, Slovakia, Czechia
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
Pfizer's Upjohn has merged with Mylan to form Viatris...

Completed

Atorvastatin Three Year Pediatric Study

Familial Hypercholesterolemia

United States, Spain, Turkey, Canada, Belgium, Germany, Greece, Hungary, Italy, Norway, Poland, Puerto Rico, Russian Federation, Slovakia, Switzerland
Amgen

Completed

Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities (TESLA)

Homozygous Familial Hypercholesterolemia

New Zealand, South Africa, France, United States, Spain, Belgium, Czechia, Italy, Netherlands, Hong Kong, Canada, Lebanon
Amgen

Completed

Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) (HAUSER-OLE)

Familial Hypercholesterolemia

Canada, Italy, Norway, Belgium, United States, Turkey, Australia, Austria, South Africa, Poland, Switzerland, Spain, Portugal, Slovenia, Brazil, Russian Federation, Hungary, United Kingdom, Colombia, Czechia, Netherlands, Greece, Ma...
Amgen

Completed

Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders (TAUSSIG)

Severe Familial Hypercholesterolemia

Italy, Czechia, United States, Israel, New Zealand, South Africa, France, Spain, Australia, Belgium, Canada, Netherlands, Hong Kong, Brazil, United Kingdom, Greece, Japan, Lebanon
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
Amgen

Completed

Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) Disorders (HAUSER-RCT)

Heterozygous Familial Hypercholesterolemia

United States, Canada, Italy, Norway, Belgium, Turkey, Australia, New Zealand, Austria, Czechia, South Africa, Poland, Spain, Switzerland, Netherlands, Taiwan, Finland, Brazil, Portugal, Slovenia, Russian Federation, Hungary, United... and more
Chiesi SA/NV

Enrolling by invitation

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

Nephropathic Cystinosis

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
Kastle Therapeutics, LLC

Completed

A Study of the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol (FOCUS FH)

Hypercholesterolemia | Heterozygous Familial

United States, Italy, Poland, Ukraine, United Kingdom, Germany, Hong Kong, Taiwan, Spain, Argentina, Belgium, Israel, Malaysia, South Africa, Turkey, Netherlands, Korea, Republic of, Canada, New Zealand, Russian Federation, Brazil, Austral... and more

Clinical Trials on Amino Acid Metabolism, Inborn Errors in Belgium

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