Clinical Trials on Ichthyosiform Erythroderma, Congenital in Italy

Total 455 results

Bellicum Pharmaceuticals

Terminated

Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant

Lymphoma, Non-Hodgkin | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Myelodysplastic Syndrome | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child

United Kingdom, Italy
Istituto Ortopedico Rizzoli

Recruiting

Functional Instability in Patients Suffering From Collagen Disease and Joint Hypermobility (VIPIA)

Collagen Diseases | Joint Hypermobility | Hypermobility Syndrome | Laxity of Ligament | Danlos Disease, Ehlers

Italy
Ellipse Technologies, Inc.

Completed

A Retrospective Review of Early Onset Spinal Deformity Patients Treated With the Ellipse MAGEC System

Thoracic Insufficiency Syndrome

Hong Kong, New Zealand, Finland, Turkey, Egypt, Italy, United Kingdom
Novo Nordisk A/S

Completed

Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study (mentor™6)

Congenital Bleeding Disorder | Congenital FXIII Deficiency

Spain, United States, Canada, Italy, United Kingdom, Hungary
Bambino Gesù Hospital and Research Institute
Autour Des Williams; Acea

Unknown

Cooperative Parent Mediated Therapy in Children With Fragile X Syndrome and Williams Syndrome

Fragile X Syndrome | Williams Syndrome

Italy
IRCCS Fondazione Stella Maris

Completed

Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11) (3AL-SPG11)

Hereditary Spastic Paraplegia | Spastic Paraplegia Type 11

Italy
University of Milano Bicocca

Completed

Frequency of COVID-19 Antibodies in Patients With Hereditary Hematological Diseases (ThalaCoV)

Covid19 | Thalassemia Major

Italy
Acceleron Pharma Inc. (a wholly owned subsidiary...

Completed

Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia

B-Thalassemia

Italy, Greece
KU Leuven

Completed

Assessment of Patterns of Patient Reported Outcomes in Adults With Congenital Heart Disease - International Study II (APPROACH-IS II)

Congenital Heart Disease

Botswana, United States, Korea, Republic of, Belgium, Sweden, France, Switzerland, Denmark, Pakistan, Norway, Taiwan, Malta, Brazil, Bulgaria, India, Austria, Argentina, Australia, Cameroon, Canada, Chile, Colombia, Ethiopia, Greece, Italy and more
Mario Negri Institute for Pharmacological Research

Not yet recruiting

Blood Flow Conditions and Sounds in AVFs (APOLLO)

End Stage Renal Disease

Italy
DePuy International

Terminated

A Multi-centre Study to Assess the Long-term Performance of the Summit™ Hip in Primary Total Hip Replacement

Osteoarthritis | Rheumatoid Arthritis | Avascular Necrosis | Post-traumatic Arthritis | Congenital Hip Dysplasia | Slipped Capital Femoral Epiphysis | Collagen Disorders | Traumatic Femoral Fractures | Nonunion of Femoral Fractures

China, Hong Kong, Italy, Korea, Republic of, New Zealand
Hoffmann-La Roche

Completed

A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia

Beta-Thalassemia

Italy, Lebanon, Thailand
Mario Negri Institute for Pharmacological Research

Terminated

Sirolimus In Autosomal Dominant Polycystic Kidney Disease And Severe Renal Insufficiency (SIRENA-II)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Italy
University of Roma La Sapienza

Completed

Thyroid Function and Structure IN Klinefelter Syndrome (THINKS)

Hypothyroidism | Thyroiditis, Autoimmune | Klinefelter Syndrome

Italy
Novartis Pharmaceuticals

Completed

Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload

Transfusional Iron Overload | β-thalassemia

Italy
Istituto Ortopedico Rizzoli

Completed

Postoperative Alpha Lipoic Acid in the Carpal Tunnel Syndrome: a Randomized Controlled Trial.

Entrapment Neuropathy, Carpal Tunnel | Compression Neuropathy, Carpal Tunnel | Median Neuropathy, Carpal Tunnel

Italy
Novartis Pharmaceuticals

Completed

A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

Transfusional Iron Overload | β-thalassemia Major | Pediatric Rare Anemia

Italy, France
Virginia Commonwealth University
University of Western Ontario, Canada, Children's Health Research Institute; Fondazione Serena Onlus - Centro Clinico NeMO Milano

Recruiting

Trial Readiness and Endpoint Assessment in Congenital Myotonic Dystrophy (TREAT-CDM)

Congenital Myotonic Dystrophy

United States, Italy, Canada
ApoPharma

Completed

Efficacy Study in Removing Excess Iron From the Heart

Hemosiderosis | Thalassemia Major

Greece, Italy
Luca Sangiorgi

Recruiting

Registry of Ehlers-Danlos Syndrome (RED)

Ehlers-Danlos Syndrome

Italy
IRCCS Eugenio Medea

Completed

Assessment of the Psychophysical State During Rehabilitation Treatment With Lokomat

Cerebral Palsy | Acquired Brain Injury | Hereditary Spastic Paraplegia

Italy
Transposon Therapeutics, Inc.

Recruiting

TPN-101 in Aicardi-Goutières Syndrome (AGS)

Aicardi-Goutières Syndrome (AGS)

United Kingdom, France, Italy
Boston University
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

International Bicuspid Aortic Valve Consortium (BAVCon) (BAVCon)

Bicuspid Aortic Valve Disease | Thoracic Aortic Disease in Patients With a Bicuspid Aortic Valve

United States, Spain, United Kingdom, Canada, Italy
Tenaya Therapeutics
Medical University of South Carolina; Johns Hopkins University; University of... and other collaborators

Recruiting

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) (RIDGE)

Arrhythmogenic Right Ventricular Cardiomyopathy

United States, Italy, United Kingdom, France, Sweden, Germany
Valerion Therapeutics, LLC
Institut de Myologie, France; Genethon

Completed

Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM) (MTM)

Myotubular Myopathy

France, Belgium, United States, Canada, Germany, Italy, Spain
Gilead Sciences

Terminated

Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Adults With Long QT Syndrome Type 3

Long QT Syndrome Type 3

United States, Germany, Canada, Netherlands, Italy, France, Israel, United Kingdom
Kyowa Hakko Kirin UK, Ltd.

