Clinical Trials on Amyloidosis, Hereditary in Netherlands

Total 187 results

Maastricht University Medical Center
Sun Pharmaceutical Industries Limited

Recruiting

Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study

Carcinoma | Carcinoma, Basal Cell | Skin Cancer | Basal Cell Nevus Syndrome | Neoplasms, Basal Cell | Basal Cell Carcinoma | Locally Advanced Basal Cell Carcinoma | Metastatic Basal Cell Carcinoma | Gorlin Syndrome | Basal Cell Tumor | Neoplasm of Skin

Netherlands
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)

Alexander Disease

United States, United Kingdom, Australia, Canada, Israel, Italy, Japan, Netherlands
Kubota Vision Inc.
Food and Drug Administration (FDA)

Completed

Safety and Efficacy of Emixustat in Stargardt Disease (SeaSTAR)

Stargardt Disease

United States, United Kingdom, Germany, Spain, Brazil, Canada, Denmark, France, Italy, Netherlands, South Africa
Passage Bio, Inc.

Suspended

Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease (GALax-C)

Leukodystrophy, Globoid Cell

United States, Brazil, Netherlands, United Kingdom, Canada, Israel
AstraZeneca
Merck Sharp & Dohme LLC

Active, not recruiting

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN (SPRINKLE)

Neurofibromatosis Type 1

United States, Spain, Germany, Netherlands, Russian Federation, Italy, Japan
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
Johns Hopkins University
Mayo Clinic; Medical University of Graz; Radboud University Medical Center; University... and other collaborators

Recruiting

Oral N-acetylcysteine for Retinitis Pigmentosa (NAC Attack)

Retinitis Pigmentosa

United States, Austria, Mexico, Netherlands, United Kingdom, Germany, Canada, Switzerland
Jaeb Center for Health Research
Foundation Fighting Blindness

Recruiting

Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (Uni-Rare)

Retinitis Pigmentosa | Inherited Retinal Degeneration

United States, United Kingdom, France, Finland, Israel, Netherlands, Switzerland, Australia, Canada, Brazil, Mexico
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
Janssen Research & Development, LLC

Completed

A Study of JNJ-64251330 in Participants With Familial Adenomatous Polyposis

Adenomatous Polyposis Coli

United States, Germany, Korea, Republic of, France, Netherlands, Spain, Puerto Rico
University of Leicester

Unknown

Chemotherapy Plus Surgery in Treating Children at Risk of or With Stage I Wilms' Tumor

Kidney Cancer

France, Netherlands, Sweden, Norway, Denmark, Portugal, Belgium, Croatia, Czech Republic, Greece, Italy, Poland, Slovenia, Spain, United Arab Emirates
Alnylam Pharmaceuticals

Completed

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

Acute Hepatic Porphyria

United States, Belgium, Bulgaria, France, Italy, Mexico, Spain, Taiwan, United Kingdom, Netherlands, Korea, Republic of, Switzerland, Czechia, Poland, Finland, Australia, Germany, Israel, Norway, South Africa
River 3 Renal Corp.

Recruiting

Study to Evaluate R3R01 in Patients With Alport Syndrome and Patients With Focal Segmental Glomerulosclerosis

Focal Segmental Glomerulosclerosis | Alport Syndrome

United States, Belgium, United Kingdom, France, Germany, Netherlands
Jaeb Center for Health Research
Foundation Fighting Blindness

Active, not recruiting

Rate of Progression in USH2A-related Retinal Degeneration (RUSH2A)

Usher Syndrome, Type 2A | Retinitis Pigmentosa 39

United States, Canada, France, Germany, Netherlands, United Kingdom
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)

Thyroid Eye Disease

United States, Spain, United Kingdom, Canada, Germany, Italy, Netherlands, Australia, France, Turkey
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
bluebird bio

Terminated

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
Pharnext S.C.A.
Premier Research Group plc; Eurofins Optimed; Synteract HCR (Syneos Health); Gr... and other collaborators

Active, not recruiting

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A (PLEO-CMT-FU)

Charcot-Marie-Tooth Disease, Type IA

United States, Belgium, Canada, France, Netherlands, Spain, United Kingdom
bluebird bio

Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
Gyroscope Therapeutics Limited
Novartis Pharmaceuticals

Active, not recruiting

GTSCOPE - To Evaluate the Natural Progression of Dry Age-related Macular Degeneration (AMD)

Macular Degeneration | Retinal Degeneration | Geographic Atrophy | Dry Age-related Macular Degeneration | Retinal Disease | Macular Atrophy

United States, Spain, United Kingdom, France, Germany, Australia, Netherlands, Poland
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
Jazz Pharmaceuticals

Completed

A Randomized Controlled Trial of Cannabidiol (GWP42003-P, CBD) for Seizures in Tuberous Sclerosis Complex (GWPCARE6)

Seizures | Tuberous Sclerosis Complex

United States, Spain, Australia, Poland, United Kingdom, Netherlands
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
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy (EMBOLDEN)

Metachromatic Leukodystrophy (MLD)

United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy
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
Sol-Gel Technologies, Ltd.

Terminated

Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome)

Basal Cell Nevus Syndrome

United States, France, Belgium, Denmark, Germany, Italy, Netherlands, Spain, United Kingdom
Janssen Research & Development, LLC

Completed

A Study of Guselkumab in Participants With Familial Adenomatous Polyposis

Adenomatous Polyposis Coli

United States, Germany, France, Israel, Netherlands, Poland, Puerto Rico, Spain, Sweden
Hoffmann-La Roche

Completed

A Study of Two Vismodegib Regimens in Participants With Multiple Basal Cell Carcinomas

Basal Cell Carcinoma

United States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Chemotherapy With or Without Surgery, Radiation Therapy, or Stem Cell Transplantation in Treating Young Patients With Kidney Tumors

Kidney Cancer

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
Children's Oncology Group
National Cancer Institute (NCI)

Completed

DNA Variations in the Gene in Young Patients With Wilms' Tumor

Kidney Cancer

United States, Canada, Puerto Rico, Australia, Netherlands, Switzerland, New Zealand
PellePharm, Inc.

Completed

Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)

Basal Cell Nevus Syndrome

United States, Spain, Germany, United Kingdom, Italy, France, Belgium, Canada, Denmark, Netherlands
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Combination Chemotherapy Alone or With Radiation Therapy in Treating Children With Kidney Cancer

Kidney Cancer

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
Novartis Pharmaceuticals

Completed

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)

Lymphangioleiomyomatosis (LAM) | Tuberous Sclerosis Complex (TSC)

United States, United Kingdom, Germany, Italy, Russian Federation, Netherlands, Japan, Canada, Poland, France, Spain
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
Travere Therapeutics, Inc.

Recruiting

Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases (EPPIK)

Focal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | Minimal Change Disease | Alport Syndrome | IgA Vasculitis

United States, Italy, Netherlands, Poland, Spain, United Kingdom, Germany, Sweden
National Cancer Institute (NCI)

Completed

Rebeccamycin Analogue in Treating Children With Solid Tumors or Non-Hodgkin's Lymphoma

Sarcoma | Lymphoma | Unspecified Childhood Solid Tumor, Protocol Specific | Brain and Central Nervous System Tumors | Neuroblastoma | Retinoblastoma

United States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
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 Amyloidosis, Hereditary in Netherlands

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