Clinical Trials on Genetics in Netherlands

Total 795 results

Origin Biosciences

Completed

A Retrospective, Observational, Noninterventional Data Collection Study for Patients With Molybdenum Cofactor Deficiency Who Have Been Previously Treated With Cyclic Pyranopterin Monophosphate (cPMP)

Molybdenum Cofactor Deficiency

United States, Australia, United Kingdom, Germany, Netherlands, Turkey
Pfizer

Completed

Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting

Hemophilia A

Spain, France, Turkey, Netherlands, Germany, Italy, Ukraine
Amicus Therapeutics

Completed

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

Fabry Disease

Canada, Belgium, United States, Australia, Netherlands
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Hypercholesterolemia, Familial

United States, Netherlands
bluebird bio

Completed

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD)

United States, France, Germany, Italy, Netherlands, United Kingdom
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
University Hospital, Toulouse
European Society for Paediatric Endocrinology

Completed

PWS European Blood Bank for Infants and Controls From 0 to 48 Months

Prader-Willi Syndrome

Sweden, Belgium, France, Germany, Netherlands, United Kingdom
Protalix
Chiesi Farmaceutici S.p.A.

Completed

Safety and Efficacy of PRX-102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)

Fabry Disease

Australia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
Eli Lilly and Company

Completed

Somatropin + Leuprorelin vs Somatropin Alone in Pubertal Children With Idiopathic Short Stature (Phoenix)

Idiopathic Short Stature (ISS)

France, Netherlands
QLT Inc.

Completed

Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

LCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)

Canada, United States, Germany, Netherlands, United Kingdom
Cerenis Therapeutics, SA

Completed

Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects (MODE)

Homozygous Familial Hypercholesterolemia

United States, Canada, Italy, Netherlands, United Kingdom
Northwestern University

Completed

Impact of the Severity of Pediatric Psoriasis on Childhood Body Mass Index

Psoriasis | Atopic Dermatitis

Chile, United States, Netherlands, Canada, Hong Kong, Italy
Khondrion BV
Julius Clinical

Completed

The KHENERGYZE Study

Mitochondrial Diseases | Mitochondrial Myopathies | MELAS Syndrome | Mitochondrial Encephalomyopathies | MIDD

Netherlands, Denmark, Germany, United Kingdom
Alnylam Pharmaceuticals

Completed

An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1

Primary Hyperoxaluria | PH1 | RNAi Therapeutic | siRNA | AGT

France, United Kingdom, Netherlands, Israel, Germany
Castle Creek Pharmaceuticals, LLC

Terminated

Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

Epidermolysis Bullosa Simplex

United States, Israel, Australia, Austria, France, Germany, Netherlands, United Kingdom
Kathelijn Fischer
Radboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaborators

Recruiting

Pharmacokinetic-guided Dosing of Emicizumab (DosEmi)

Adolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, Severe

Netherlands
Edgewise Therapeutics, Inc.
Medpace, Inc.; SYSNAV; ImagingNMD

Recruiting

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Becker Muscular Dystrophy

United States, Netherlands, United Kingdom
Orchard Therapeutics

Recruiting

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT (HURCULES)

MPS-IH (Hurler Syndrome)

Netherlands, United States, Italy, United Kingdom
Christoph Gasche
Prof. Dr. Gabriela Möslein, Germany; Prof. Dr. Hans Vasen, The Netherlands; Prof... and other collaborators

Terminated

Mesalamine for Colorectal Cancer Prevention Program in Lynch Syndrome (MesaCAPP)

Colorectal Cancer

Netherlands, Austria, Germany, Israel, Poland, Sweden
Genzyme, a Sanofi Company

Completed

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

Glycogen Storage Disease Type II

United States, France, Netherlands
Kyowa Kirin Pharmaceutical Development Ltd

Recruiting

A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

X-Linked Hypophosphatemia

France, Netherlands, United Kingdom
Sanofi
Regeneron Pharmaceuticals

Completed

An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia

Hypercholesterolemia

Slovenia, Taiwan, Russian Federation, Canada, Netherlands, Brazil, Denmark, Mexico, Spain, Turkey
Kastle Therapeutics, LLC
Ionis Pharmaceuticals, Inc.

Completed

Study of ISIS 301012 (Mipomersen) in Heterozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy

Hypercholesterolemia, Familial

United States, Netherlands
Horizon Pharma USA, Inc.

