Clinical Trials on Hereditary Corneal Dystrophy in Italy

Total 345 results

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
Amgen

Active, not recruiting

TEPEZZA® (Teprotumumab-trbw) Post-Marketing Requirement Study

Thyroid Eye Disease

United States, Spain, Germany, Italy, France, United Kingdom
University of Iowa
Children's Hospital of Philadelphia; University College London Hospitals; National... and other collaborators

Recruiting

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT (INC-6603)

Charcot Marie Tooth Disease

United States, United Kingdom, Australia, Italy
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
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
Viking Therapeutics, Inc.

Recruiting

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

United States, France, Germany, Italy, United Kingdom
Hoffmann-La Roche

Recruiting

A Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Participants With Thyroid Eye Disease

Thyroid Eye Disease | TED

Japan, Australia, Singapore, United States, Argentina, Germany, Italy, Hong Kong
Amgen

Recruiting

Apremilast Pediatric Study in Children With Active Oral Ulcers Associated With Behçet's Disease (BEAN)

Behçet Disease

Spain, United Kingdom, Turkey, France, Greece, Italy, Israel, Switzerland
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
Viridian Therapeutics, Inc.

Recruiting

A Randomized, Double-masked, Placebo-controlled Safety, Tolerability, and Efficacy Study of VRDN-001, a Humanized Monoclonal Antibody Directed Against the IGF-1 Receptor, in Participants With Chronic Thyroid Eye Disease (TED) (THRIVE-2)

Thyroid Eye Disease

Italy, Israel, United Kingdom, United States, France, Spain, Poland, Turkey, Australia, Germany, Hungary
Sling Therapeutics, Inc.

Active, not recruiting

A Phase 2b, Study of Linsitinib in Subjects With Active, Moderate to Severe Thyroid Eye Disease (TED) (LIDS)

Eye Diseases | Endocrine System Diseases | Thyroid Diseases | Thyroid Eye Disease | Graves Ophthalmopathy | Exophthalmos | Orbital Diseases | Thyroid Associated Ophthalmopathy | Graves Orbitopathy | Proptosis | IGF1R | Hashimoto

United States, Spain, Italy, United Kingdom, Canada
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
Immunovant Sciences GmbH

Recruiting

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

Thyroid Eye Disease

United States, Italy, Poland, United Kingdom, Canada, Israel, Germany, Japan, Georgia
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
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
Michael Shy
Johns Hopkins University; University of Colorado, Denver; King's College Hospital... and other collaborators

Recruiting

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others (INC-6601)

Charcot Marie Tooth Disease

United States, Australia, Italy, United Kingdom, Canada
University of Iowa
Johns Hopkins University; University of Colorado, Denver; King's College Hospital... and other collaborators

Recruiting

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2 (INC-6602)

Charcot-Marie-Tooth Disease, Type Ia (Disorder) | HMSN

United States, Italy, United Kingdom, Australia
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
AstraZeneca

Recruiting

PASS of Paediatric Patients Initiating Selumetinib

Neurofibromatosis Type 1

Spain, United Kingdom, Israel, France, Italy, Portugal, Switzerland, Germany, Austria
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
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
University Hospital Muenster

Completed

Pharmacokinetic Study of Doxorubicin in Children With Cancer (Doxo)

Soft Tissue Sarcoma | Acute Lymphoblastic Leukemia | Neuroblastoma | Wilms Tumor

Germany, France, United Kingdom, Italy
Novartis Pharmaceuticals

Completed

Phase III Study in Refractory Behcet's Disease (SHIELD)

Behcet Disease

India, Turkey, Spain, Germany, Italy, France, Israel, Greece, Korea, Republic of, Hong Kong, United States, Egypt, Jordan, Singapore, Switzerland, Taiwan, Tunisia
Noema Pharma AG

Recruiting

Basimglurant in Children, Adolescents, and Young Adults With TSC

Tuberous Sclerosis Complex

United States, Turkey, Poland, Spain, United Kingdom, India, Italy, Israel
AstraZeneca
Merck Sharp & Dohme LLC

Active, not recruiting

Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)

Neurofibromatosis 1 | Plexiform Neurofibroma (PN)

United States, Canada, France, Italy, Germany, China, United Kingdom, Spain, Japan, Russian Federation, Australia, Poland, Brazil
Pharnext S.C.A.
Worldwide Clinical Trials

Active, not recruiting

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PREMIER)

Charcot-Marie-Tooth Disease

United States, Spain, Belgium, Italy, Israel, Canada, Denmark, France, Germany
Novartis Pharmaceuticals

Withdrawn

Efficacy, Safety and Tolerability of Topically Applied LDE225 Cream (Hedgehog Pathway Inhibitor) in Adult Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)

Basal Cell Carcinoma

Greece, Switzerland, Germany, United Kingdom, Belgium, Canada, Czechia, Finland, Italy, Spain
Medical University of Vienna
Medical University of Graz; Charite University, Berlin, Germany; Hannover Medical... and other collaborators

Completed

Quality Improvement Project - "My Logbook! - I Know my Way Around!"; ("Mein Logbuch - Ich Kenne Mich Aus!")

Sarcoma | Hematologic Diseases | Leukemia | Brain Tumor | Neurofibromatoses | Neuroblastoma

Austria, Germany, Switzerland, Italy
Amgen

Completed

A Phase 3 Randomized, Double-blind Study to Evaluate the Efficacy and Safety of Apremilast (CC-10004) in Subjects With Active Behçet's Disease

Behçet's Syndrome

United States, Korea, Republic of, Israel, Italy, Turkey, France, Germany, Greece, Lebanon, Japan
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

Completed

Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

Familial Cold Autoinflammatory Syndrome | Cryopyrin-Associated Periodic Syndromes | Neonatal Onset Multisystem Inflammatory Disease | Muckle Wells Syndrome

United States, Germany, Italy, Spain, United Kingdom, Turkey, France, Belgium, India
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
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
Marinus Pharmaceuticals

Enrolling by invitation

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, China, Italy, France, Israel, Germany, Canada, Australia
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
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
Marinus Pharmaceuticals

Active, not recruiting

Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (TrustTSC)

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, Israel, Italy, Germany, France, Canada
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
Pfizer's Upjohn has merged with Mylan to form Viatris...

Terminated

Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP) (CHIP)

Adenomatous Polyposis Coli

Italy, United States, Belgium, Spain, Puerto Rico, Sweden, Israel, Czechia, Hong Kong, Hungary, Slovakia, South Africa, Ukraine, United Kingdom
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
Chinook Therapeutics U.S., Inc.

Active, not recruiting

Atrasentan in Patients With Proteinuric Glomerular Diseases (AFFINITY)

Diabetic Kidney Disease | Focal Segmental Glomerulosclerosis | Immunoglobulin A Nephropathy | IgA Nephropathy | Diabetic Nephropathy Type 2 | Alport Syndrome

United States, Spain, Korea, Republic of, Australia, Italy, United Kingdom
University of Siena

Recruiting

AutoInflammatory Disease Alliance Registry (AIDA) (AIDA)

Behcet Syndrome | Uveitis | Hereditary Autoinflammatory Diseases | Schnitzler Syndrome | Scleritis | PFAPA Syndrome | Still Disease | Autoinflammatory Syndrome, Unspecified | Vexas Syndrome

Egypt, Spain, Turkey, Italy, Saudi Arabia, Greece, Belgium, United States, Algeria, Brazil, Germany, Ghana, Iran, Islamic Republic of, Lebanon, Morocco, Poland, Portugal, Romania, Taiwan
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
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, Netherlands, Japan, Canada, Finland, Sweden, United Kingdom, Argentina, France, Brazil, Israel

Clinical Trials on Hereditary Corneal Dystrophy in Italy

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