Clinical Trials on Congenital Cataract in Italy

Total 489 results

Merck KGaA, Darmstadt, Germany
Merck Serono S.A., Geneva

Completed

First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study

Turner Syndrome | Idiopathic Growth Hormone Deficiency

Spain, France, Canada, United Kingdom, Czech Republic, Argentina, Germany, Italy, Sweden
Edimer Pharmaceuticals

Completed

Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (ECP-002)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Patients With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

Congenital Adrenal Hyperplasia

United States, United Kingdom, France, Greece, Italy, Spain, Belgium, Canada, Germany, Poland
Merck KGaA, Darmstadt, Germany

Completed

Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®

Growth Hormone Deficiency | Turner Syndrome

Argentina, Australia, Austria, Canada, France, Germany, Italy, Norway, Russian Federation, Singapore, Spain, Sweden, United Kingdom
EspeRare Foundation
Iqvia Pty Ltd; Pierre Fabre Medicament

Recruiting

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (EDELIFE)

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

United States, Spain, France, Germany, Italy, United Kingdom
Edimer Pharmaceuticals

Completed

Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (ECP-015)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
Spruce Biosciences

Active, not recruiting

A Ph2b to Evaluate Tildacerfont in the Reduction of Glucocorticoid Steroid Doses in Adult CAH

Congenital Adrenal Hyperplasia

United States, Australia, Brazil, Estonia, Germany, Korea, Republic of, Latvia, Lithuania, Poland, Romania, Spain, Sweden, Turkey, Canada, Italy, United Kingdom
Edimer Pharmaceuticals

Active, not recruiting

Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 (ECP-002e)

X-linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
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
Bristol-Myers Squibb

Recruiting

A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (α)-Thalassemia

Anemia

Thailand, China, Canada, Turkey, Greece, Taiwan, Italy, Hong Kong
Hoffmann-La Roche

Completed

A Non-Drug Study of The Suitability of Neurocognitive Tests And Functioning Scales For The Measurement of Cognitive And Functioning Changes in Individuals With Down Syndrome

Down Syndrome

United States, France, Italy, Canada, United Kingdom, Argentina, Spain
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Enrolling by invitation

A Long-term Follow-up Study in Participants Who Received CTX001

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

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

Recruiting

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD). (CROSSWALK-a)

Sickle Cell Disease

United States, Brazil, Italy, Spain, Kenya, Netherlands, United Kingdom, France, South Africa
Neurocrine Biosciences

Active, not recruiting

Global Safety and Efficacy Registration Study of Crinecerfont for Congenital Adrenal Hyperplasia (CAHtalyst)

Congenital Adrenal Hyperplasia

United States, Spain, Belgium, Austria, Bulgaria, Canada, Czechia, France, Germany, Greece, Israel, Italy, Netherlands, Poland, Portugal, Serbia, Sweden, United Kingdom
Sergiusz Jozwiak
Medical University of Vienna; Charite University, Berlin, Germany; Institut National... and other collaborators

Unknown

Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex (EPISTOP)

Epilepsy | Tuberous Sclerosis Complex | TSC

Poland, Austria, Belgium, Czech Republic, France, Germany, Italy, Netherlands
Spruce Biosciences

Terminated

A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH

Congenital Adrenal Hyperplasia

United States, Australia, Brazil, Canada, Denmark, Estonia, Germany, Ireland, Italy, Korea, Republic of, Latvia, Lithuania, Netherlands, Poland, Romania, Spain, Sweden, Switzerland, Turkey, United Kingdom
TWi Biotechnology, Inc.

Recruiting

An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)

Generalized Epidermolysis Bullosa Simplex

United States, Taiwan, Australia, Philippines, Austria, Italy, Poland
Celgene
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway...

Recruiting

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Beta-Thalassemia

United States, Turkey, Italy, Thailand, Germany, Greece, China, India, Lebanon
Andreas L. Serra

Unknown

The Eurocyst Initiative: Building a Network of ADPKD Reference Centers Across Europe

Autosomal Dominant Polycystic Kidney Disease

France, Switzerland, Germany, Netherlands, Belgium, Czech Republic, Italy, Spain, Turkey, United Kingdom
Novartis Pharmaceuticals

Completed

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study (THALASSA)

Non-transfusion Dependent Thalassemia

Greece, Taiwan, Italy, United Kingdom, Lebanon, Turkey, Malaysia, Thailand, United States
Octapharma

Recruiting

Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery

Congenital Antithrombin Deficiency

Spain, Italy, United States, Serbia, France, Hungary, Austria, Germany, United Kingdom, Israel
Bayer

Completed

Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis (TAURUS)

Hemophilia A, Congenital

Slovenia, Italy, Germany, France, United States, Belgium, Canada, Colombia, Greece, Luxembourg, Netherlands, Spain, Taiwan
Hoffmann-La Roche

Recruiting

Study to Investigate the Pharmacokinetics and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype. (Aldebaran)

Angelman Syndrome

United States, Australia, Spain, Italy, France, Germany
Natera, Inc.
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Terminated

Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial (PreNATUS)

Aneuploidy | Trisomy 21 | Trisomy 18 | Trisomy 13

United States, Ireland, Canada, Italy, Korea, Republic of, Spain
Grifols Therapeutics LLC
Grifols Biologicals, LLC

Terminated

Alphanate in Immune Tolerance Induction Therapy

Hemophilia A, Congenital

United States, Spain, Italy, India, Canada, Russian Federation
Gedeon Richter Plc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of RGH-706 in Prader-Willi Syndrome

Prader-Willi Syndrome

United States, Spain, France, Italy, Czechia
Novartis Pharmaceuticals

Recruiting

Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

Sickle Cell Disease

United States, Belgium, Spain, Lebanon, Brazil, Italy, Oman, Turkey, Germany, Colombia, France
ADDMEDICA SASA

Completed

European Sickle Cell Disease Cohort - Hydroxyurea (ESCORT-HU)

Sickle Cell Disease

France, Germany, French Guiana, Greece, Guadeloupe, Italy, Martinique
Celgene Corporation
ICON Clinical Research

Completed

Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality

Myelodysplastic Syndromes

Germany, Israel, United Kingdom, Spain, Belgium, Italy, France, Netherlands, Sweden
Protagonist Therapeutics, Inc.

Completed

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (TRANSCEND)

Chronic Anemia | β-thalassemia | Ineffective Erythropoiesis

United States, Malaysia, Turkey, Thailand, United Kingdom, Italy, Lebanon, Tunisia, Greece
Albireo

Recruiting

Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

Biliary Atresia

Netherlands, United States, Spain, Italy, Taiwan, Korea, Republic of, France, Germany, United Kingdom, Canada, Belgium, China, Australia, Poland, Turkey, Malaysia, New Zealand, Hungary, Israel
Embo-Flüssigkeiten A.G.

Active, not recruiting

Treatment of Cerebral Arteriovenous Malformations With SQUID Liquid Embolic Agent (CHOICE) (CHOICE)

Cerebral Arteriovenous Malformation

France, Denmark, Germany, Belgium, Italy
Scioderm, Inc.
Amicus Therapeutics

Completed

ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

Epidermolysis Bullosa

Italy, Spain, Serbia, United States, Australia, Netherlands, Austria, Germany, Israel, Lithuania, France, United Kingdom, Poland
Galapagos NV

Terminated

A Study to Evaluate the Effects of GLPG2737 in Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal Dominant Polycystic Kidney Disease

Belgium, Netherlands, Italy, Spain, Czechia, Germany, Poland
La Jolla Pharmaceutical Company

Terminated

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Beta-Thalassemia

Australia, Thailand, United States, Greece, Italy, Lebanon, Turkey, United Kingdom
Novo Nordisk A/S

Completed

Safety of NNC 0172-0000-2021 in Healthy Male Subjects and Subjects With Haemophilia A or B (Explorer 1)

Healthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

Austria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
Pfizer

Active, not recruiting

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)

Sickle Cell Disease

United States, Oman, United Kingdom, France, Egypt, Ghana, Italy, Kenya, Nigeria, Saudi Arabia
LMU Klinikum
University of Pisa; German Federal Ministry of Education and Research; European...

Recruiting

Global Registry and Natural History Study for Mitochondrial Disorders

Mitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEO

Austria, Germany, Italy
Cyprus Institute of Neurology and Genetics

Recruiting

Study of the Role of Genetic Modifiers in Hemoglobinopathies (INHERENT)

Sickle Cell Disease | Hemoglobinopathies | Thalassemia Alpha | Thalassemia, Beta

United States, Denmark, Spain, Belgium, Portugal, Congo, The Democratic Republic of the, Greece, Argentina, Brunei Darussalam, Cyprus, Israel, Italy, Nigeria, Malaysia, Angola, Pakistan
Hoffmann-La Roche

Recruiting

A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD) (CROSSWALK-c)

Sickle Cell Disease

Spain, United States, Brazil, United Kingdom, Italy, Turkey, Kenya, Netherlands, France, South Africa, Lebanon
Silence Therapeutics plc

Completed

A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome

Non-transfusion-dependent Thalassemia | Low Risk Myelodysplastic Syndrome | Very-Low Risk Myelodysplastic Syndrome

Thailand, Israel, Jordan, United Kingdom, Germany, Italy, Malaysia
Amryt Research Limited

Completed

Phase III Efficacy and Safety Study of Oleogel-S10 in Epidermolysis Bullosa (EASE)

Epidermolysis Bullosa

United States, Spain, Israel, Australia, France, Denmark, Switzerland, Italy, Hong Kong, Argentina, Austria, Brazil, Chile, Colombia, Czechia, Georgia, Germany, Greece, Hungary, Ireland, Romania, Russian Federation, Serbia, Singapore, U... and more
Novartis Pharmaceuticals

Completed

Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).

Sickle Cell Disease

United States, Italy, United Kingdom, France, Canada
Novo Nordisk A/S

Completed

Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A (pathfinder™1)

Congenital Bleeding Disorder | Haemophilia A

United States, Germany, Switzerland, United Kingdom, Italy, Japan, Turkey
Ipsen

Completed

Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq (OPTIMA)

Renal Insufficiency, Chronic | Pituitary Diseases | Dwarfism | Turner Syndrome

France, Germany, Finland, Spain, Belgium, Czechia, Denmark, Greece, Italy, Romania, Russian Federation, Slovakia, Ukraine, United Kingdom
Novo Nordisk A/S

Completed

Observational Registry of the Treatment of Glanzmann's Thrombasthenia

Congenital Bleeding Disorder | Glanzmann's Disease

Spain, Bulgaria, Pakistan, Belgium, Germany, France, United Kingdom, Italy, Hungary, Netherlands, United States, Algeria, Austria, Sweden, Switzerland
Merck Sharp & Dohme LLC

Completed

A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Children With Sickle Cell Disease (V114-023/PNEU-SICKLE)

Pneumococcal Infections

United States, Brazil, Colombia, Dominican Republic, Greece, Italy, Panama
Otsuka Pharmaceutical Development & Commercialization...

Completed

Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Germany, Italy, Belgium, United Kingdom
Hoffmann-La Roche

Completed

A Study of RG1662 in Adults and Adolescents With Down Syndrome (CLEMATIS)

Down Syndrome

United States, Italy, Argentina, France, Spain, Mexico, Canada, New Zealand, Singapore, United Kingdom
Pierre Fabre Dermatology

Completed

Study to Demonstrate the Efficacy and Safety of Propranolol Oral Solution in Infants With Proliferating Infantile Hemangiomas Requiring Systemic Therapy

Infantile Hemangioma

Germany, United States, France, Spain, Peru, Australia, Lithuania, Italy, Canada, Czech Republic, Hungary, Mexico, New Zealand, Poland, Romania, Russian Federation

Clinical Trials on Congenital Cataract in Italy

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