Clinical Trials on Phosphomannomutase 2 Congenital Disorder of Glycosylation in Belgium

Total 25 results

Pierre Wauthy

Completed

Development of a Quality of Life Assessment Tool for Adult Patients With a Congenital Heart Disease, Having Had Surgery During Childhood

Congenital Heart Disease

Belgium
Pierre Wauthy

Completed

Long-term Quality of Life of Patients With a Congenital Heart Disease, Treated by Surgery in Adulthood at the Brugmann Hospital.

Congenital Heart Disease

Belgium
Universitair Ziekenhuis Brussel

Unknown

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

Sleep Disorder

Belgium
University Hospital, Ghent

Recruiting

Uterus Transplantation From a Multi-organ Donor

Infertility, Female | Mayer Rokitansky Kuster Hauser Syndrome

Belgium
Cliniques universitaires Saint-Luc- Université...

Recruiting

Unraveling the Klinefelter's Disease Physiopathology (KLINEFELTER)

Infertility, Male | Klinefelter Syndrome

Belgium
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
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
Universiteit Antwerpen
University Hospital, Antwerp

Recruiting

Gait in Rare Diseases (GAGA)

Tuberous Sclerosis | STXBP1 Encephalopathy With Epilepsy

Belgium
University Ghent

Terminated

Patient-based Care Versus Standard Care for Patients With hEDS/HSD and Multidirectional Shoulder Instability

Hypermobile Ehlers-Danlos Syndrome | Hypermobility Syndrome | Multidirectional Subluxation of Shoulder

Belgium
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Sergiusz Jozwiak
Medical University of Vienna; Charite University, Berlin, Germany; Institut National... and other collaborators

Unknown

Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex (EPISTOP)

Epilepsy | Tuberous Sclerosis Complex | TSC

Poland, Austria, Belgium, Czech Republic, France, Germany, Italy, Netherlands
Ipsen

Completed

Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq (OPTIMA)

Renal Insufficiency, Chronic | Pituitary Diseases | Dwarfism | Turner Syndrome

France, Germany, Finland, Spain, Belgium, Czechia, Denmark, Greece, Italy, Romania, Russian Federation, Slovakia, Ukraine, United Kingdom
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
University Medical Center Groningen

Not yet recruiting

HYDROchlorothiazide to PROTECT Polycystic Kidney Disease Patients and Improve Their Quality of Life (HYDRO-PROTECT)

ADPKD

Netherlands, United Kingdom, Belgium, Germany, France, Spain
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
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Congenital Adrenal Hyperplasia

United States, United Kingdom, France, Greece, Italy, Spain, Belgium, Canada, Germany, Poland
Novo Nordisk A/S

Completed

Observational Prospective Study on Patients Treated With Norditropin®

Chronic Kidney Disease | Turner Syndrome | Noonan Syndrome | Chronic Renal Insufficiency | Growth Hormone Disorder | Growth Hormone Deficiency in Children | Adult Growth Hormone Deficiency | Genetic Disorder | Foetal Growth Problem | Small for Gestational Age

Israel, Saudi Arabia, Spain, Argentina, Russian Federation, Germany, Netherlands, Italy, Belgium, United Kingdom, Slovenia, Ireland, Sweden, Denmark, Hungary, Finland, Norway, France, Czechia, Lithuania, Luxembourg, Serbia, Switzerland
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

Congenital Adrenal Hyperplasia

United States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
Novo Nordisk A/S

Recruiting

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow (REAL 8)

SGA, Turner Syndrome, Noonan Syndrome, ISS

Korea, Republic of, United States, Ireland, United Kingdom, Finland, France, Netherlands, Japan, Thailand, Belgium, China, Israel, Brazil, India, Portugal, Austria, Bulgaria, Canada, Croatia, Germany, Greece, Malaysia, Poland, Saudi Arabia and more
Novartis Pharmaceuticals

Active, not recruiting

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

Tuberous Sclerosis Complex

United States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
Novartis Pharmaceuticals

Completed

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1) (EXIST-1)

Tuberous Sclerosis | Subependymal Giant Cell Astrocytoma

United States, Belgium, Canada, Germany, Italy, Russian Federation, Netherlands, Poland, Australia, United Kingdom
Novartis Pharmaceuticals

Completed

A Placebo-controlled Study of Efficacy & Safety of 2 Trough-ranges of Everolimus as Adjunctive Therapy in Patients With Tuberous Sclerosis Complex (TSC) & Refractory Partial-onset Seizures (EXIST-3)

Tuberous Sclerosis Complex-associated Refractory Seizures

United States, Colombia, Belgium, France, Japan, Taiwan, Turkey, Spain, Thailand, Korea, Republic of, Australia, Hungary, United Kingdom, Canada, Norway, Germany, Italy, Russian Federation, Denmark, Argentina, Netherlands, Greece, P... and more
Newcastle University
Assistance Publique - Hôpitaux de Paris; Medical University of Vienna; University... and other collaborators

Recruiting

The European NAFLD Registry

Cardiovascular Diseases | Hypertension | Obesity | Hepatocellular Carcinoma | Type 2 Diabetes | NAFLD | Fibrosis, Liver | NASH - Nonalcoholic Steatohepatitis | Dyslipidaemia | NASH | Steatosis of Liver | Other Associated Comorbidities

Netherlands, Germany, United Kingdom, Finland, Belgium, Greece, Italy, Sweden, Portugal, Spain, France, Switzerland
Tesaro, Inc.
European Organisation for Research and Treatment of Cancer - EORTC; Breast... and other collaborators

Terminated

A Phase III Trial of Niraparib Versus Physician's Choice in HER2 Negative, Germline BRCA Mutation-positive Breast Cancer Patients (BRAVO)

Ovarian Neoplasms | BRCA1 Gene Mutation | BRCA2 Gene Mutation | Neoplasms, Breast | Human Epidermal Growth Factor 2 Negative Carcinoma of Breast | Carcinoma of Breast

United States, Spain, Belgium, Israel, United Kingdom, Italy, Poland, Canada, Hungary, Netherlands, France, Portugal, Greece, Iceland
Scholar Rock, Inc.

Active, not recruiting

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (SAPPHIRE)

Neuromuscular Diseases | Neuromuscular Manifestations | Atrophy | Muscular Atrophy | Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Spinal Muscular Atrophy Type 3 | SMA | Spinal Muscular Atrophy Type 2 | Anti-myostatin

United States, Germany, Belgium, Spain, United Kingdom, Netherlands, France, Italy, Poland

Clinical Trials on Phosphomannomutase 2 Congenital Disorder of Glycosylation in Belgium

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Türkiye

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations