Clinical Trials on Autosomal or Sex Linked Recessive Genetic Disease in Belgium

Total 573 results

Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney (ARPKD)

United States, United Kingdom, Belgium, Poland, France, Germany, Italy
Otsuka Pharmaceutical Development & Commercialization...

Recruiting

A Study to See if Tolvaptan Can Delay Dialysis in Infants and Children Who at Enrollment Are 28 Days to Less Than 12 Weeks Old With Autosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney Disease (ARPKD)

United States, Belgium, Spain, Germany, United Kingdom, Poland
Marinus Pharmaceuticals
University of California, Davis; U.S. Army Medical Research and Development...

Completed

Ganaxolone Treatment in Children With Fragile X Syndrome

Fragile x Syndrome

Belgium, United States
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
Andreas L. Serra

Unknown

The Eurocyst Initiative: Building a Network of ADPKD Reference Centers Across Europe

Autosomal Dominant Polycystic Kidney Disease

France, Switzerland, Germany, Netherlands, Belgium, Czech Republic, Italy, Spain, Turkey, United Kingdom
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
Cliniques universitaires Saint-Luc- Université...

Recruiting

Unraveling the Klinefelter's Disease Physiopathology (KLINEFELTER)

Infertility, Male | Klinefelter Syndrome

Belgium
Otsuka Pharmaceutical Europe Ltd

Unknown

A Study Measuring Quality of Life, Treatment Preference and Satisfaction of ADPKD Patients in Europe (ACQUIRE)

Autosomal Dominant Polycystic Kidney Disease

Spain, United Kingdom, Germany, France, Belgium, Switzerland, Austria
Universitaire Ziekenhuizen KU Leuven
KU Leuven

Completed

Angelman & Rett Syndrome: Interaction and Communication

Communication | Parent-Child Relations | Behavior, Child

Belgium
Galapagos NV

Terminated

A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal Dominant Polycystic Kidney Disease

Belgium, Netherlands, Italy, Spain, Czechia, Germany, Poland
Otsuka Pharmaceutical Development & Commercialization...

Completed

Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Germany, Italy, Belgium, United Kingdom
Radboud University Medical Center
Ipsen

Completed

Lanreotide as Treatment of Polycystic Livers (LOCKCYST)

Liver Diseases | Polycystic Kidney, Autosomal Dominant | Hepatomegaly | Polycystic Liver Disease

Netherlands, Belgium
Sanofi

Terminated

Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat (STAGED-PKD-EXT)

Congenital Cystic Kidney Disease

Germany, Spain, United States, Australia, Belgium, Japan, Korea, Republic of, Netherlands
Universitair Ziekenhuis Brussel
Marguerite - Marie Delacroix Foundation

Not yet recruiting

Combining Exome and Transcriptome Data to Unravel the Genetic Basis of the Lissencephalies

Classical Lissencephalies and Subcortical Band Heterotopias

Belgium
Santhera Pharmaceuticals

Completed

Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

Duchenne Muscular Dystrophy

Belgium
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
Cliniques universitaires Saint-Luc- Université...

Completed

Gait Evaluation in Haemophiliac Patients (GAIT_GENERAL)

Hemophilia A | Hemophilia B

Belgium
Santhera Pharmaceuticals

Completed

Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy (DELPHI)

Duchenne Muscular Dystrophy (DMD)

Belgium
Cliniques universitaires Saint-Luc- Université...

Completed

Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy

Haemophilia A | Haemophilia B

Belgium
Laurent Servais
SYSNAV

Recruiting

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Duchenne Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy

Belgium
BioMarin Pharmaceutical

Completed

Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

Belgium, Netherlands, Italy, Sweden
Universiteit Antwerpen
University Hospital, Antwerp; Cliniques universitaires Saint-Luc- Université...

Recruiting

Gaining Insight Into the Complexity of Pain in Patients With Haemophilia

Hemophilia A | Hemophilia B

Belgium
BioMarin Pharmaceutical

Terminated

Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

Netherlands, Belgium, Italy, Sweden
Otsuka Pharmaceutical Development & Commercialization...

Completed

Tolvaptan Extension Study in Participants With ADPKD (TEMPO 4/4)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Australia, Belgium, Germany, Italy, Poland, Romania, United Kingdom, Argentina, Canada, France, Netherlands, Russian Federation
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Tolvaptan Phase 3 Efficacy and Safety Study in Autosomal Dominant Polycystic Kidney Disease (ADPKD) (TEMPO3:4)

Polycystic Kidney Disease, Autosomal Dominant

United States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany
Sarepta Therapeutics, Inc.

Completed

Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

Duchenne Muscular Dystrophy

Belgium, France, Italy, United Kingdom
BioMarin Pharmaceutical

Terminated

Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

Belgium, Netherlands, Italy, Sweden
Radboud University Medical Center
Ipsen

Unknown

Open-Label Extension of LOCKCYST Trial (LOCKCYST-ext)

Liver Diseases | Hepatomegaly | Polycystic Liver Disease | Polycystic Kidney | Autosomal Dominant

Netherlands, Belgium
Sarepta Therapeutics, Inc.

Terminated

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)

Duchenne Muscular Dystrophy

Belgium, France, Italy, United Kingdom
Eloxx Pharmaceuticals, Inc.
SGS Life Sciences, a division of SGS Belgium NV

Completed

Phase 1 Study of ELX-02 in Healthy Adults

Genetic Disease | Nonsense Mutation

Belgium
BioMarin Pharmaceutical

Terminated

A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy

United Kingdom, Italy, Belgium, Netherlands, France
Eloxx Pharmaceuticals, Inc.
SGS Life Sciences, a division of SGS Belgium NV

Completed

Phase 1 Study of ELX-02 in Healthy Adult Subjects

Genetic Disease | Nonsense Mutation

Belgium
Genzyme, a Sanofi Company

Terminated

A Medical Research Study Designed to Determine if Venglustat Can be a Future Treatment for ADPKD Patients (STAGED-PKD)

Polycystic Kidney, Autosomal Dominant

Belgium, Germany, Czechia, United States, Italy, Poland, Argentina, Australia, Austria, Canada, China, Denmark, France, Israel, Japan, Korea, Republic of, Netherlands, Portugal, Romania, Spain, Taiwan, Turkey, United Kingdom
BioMarin Pharmaceutical

Terminated

Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Belgium, Netherlands, France, United Kingdom, Italy
Otsuka Pharmaceutical Development & Commercialization...
Otsuka Pharmaceutical Co., Ltd.

Completed

Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease (OVERTURE)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States, Argentina, Australia, Belgium, Brazil, Canada, Czech Republic, France, Germany, Italy, Japan, Netherlands, Norway, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
University Hospital, Ghent

Completed

Genetic Screening and Assisted Oocyte Activation in Couples With Diminished/Aberrant Embryonic Development. (AOA)

Genetic Disease | Embryo | Embryo Disorder

Belgium
Otsuka Pharmaceutical Development & Commercialization...

Completed

Efficacy and Safety of Tolvaptan in Subjects With Chronic Kidney Disease Between Late Stage 2 to Early Stage 4 Due to Autosomal Dominant Polycystic Kidney Disease

Chronic Kidney Disease | Autosomal Dominant Polycystic Kidney Disease

Australia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania
Queen Fabiola Children's University Hospital
Center of Human Genetics - ULB in Brussels; Interuniversity Institute of Bioinformatics...

Unknown

Method of Genetic Analysis in Genodermatoses (GENODERM)

Genodermatosis | Rare Genetic Disease With Cutaneous Expression

Belgium
Wave Life Sciences Ltd.

Completed

Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy

Belgium, United Kingdom, France, United States, Italy, Canada, Netherlands
University Hospital, Ghent

Recruiting

Uterus Transplantation From a Multi-organ Donor

Infertility, Female | Mayer Rokitansky Kuster Hauser Syndrome

Belgium
Denali Therapeutics Inc.

Enrolling by invitation

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Mucopolysaccharidosis II

United States, Canada, Belgium, Czechia, Netherlands
Reata, a wholly owned subsidiary of Biogen

Terminated

A Trial of Bardoxolone Methyl in Patients With ADPKD - FALCON (FALCON)

Autosomal Dominant Polycystic Kidney | ADPKD

United States, Spain, United Kingdom, Australia, France, Japan, Germany, Belgium, Czechia, Italy
Swedish Orphan Biovitrum

Completed

A Non-interventional Retrospective Study of the Current Treatment Practice in European Haemophilia Care

Haemophilia A | Haemophilia B

France, Germany, Italy, Spain, Sweden, Belgium, 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
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
Otsuka Pharmaceutical Development & Commercialization...

Completed

Long Term Safety of Immediate-release Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant

Belgium, Germany, Italy, Spain, Sweden, United States, Canada, Australia, Netherlands, Czechia, Poland, Denmark, Israel, Hungary, Russian Federation, South Africa, United Kingdom, Romania, Argentina, Norway
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
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

United States, Canada, Israel, United Kingdom, Spain, Denmark, France, Belgium, Italy, Netherlands, Switzerland
Janssen Research & Development, LLC
Janssen Research & Development, LLC

Active, not recruiting

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

X-Linked Retinitis Pigmentosa

Belgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
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

Clinical Trials on Autosomal or Sex Linked Recessive Genetic Disease in Belgium

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ethiopia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations