Clinical Trials on Pompe Disease Infantile-Onset in Netherlands

Total 37 results

Genzyme, a Sanofi Company

Completed

A Study of rhGAA in Patients With Late-Onset Pompe Disease

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

Netherlands
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
Spark Therapeutics

Completed

Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease

Lysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Italy, United Kingdom, Netherlands, France, Germany
Genzyme, a Sanofi Company

Completed

Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

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

United States, Netherlands, United Kingdom, Germany
Genzyme, a Sanofi Company

Completed

A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease

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

United States, France, Netherlands
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
Spark Therapeutics

Active, not recruiting

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

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

Completed

Late-Onset Treatment Study Extension Protocol

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

United States, France, Canada, Netherlands, Australia
Jip Groen
InBiome

Recruiting

Molecular Culture for the Diagnosis of Neonatal Sepsis

Microbial Colonization | Neonatal Infection | Neonatal Sepsis, Early-Onset | Microbial Disease | Clinical Sepsis | Culture Negative Neonatal Sepsis | Neonatal Sepsis, Late-Onset | Culture Positive Neonatal Sepsis

Netherlands
prof. dr. Frans B. Plötz
Dutch Society of Pediatrics; Zorgevaluatie Nederland; Care4Neo; everywhereIM

Recruiting

Safely Reduce Newborn Antibiotic Exposure With the Early-onset Sepsis Calculator

EOS | Early-Onset Sepsis, Neonatal

Netherlands
Alnylam Pharmaceuticals

Recruiting

A Study to Evaluate the Safety and Tolerability of ALN-APP in Patients With EOAD

Early-Onset Alzheimer Disease

United Kingdom, United States, Netherlands, Canada
Groningen Research Institute for Asthma and COPD
Numico Research Wageningen, the Netherlands

Completed

Double-Blind, Parallel, Randomised Study to Investigate the Effect of Oral Probiotics in Infants With Atopic Dermatitis

Atopic Dermatitis | Atopic Eczema | Infantile Eczema

Netherlands
HagaZiekenhuis

Recruiting

Peri-Operative Magnesium Infusion to Prevent Atrial Fibrillation Evaluated. (POMPAE)

Atrial Fibrillation New Onset

Netherlands
University Medical Center Groningen

Unknown

An Integrative Genomic Approach to Solve tHe Puzzle of sevERe earLy-Onset COPD (SHERLOCk)

Chronic Obstructive Pulmonary Disease | COPD | Severe Early-Onset COPD | SEO-COPD

Netherlands
UMC Utrecht
Apifix

Withdrawn

Limited-efficacy Testing of Spring Distraction System (SDS) and Unilateral One-way Rod (MID-C) for Early Onset Scoliosis (UniPOWR)

Idiopathic Scoliosis | Early-Onset Scoliosis Deformity of Spine (Disorder)

Netherlands
Gerdien Tramper

Completed

General Risk Factors and Inflammatory Determinants in Older Patients With Asthma (GRANDMA)

Asthma | Childhood Asthma | Late-Onset Asthma

Netherlands
Nij Smellinghe Hosptial

Completed

Study the Usefulness of Bio-impedance Spectroscopy in the Early Assessment of Breast Cancer Related Lymphoedema

Lymphedema | Breast Cancer Stage II | Early Disease Onset

Netherlands
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
Erasmus Medical Center

Recruiting

Pentoxifylline Dose Optimization in Neonatal Sepsis

Neonatal Late Onset Sepsis

Netherlands, Poland
Swedish Orphan Biovitrum

Recruiting

Assessment of Macrophage Activation syndromE in STill's Disease (AMETHYST)

Still's Disease, Adult-Onset | Macrophage Activation Syndrome | Stills Disease, Juvenile-Onset

United States, Canada, France, Germany, Italy, Netherlands, Spain
Erasmus Medical Center

Terminated

Web-based Attention Bias Modification Treatment for Childhood Anxiety Disorders (ATTENTIO)

Generalized Anxiety Disorder | Social Anxiety Disorder | Specific Phobia | Anxiety Disorder | Anxiety Disorder of Childhood | Separation Anxiety Disorder of Childhood, Early Onset

Netherlands
St George's, University of London
Assistance Publique - Hôpitaux de Paris; MRC/UVRI and LSHTM Uganda Research... and other collaborators

Recruiting

Serocorrelate of Protection Against GBS (PREPARE WP3)

Group B Streptococcus Carrier in Childbirth | Group B Streptococcal Infection, Late-Onset | Group B Streptococcal Infection, Early-Onset | Group B Streptococcus Neonatal Sepsis | Group B Strep Infection

France, Italy, Malawi, Netherlands, Uganda, United Kingdom
Vigil Neuroscience, Inc.

Active, not recruiting

A Study of VGL101 in Patients With Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

ALSP

United States, France, Germany, Netherlands, United Kingdom
Vigil Neuroscience, Inc.

Recruiting

Natural History Study in Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia (ALSP)

ALSP

United States, Canada, Germany, Netherlands, United Kingdom, Brazil
Radboud University Medical Center

Recruiting

Societal Merit of Intervention on Hearing Loss Evaluation (SMILE)

Hearing Loss, Sensorineural | Hearing Loss, Bilateral | Hearing Loss, Adult-Onset | Hearing Loss, Bilateral Sensorineural, Progressive

Netherlands
Ataxia Study Group
German Center for Neurodegenerative Diseases (DZNE)

Recruiting

Sporadic Degenerative Ataxia With Adult Onset: Natural History Study (SPORTAX-NHS)

Late Onset Sporadic Cerebellar Ataxia

Norway, Austria, Germany, Netherlands, Italy
Eisai Inc.

No longer available

An Extended Access Program for Perampanel

Partial Onset Seizures

Italy, Spain, Belgium, Israel, Poland, Taiwan, Czechia, Finland, Thailand, Malaysia, Argentina, Chile, Canada, Hungary, Australia, Lithuania, Latvia, Estonia, Netherlands, Greece, Hong Kong
Amicus Therapeutics

Active, not recruiting

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Pompe Disease

United States, Netherlands, United Kingdom, Australia, Germany, New Zealand
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
PRInnovation GmbH
Julius Clinical; Federal Agency for Disruptive Innovation - SPRIN-D; Priavoid

Recruiting

Study to Assess Safety and Efficacy of PRI-002 in Patients With MCI to Mild Dementia Due to Alzheimer's Disease (AD) (PRImus-AD)

Mild Cognitive Impairment Due to Alzheimer's Disease | Alzheimer's Disease, Early Onset

Germany, Spain, Italy, Czechia, Netherlands, Poland
Bristol-Myers Squibb

Recruiting

Study to Evaluate the Efficacy, Safety, and Tolerability of an Anti-MTBR Tau Monoclonal Antibody (BMS-986446) in Participants With Early Alzheimer's Disease (TargetTau-1)

Alzheimer Disease, Early Onset

United States, Australia, Belgium, Canada, Denmark, France, Japan, Korea, Republic of, Netherlands, Poland, Singapore, Spain, Sweden, United Kingdom
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
Janssen Research & Development, LLC

Completed

A Study to Characterize the Clinical Course of Pregnant Women and Children at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn

Erythroblastosis, Fetal | Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN)

United States, Canada, Netherlands, Belgium, Australia, Germany, Spain, Sweden, United Kingdom
Novartis Pharmaceuticals

Completed

Study of Efficacy and Safety of Dabrafenib in Combination With Trametinib in Pediatric Patients With BRAF V600 Mutation Positive LGG or Relapsed or Refractory HGG Tumors

Glioblastoma | Astrocytoma | Anaplastic Astrocytoma | Anaplastic Ganglioglioma | Anaplastic Pleomorphic Xanthoastrocytoma | Ganglioglioma | Pleomorphic Xanthoastrocytoma | Diffuse Astrocytoma | Anaplastic Oligodendroglioma | Pilocytic Astrocytoma | Gangliocytoma | Central Neurocytoma | Oligodendroglioma, Childhood and other conditions

United States, Italy, Japan, Germany, Spain, France, Canada, Australia, Brazil, Israel, Russian Federation, Argentina, United Kingdom, Finland, Belgium, Denmark, Czechia, Sweden, Switzerland, Netherlands
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
Novartis Pharmaceuticals

Recruiting

Pediatric Long-Term Follow-up and Rollover Study

Glioblastoma | Astrocytoma | Anaplastic Astrocytoma | Neurofibromatosis Type 1 | Anaplastic Ganglioglioma | Anaplastic Pleomorphic Xanthoastrocytoma | Ganglioglioma | Pleomorphic Xanthoastrocytoma | Diffuse Astrocytoma | Anaplastic Oligodendroglioma | Pilocytic Astrocytoma | Gangliocytoma | Central Neurocytoma | Oligodendroglioma... and other conditions

United States, Belgium, Spain, Denmark, Germany, Czechia, Italy, Australia, Russian Federation, France, Brazil, Netherlands, Japan, Canada, Finland, Sweden, United Kingdom, Argentina, Israel

Clinical Trials on Pompe Disease Infantile-Onset in Netherlands

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Macedonia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations