Clinical Trials on Genetic Association Studies in Netherlands

Total 794 results

Amryt Pharma

Recruiting

LOWER: Lomitapide Observational Worldwide Evaluation Registry (LOWER)

Homozygous Familial Hypercholesterolemia

Italy, United States, Argentina, Canada, France, Greece, Netherlands, United Kingdom
Hoffmann-La Roche

Completed

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors (STASEY)

Hemophilia A

Canada, Australia, Belgium, India, Germany, Israel, Italy, Spain, Colombia, United Kingdom, Netherlands, Saudi Arabia, Hungary, Guatemala, Russian Federation, Poland, Switzerland, Finland, Panama, Brazil, Mexico, Portugal, Romania, Sw...
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
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
Viridian Therapeutics, Inc.

Active, not recruiting

A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED) (THRIVE)

Thyroid Eye Disease

United States, Spain, United Kingdom, Canada, Germany, Italy, Netherlands, Australia, France, Turkey
Novartis Pharmaceuticals

Completed

A Phase I Study of Oral Asciminib (ABL001) in Patients With CML or Ph+ ALL

Chronic Myelogenous Leukemia | Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia

Germany, Australia, Netherlands, Spain, Korea, Republic of, France, Japan, Italy, Singapore, United States
Bioverativ, a Sanofi company
Swedish Orphan Biovitrum

Completed

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A

Hemophilia A

United States, France, Italy, Spain, Netherlands, United Kingdom, Ireland, New Zealand, Poland, Australia, Germany, Brazil, Sweden, Canada
Hoffmann-La Roche

Completed

An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies

Huntington Disease

United States, Canada, Netherlands, Germany, Spain, United Kingdom, Austria, Italy
Agios Pharmaceuticals, Inc.

Recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Sickle Cell Disease

United Kingdom, United States, Israel, Belgium, Netherlands, Germany, Canada, Brazil, France, Italy, Lebanon, Oman, Ghana, Kenya, Nigeria, Saudi Arabia, Turkey
Parion Sciences
Vertex Pharmaceuticals Incorporated

Completed

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia (CLEAN-PCD)

Primary Ciliary Dyskinesia

United States, United Kingdom, Netherlands, Denmark, Canada, Italy, Germany, Poland
The Medicines Company

Completed

Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C) (ORION-1)

Diabetes | Familial Hypercholesterolemia | Atherosclerotic Cardiovascular Disease

Canada, Netherlands, United States, United Kingdom, Germany
AbbVie

Terminated

Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/ABBV-119 or Galicaftor/Navocaftor/ABBV-576 Combination Therapies in Adult Participants With Cystic Fibrosis

Cystic Fibrosis (CF)

United States, Australia, Belgium, Hungary, Netherlands, New Zealand, Slovakia, United Kingdom
PTC Therapeutics

Completed

A Study of PTC923 in Participants With Phenylketonuria

Phenylketonuria

Spain, United States, Australia, Germany, United Kingdom, Brazil, Denmark, Canada, France, Georgia, Italy, Mexico, Netherlands, Portugal, Turkey
Vertex Pharmaceuticals Incorporated

Completed

Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Denmark, Germany, Italy, Canada
Baxalta now part of Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A

Hemophilia A

Spain, United States, Denmark, Italy, Taiwan, Malaysia, Belgium, Thailand, Turkey, Austria, Bulgaria, Canada, Finland, France, Germany, Hong Kong, Hungary, Korea, Republic of, Netherlands, Norway, Singapore, Ukraine, United Kingdom
Sol-Gel Technologies, Ltd.
Premier Research Group plc

Recruiting

Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome

Gorlin Syndrome

United States, Netherlands, Spain, France, United Kingdom, Germany, Poland
Boston Children's Hospital
Agios Pharmaceuticals, Inc.

