Clinical Trials on Genetic Diseases, Inborn in Belgium

Total 11175 results

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
Bayer

Active, not recruiting

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study) (LIFE-ACTIVE)

Hemophilia A

Canada, Spain, United States, Belgium, Italy, Taiwan, Slovenia
University Hospital, Antwerp

Enrolling by invitation

Viral Infections and Airway Microbiome in Young Children With Cystic Fibrosis

Respiratory Viral Infection | Respiratory Morbidity | Cystic Fibrosis in Children | Respiratory Bacterial Infection | Inflammation Lungs

Belgium
University Hospital, Antwerp
Belgium Health Care Knowledge Centre

Recruiting

The Belgian Endothelial Surgical Transplant of the Cornea (BESTCornea)

Corneal Edema | Fuchs' Endothelial Dystrophy | Bullous Keratopathy | Pseudophakic Bullous Keratopathy | Corneal Endothelial Disorder

Belgium
ProQR Therapeutics

Terminated

Extension Study to Study PQ-110-001 (NCT03140969) (INSIGHT)

Eye Diseases | Neurologic Manifestations | Eye Diseases, Hereditary | Sensation Disorders | Vision Disorders | Blindness | Leber Congenital Amaurosis | Leber Congenital Amaurosis 10 | Retinal Disease | Eye Disorders Congenital

United States, 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
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
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
University Hospital, Antwerp
University Hospital, Ghent

Recruiting

Genome-wide Epistasis for Cardiovascular Severity in Marfan Study (GEMS)

Marfan Syndrome | FBN1 Mutation

Belgium
KU Leuven

Unknown

Microarray CGH Analysis of Circulating Tumoral Plasma DNA in NF1 Patients With MPNSTs

Neurofibromatosis Type 1

Belgium
Orphalan
Ergomed

Recruiting

International Wilson's Disease Patient Registry (iWilson Registry)

Wilson's Disease

Spain, United Kingdom, Belgium, Germany, Poland, France, Saudi Arabia
University Hospital, Ghent

Terminated

Investigating the Cryopreserved Blastocyst's ImplantatiOn Potential After Genetic Screening (BIOPS)

Infertility | Preimplantation Genetic Screening

Belgium
University Hospital, Toulouse
Epidemiological and Clinical Research Information Network; International Clinical...

Completed

Oxytocin Treatment in Neonates and Infants With Prader-Willi Syndrome (OTBB3)

Prader-Willi Syndrome

France, Belgium, Germany
Idorsia Pharmaceuticals Ltd.

Completed

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease (MODIFY)

Fabry Disease

United States, Spain, Belgium, Australia, Austria, Canada, Germany, Ireland, Italy, Netherlands, Norway, Poland, Switzerland, United Kingdom
ApoPharma

Completed

A Study Investigating the Long-term Safety and Efficacy of Deferiprone in Patients With Friedreich's Ataxia

Friedreich's Ataxia

Belgium, France, Italy, Spain
Alnylam Pharmaceuticals

Active, not recruiting

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1 (ILLUMINATE-C)

Primary Hyperoxaluria Type 1 | Primary Hyperoxaluria

United States, Italy, France, Lebanon, Belgium, Netherlands, Israel, United Arab Emirates, Australia, Jordan, Turkey
Almirall, S.A.

Completed

A Study of Lebrikizumab in Combination With Topical Corticosteroids in Participants Having Atopic Dermatitis (AD) That Are Not Adequately Controlled or Non-eligible for Cyclosporine

Eczema | Dermatitis, Atopic

Austria, Belgium, France, Germany, Netherlands, Poland, Spain, United Kingdom
Italfarmaco

Recruiting

Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy (ULYSSES)

Duchenne Muscular Dystrophy

Canada, Italy, France, Belgium, Germany, Netherlands, United Kingdom
KU Leuven

Unknown

Analysis of Peripheral Nerve Sheath Tumors (PNSTs) in Neurofibromatosis Type 1 (NF1) Patients

Neurofibromatosis Type 1

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
Universitaire Ziekenhuizen KU Leuven

Unknown

Ophthalmological Screening and Follow-up of Optic Pathway Gliomas in Children With Neurofibromatosis Type 1.

Neurofibromatosis Type 1

Belgium
GlaxoSmithKline

Completed

Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy

Muscular Dystrophies

Germany, Belgium, Turkey, Netherlands, Spain, Israel, Australia, France, United Kingdom
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
Astellas Pharma Inc

Completed

Long-term Safety of Protopic in Atopic Eczema

Dermatitis, Atopic | Eczema, Atopic

France, Germany, United Kingdom, Spain, Belgium, Netherlands, Finland, Poland, Ireland, Hungary, Denmark, Latvia
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...
University Hospital, Antwerp
Kom Op Tegen Kanker; Stichting tegen Kanker

Recruiting

First-in-human Interleukin-15-transpresenting Wilms' Tumor Protein 1-targeting Autologous Dendritic Cell Vaccination in Cancer Patients (IL15-TransDC)

Esophageal Cancer | Ovarian Cancer | Pancreas Cancer | Liver Cancer

Belgium
Minoryx Therapeutics, S.L.

Completed

A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients (FRAMES)

Friedreich Ataxia

Spain, Belgium, France, Germany
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
BioMarin Pharmaceutical

Terminated

A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy

United States, Netherlands, Belgium, Germany, Argentina, Brazil, France, Italy, Sweden, Turkey
Novartis Pharmaceuticals

Completed

Canakinumab in the Treatment of Acute Gout Flares and Prevention of New Flares in Patients Unable to Use Non-steroidal Anti-inflammatory Drugs (NSAIDs) and/or Colchicine Including a 12 Weeks Extension and an Open-label 48 Weeks Extension Study (β-RELIEVED)

Acute Gout

Colombia, Switzerland, Germany, Russian Federation, Singapore, Australia, Belgium, Canada, Estonia, Guatemala, Latvia, Lithuania, Mexico, Norway, Poland, Sweden, Ukraine
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
Cellaion SA

Terminated

HepaStem Long-Term Safety Registry (PROLONGSTEM)

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

Belgium, Spain, France, Bulgaria, Poland
Immunovant Sciences GmbH

Recruiting

Extension Study to Assess Batoclimab in Participants With Thyroid Eye Disease

Thyroid Eye Disease

United States, Hungary, Spain, Latvia, Belgium
Regeneron Pharmaceuticals
Sanofi

Completed

A Study to Assess the Efficacy and Safety of Dupilumab in Participants With Severe Atopic Dermatitis (AD) That Are Not Controlled With Oral Cyclosporine A (CSA) or for Those Who Cannot Take Oral CSA Because it is Not Medically Advisable

Atopic Dermatitis

Germany, Poland, Belgium, Netherlands, Russian Federation, Ireland, United Kingdom, Slovakia, Austria, Spain
Astellas Pharma Inc

Completed

Equivalence of the Response to Vaccination of Tacrolimus Ointment to a Steroid Ointment Regimen in Children With Atopic Dermatitis

Atopic Dermatitis

Australia, Belgium, Poland, Hungary, Portugal, Germany, Iceland, Malta
Kamada, Ltd.
Syneos Health

Recruiting

Evaluate Efficacy and Safety of "Kamada-AAT for Inhalation" in Patients With AATD (InnovAATe)

Alpha 1-Antitrypsin Deficiency

United Kingdom, Sweden, Ireland, Netherlands, Belgium, Finland
Ardea Biosciences, Inc.

Completed

Open-Label Lesinurad Monotherapy Extension Study in Gout

Gout

United States, Canada, Belgium, Australia, Germany, New Zealand, South Africa
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
Novartis Pharmaceuticals

Completed

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Anemia | Hemosiderosis

United States, Germany, Belgium, United Kingdom, Italy, Canada, France
dr. Laura C. G. de Graaff-Herder

Not yet recruiting

Growing up With the Young Endocrine Support System (YESS!) (YESS)

Congenital Adrenal Hyperplasia | Hypogonadotropic Hypogonadism | Growth Hormone Deficiency | Turner Syndrome | Addison's Disease | Klinefelter Syndrome | Combined Pituitary Hormone Deficiency | Androgen Insensitivity Syndrome | Thyroid Dysgenesis

Spain, United Kingdom, Belgium, Netherlands
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
Insmed Incorporated

Completed

Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)

Cystic Fibrosis

Poland, Ukraine, Serbia, Belgium, Hungary, Slovakia, North Macedonia
LEO Pharma

Completed

Tralokinumab in Combination With Topical Corticosteroids in Subjects With Severe Atopic Dermatitis - ECZTRA 7 (ECZTRA 7)

Atopic Dermatitis

Belgium, France, Germany, Poland, United Kingdom, Czechia, Spain
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
Belite Bio, Inc

Active, not recruiting

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 (DRAGON)

Stargardt Disease 1

United States, Australia, Belgium, China, France, Germany, Hong Kong, Netherlands, Switzerland, Taiwan, United Kingdom
Universitair Ziekenhuis Brussel

Unknown

Characterizing Sleep Disorders in Children and Adults With Tuberous Sclerosis Complex (TSC)

Sleep Disorder

Belgium
Wave Life Sciences Ltd.

Terminated

Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)

Duchenne Muscular Dystrophy

United States, Belgium, United Kingdom, Czechia, Canada, France, Italy, Sweden
Universitaire Ziekenhuizen KU Leuven

Unknown

Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis (NEFIMAR)

Magnetic Resonance Imaging | Neurofibromatosis 1 | Whole Body Imaging | Diffusion Magnetic Resonance Imaging | Peripheral Nerve Sheath Tumors, Malignant

Belgium
Insmed Incorporated

Completed

Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase

Cystic Fibrosis

Poland, Ukraine, Serbia, Belgium, Hungary, Slovakia, North Macedonia
Novartis Pharmaceuticals

Completed

Long Term Study of Canakinumab (ACZ885) in Patients With Gout

Gouty Arthritis

Colombia, Spain, Taiwan, Russian Federation, Guatemala, Belgium, United States, Argentina, Czechia, Germany, Hungary, Poland, Portugal, Singapore, Slovakia, South Africa, Turkey, United Kingdom

Clinical Trials on Genetic Diseases, Inborn in Belgium

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