Completed

Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis

Cystinosis

France, United States, United Kingdom, Belgium, Italy, Netherlands
Perspectum

Recruiting

Repeatability and Reproducibility of Multiparametric MRI

Liver Diseases | Cirrhosis | Primary Sclerosing Cholangitis | Autoimmune Hepatitis | Hemochromatosis | Non-Alcoholic Fatty Liver Disease | Non-Alcoholic Steatohepatitis | Primary Biliary Cirrhosis | Viral Hepatitis

Netherlands, United Kingdom
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
Santhera Pharmaceuticals

Completed

Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Freidreich's Ataxia

Germany, Netherlands, France, Austria, Belgium
Radboud University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development

Unknown

Implementation of a New Strategy to Identify HNPCC Patients

Colorectal Neoplasms | Hereditary Nonpolyposis Colorectal Cancer

Netherlands
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
LoneStar Heart, Inc.

Completed

A Randomized, Controlled Study to Evaluate Algisyl-LVR™ as a Method of Left Ventricular Augmentation for Heart Failure (AUGMENT-HF)

Heart Failure | Dilated Cardiomyopathy

Italy, Germany, Australia, Netherlands, New Zealand, Romania
Dyne Therapeutics

Recruiting

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 (ACHIEVE)

Myotonic Dystrophy Type 1 (DM1)

United Kingdom, France, Netherlands, Italy, Germany, New Zealand
LYSOGENE

Active, not recruiting

Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance)

Mucopolysaccharidosis Type IIIA

Germany, United States, United Kingdom, France, Netherlands
Sarepta Therapeutics, Inc.

Active, not recruiting

Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)

Duchenne Muscular Dystrophy

United States, Belgium, Spain, United Kingdom, Netherlands, Germany, Canada, Italy
Sanofi

Completed

Proof of Concept Study of Rilzabrutinib in Adult Patients With Moderate-to-severe Atopic Dermatitis

Atopic Dermatitis

Netherlands, Chile, United States, Canada, Czechia, Germany, Poland
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
Rhythm Pharmaceuticals, Inc.

Completed

Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity

Obesity | Genetic Obesity | Obesity Due to Melanocortin 4 Receptor Deficiency

United States, United Kingdom, Germany, Netherlands, France, Canada, Greece, Israel, Spain
AstraZeneca

Completed

An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia

Familial Hypercholesterolaemia

United States, Netherlands, Norway, Canada, Belgium
QLT Inc.

Completed

Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations

LCA (Leber Congenital Amaurosis) | RP (Retinitis Pigmentosa)

United States, Canada, Germany, Netherlands, United Kingdom
AstraZeneca

Completed

A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia (HYDRA)

Homozygous Familial Hypercholesterolemia (HoFH)

Malaysia, Canada, Israel, Netherlands, Taiwan, Belgium, Denmark
Minoryx Therapeutics, S.L.

Active, not recruiting

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients. (Advance)

Adrenoleukodystrophy

Spain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
Intellia Therapeutics

Active, not recruiting

NTLA-2002 in Adults With Hereditary Angioedema (HAE) (NTLA-2002)

Hereditary Angioedema

France, New Zealand, Germany, United Kingdom, Netherlands, Australia
Scioderm, Inc.
Amicus Therapeutics

Terminated

Open Label Extension Study to Evaluate the Long-term Safety of Zorblisa (SD-101-6.0) in Patients With Epidermolysis Bullosa

Epidermolysis Bullosa

Poland, Spain, Serbia, Netherlands, United States, Austria, Australia, Israel, Germany, France, United Kingdom, Lithuania
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
Santhera Pharmaceuticals

Completed

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) (DELOS)

Muscular Dystrophy, Duchenne | Ambulatory Care

United States, Spain, Austria, Belgium, France, Germany, Italy, Netherlands, Sweden, Switzerland
Jaeb Center for Health Research
Usher 1F Collaborative; Marjorie C. Adams Foundation

Active, not recruiting

Rate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F (RUSH1F)

Retinal Degeneration | Retinitis Pigmentosa | Eye Diseases, Hereditary

Switzerland, United States, Germany, Netherlands, Canada, France, Israel
Ferring Pharmaceuticals

Completed

An Open-Label, Multi-centre, Phase III Study of Local Tolerability of ZOMACTON 10MG

Turner's Syndrome

Czech Republic, France, Netherlands
Rhythm Pharmaceuticals, Inc.

Completed

Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Patients With Specific Gene Defects in the MC4R Pathway

Bardet-Biedl Syndrome | POMC Deficiency

Netherlands, United States, Germany, Canada, Puerto Rico, United Kingdom
Shire

Terminated

An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.

Sanfilippo Syndrome | Mucopolysaccharidosis (MPS)

United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
Ionis Pharmaceuticals, Inc.

Active, not recruiting

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Hereditary Angioedema

Netherlands, United States
Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, Japan, United Kingdom, Italy, Australia, Poland, Bulgaria, Czechia, Netherlands

Clinical Trials on Genetics in Netherlands

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