Completed

Pyruvate Kinase Deficiency Natural History Study (PKD NHS)

Pyruvate Kinase Deficiency | Congenital Non-Spherocytic Hemolytic Anemia

United States, Germany, Canada, Czechia, Italy, Netherlands
Novartis Pharmaceuticals

Completed

Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease (STEADFAST)

Sickle Cell Disease (SCD)

United Kingdom, Spain, Greece, Brazil, Turkey, United States, France, Netherlands, Lebanon, Panama, Ireland
Pfizer

Terminated

A Study of ARRY-371797 (PF-07265803) in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation (REALM-DCM)

Dilated Cardiomyopathy | Lamin A/C Gene Mutation

United States, Spain, Canada, Belgium, Italy, Argentina, Mexico, Netherlands, Norway, United Kingdom
PTC Therapeutics
Cystic Fibrosis Foundation; ECFS-Clinical Trial Network (ECFS-CTN)

Completed

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

Cystic Fibrosis

United States, Spain, United Kingdom, Netherlands, Germany, France, Belgium, Italy, Australia, Argentina, Brazil, Bulgaria, Canada, Greece, Israel, Poland
Pfizer

Completed

A 52 Week Study To Assess The Use Of Bococizumab (PF-04950615; RN316) In Subjects With Heterozygous Familial Hypercholesterolemia (SPIRE-FH)

Heterozygous Familial Hypercholesterolemia

Canada, Netherlands, United Kingdom, United States, Spain, Poland, Finland, Norway, South Africa, Italy, Bulgaria
Clementia Pharmaceuticals Inc.

Terminated

An Efficacy and Safety Study of Palovarotene for the Treatment of MO (MO-Ped)

Exostoses, Multiple Hereditary

United States, Spain, Canada, Japan, Italy, Portugal, United Kingdom, Turkey, Australia, Belgium, France, Netherlands
Agios Pharmaceuticals, Inc.

Completed

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
Daiichi Sankyo, Inc.

Terminated

Efficacy and Safety of the ACAT Inhibitor CS-505 (Pactimibe) for Reducing the Progression of Carotid Artery Disease. This Study is Also Known as CAPTIVATE.

Atherosclerosis | Heterozygous Familial Hypercholesterolemia

United States, United Kingdom, Spain, South Africa, Israel, Canada, Netherlands, Norway, Sweden
Merck Sharp & Dohme LLC

Recruiting

A Study of Pembrolizumab (MK-3475) in Pediatric Participants With an Advanced Solid Tumor or Lymphoma (MK-3475-051/KEYNOTE-051)

Melanoma | Lymphoma | Solid Tumor | Classical Hodgkin Lymphoma | Microsatellite-instability-high Solid Tumor

France, Germany, Italy, Netherlands, Sweden, United Kingdom, Brazil, Israel, Korea, Republic of, United States
Novartis Pharmaceuticals

Completed

Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis (EAGER)

Cystic Fibrosis

United States, Germany, United Kingdom, Colombia, Spain, France, Netherlands, Israel, Canada, Italy, Chile, Australia, Hungary, Mexico
Boehringer Ingelheim

Completed

Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis

Cystic Fibrosis

United States, Australia, Belgium, France, Germany, Italy, Netherlands, New Zealand, Portugal, Russian Federation, United Kingdom
Bioverativ, a Sanofi company

Active, not recruiting

Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A (XTEND-ed)

Hemophilia A

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, China, France, Germany, Greece, Hungary, Ireland, Italy, Japan, Korea, Republic of, Netherlands, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom
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
Pharnext SA

Completed

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT) (PLEO-CMT)

Charcot-Marie-Tooth Disease Type 1A

United States, United Kingdom, Belgium, Canada, France, Netherlands, Spain, Germany
Genzyme, a Sanofi Company

Completed

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Gaucher Disease

United States, Sweden, India, Netherlands, Austria, Japan, China, Serbia, France, Croatia, Australia, Portugal, Canada, Brazil, Greece, Romania, Russian Federation
Cancer Prevention Pharmaceuticals, Inc.

Completed

Trial of Eflornithine Plus Sulindac in Patients With Familial Adenomatous Polyposis (FAP)

Familial Adenomatous Polyposis

United States, Belgium, Germany, Canada, Netherlands, Spain, United Kingdom
Corbus Pharmaceuticals Inc.
Cystic Fibrosis Foundation

Completed

Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

Cystic Fibrosis

United States, United Kingdom, Sweden, Canada, France, Austria, Germany, Netherlands, Italy, Belgium, Portugal, Bulgaria, Czechia, Greece, Hungary, Poland, Romania, Russian Federation, Serbia, Slovakia, Spain
BioMarin Pharmaceutical

Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

Sweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
Ancora Heart, Inc.

Recruiting

Clinical Evaluation of the AccuCinch® Ventricular Restoration System in Patients Who Present With Symptomatic Heart Failure With Reduced Ejection Fraction (HFrEF): The CORCINCH-HF Study

Dilated Cardiomyopathy | Heart Failure With Reduced Ejection Fraction (HFrEF)

United States, France, Netherlands, Czechia, Belarus, Belgium, Serbia
Baxalta now part of Shire

Completed

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Hemophilia A

United States, Spain, Netherlands, Australia, Czechia, Korea, Republic of, Poland, Germany, Taiwan, Israel, Ukraine, United Kingdom, Malaysia, Japan, Austria, Switzerland, Sweden, Bulgaria, Lithuania, Romania
Vertex Pharmaceuticals Incorporated

Completed

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

Cystic Fibrosis

United States, United Kingdom, Germany, Spain, Switzerland, Canada, Netherlands, Italy, Ireland, Sweden, France, Denmark
Bayer

Terminated

BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Hemophilia A

Belgium, Italy, Spain, United States, Canada, Switzerland, Estonia, Taiwan, France, Chile, Netherlands, United Kingdom, Austria, New Zealand, South Africa, Romania, Croatia, Israel, Germany, Poland, Australia, Denmark, Turkey, Argentina, Lithuan... and more
Vertex Pharmaceuticals Incorporated

Completed

A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

Cystic Fibrosis

United States, Spain, Ireland, Belgium, Netherlands, United Kingdom, Australia, France, Canada, Denmark, Germany, Italy, Israel
Gilead Sciences

Completed

Aztreonam Lysine for Pseudomonas Infection Eradication Study (ALPINE)

Cystic Fibrosis

United States, France, Spain, Germany, Austria, Belgium, Italy, Netherlands, Poland
Shire
Takeda Development Center Americas, Inc.

Completed

A Study of Long-Term Safety and Efficacy of Lanadelumab for Prevention of Acute Attacks of Non-histaminergic Angioedema With Normal C1-Inhibitor

Angioedema

United States, France, Netherlands, Spain, Hungary, Italy, Canada, Poland, Japan, 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
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
Alnylam Pharmaceuticals

Active, not recruiting

HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Transthyretin Amyloidosis | Amyloidosis, Hereditary

United States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T)

Transfusion-dependent Alpha-Thalassemia | Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United States, France, Canada, Malaysia, Germany, Netherlands, Bulgaria, United Kingdom, Turkey, Italy, Greece, United Arab Emirates, Brazil, Denmark, Lebanon, Saudi Arabia
Zogenix, Inc.
Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.

Active, not recruiting

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Dravet Syndrome | Epileptic Encephalopathy | Lennox Gastaut Syndrome

United States, Australia, Belgium, Canada, Denmark, France, Germany, Italy, Mexico, Netherlands, Poland, Spain, Sweden, United Kingdom
Teva Branded Pharmaceutical Products R&D, Inc.

Completed

A Clinical Study in Participants With Huntington's Disease (HD) to Assess Efficacy and Safety of Three Oral Doses of Laquinimod (LEGATO-HD)

Huntington's Disease

United States, Canada, Czechia, Germany, Italy, Netherlands, Portugal, Russian Federation, Spain, United Kingdom
Vertex Pharmaceuticals Incorporated

Completed

A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Cystic Fibrosis

United States, Belgium, Netherlands, United Kingdom, Canada, Australia, France, Germany, Italy, Czechia, Austria, Sweden, Greece
Ionis Pharmaceuticals, Inc.

Completed

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, France, Netherlands, Spain, United Kingdom, Belgium, Bulgaria, Canada, Denmark, Germany, Israel, Italy, Poland, Turkey
Insmed Incorporated

Completed

Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections

Pseudomonas Aeruginosa Infection

France, Poland, United Kingdom, Germany, Spain, Belgium, Bulgaria, Denmark, Greece, Canada, Netherlands, Hungary, Italy, Ireland, Sweden, Slovakia, Austria, Serbia

Clinical Trials on Genetic Association Studies in Netherlands

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