Completed

A Study of KW-3357 in Congenital Antithrombin Deficiency

Congenital Antithrombin Deficiency

France, Germany, Italy, Sweden, United Kingdom
IRCCS San Raffaele
Università Vita-Salute San Raffaele

Recruiting

Mechanisms of Somatic Mutation and Tumor Initiation in Pre-malignant Kidney Tubule Cells (SoMuKT)

Carcinoma, Renal Cell | Von Hippel-Lindau Disease

Italy
Abbott

Completed

Retrospective Palivizumab Study in Children With Hemodynamically Significant Congenital Heart Disease

Severe Respiratory Syncytial Virus Infection

Austria, Belgium, France, Germany, Italy, Norway, Poland, Slovenia, Spain, United Kingdom
A. Manzoni Hospital

Terminated

Everolimus on CKD Progression in ADPKD Patients

Polycystic Kidney Diseases

Italy
Novartis

Completed

Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Fragile X Syndrome

France, Italy, Switzerland
Mario Negri Institute for Pharmacological Research

Completed

Somatostatin in Polycystic Kidney: a Long-term Three Year Follow up Study (ALADIN)

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Italy
Ente Ospedaliero Ospedali Galliera
University of Campania "Luigi Vanvitelli"; Reggio Calabria; Ospedale Maggiore... and other collaborators

Completed

Efficacy and Safety of Neridronate (Nerixia®)to Treat Osteoporosis in Patients With TM and TI (Nerixia)

Osteoporosis | Thalassemia Major | Thalassemia Intermedia

Italy
IRCCS Fondazione Stella Maris

Recruiting

Optical Imaging as a Tool for Monitoring Brain Function in Fragile X Syndrome

Fragile X Syndrome

Italy
Novartis Pharmaceuticals

Completed

Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.

Thalassemia Major

Thailand, Turkey, Italy, Lebanon, Greece
University of Genova

Unknown

Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A) (TreSPE)

Charcot-Marie-Tooth Disease | Charcot-Marie-Tooth Disease Type 1A

Italy
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

Hematologic Diseases | Genetic Diseases, Inborn | Hemoglobinopathies | Thalassemia | Beta-Thalassemia

United States, Germany, Italy, United Kingdom, Canada
Celgene

Completed

To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care

Beta-Thalassemia

Greece, Italy, Lebanon, Thailand, Turkey
AstraZeneca

Completed

PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) (HESTIA4)

Sickle Cell Disease

Belgium, United Kingdom, Italy, Spain, Kenya, Lebanon
University of Campania "Luigi Vanvitelli"
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; University of Milan and other collaborators

Recruiting

The PRO-FUTURE Project (PRO-FUTURE)

Chronic Kidney Diseases | Ureteropelvic Junction Obstruction

Italy
Crinetics Pharmaceuticals Inc.

Recruiting

Evaluate the Safety, Efficacy, and Pharmacokinetics of CRN04894 in Participants With Congenital Adrenal Hyperplasia (TouCAHn)

Congenital Adrenal Hyperplasia | Classic Congenital Adrenal Hyperplasia

Brazil, India, United States, Argentina, Italy, United Kingdom
Institut National de la Santé Et de la Recherche...
UPCET; PARTNERS for International clinical Research; European Fundation for the... and other collaborators

Active, not recruiting

Prophylactic Treatment of the Ductus Arteriosus in Preterm Infants by Acetaminophen (TREOCAPA)

Acetaminophen | Patency of the Ductus Arteriosus | Extreme Prematurity

France, Norway, Belgium, Estonia, Ireland, Switzerland, Sweden, Denmark, Finland, Hungary, Italy, Portugal
Taewoong Medical Co., Ltd.
Avania

Active, not recruiting

PULSTA Transcatheter Pulmonary Valve Pre-Approval Study

Heart Diseases | Tetralogy of Fallot | Pulmonary Valve Stenosis | Pulmonary Valve Regurgitation

Germany, Spain, Korea, Republic of, Italy, Netherlands, Turkey
IRCCS San Raffaele

Recruiting

Overtaking Intra and Inter Tumoral Heterogeneity In Von Hippel-Lindau Related Renal Cancer (ITHORinVHL)

Clear Cell Renal Cell Carcinoma | VHL Syndrome

Italy
Mario Negri Institute for Pharmacological Research

Recruiting

Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases (ANTHEM)

Healthy | Autosomal Dominant Polycystic Kidney | Atypical Hemolytic Uremic Syndrome | Membranoproliferative Glomerulonephritis

Italy
Azienda Ospedaliero-Universitaria Consorziale Policlinico...
Institute of Biomembranes, Bioenergetics and Molecular Biotechnologies; Istituti... and other collaborators

Completed

Establishment of the Human Intestinal and Salivary Microbiota Biobank - Kidney Diseases (BIOMIS-Nefro)

Autosomal Dominant Polycystic Kidney Disease (ADPKD) | IgA Nephropathy (IgAN) | Diabetic Kidney Disease (DKD) | Advanced-Chronic Kidney Disease (CKD)

Italy
Celgene

Terminated

Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.

Beta Thalassemia Intermedia | Beta Thalassemia Major

France, United Kingdom, Italy, Greece
Grifols Therapeutics LLC
Instituto Grifols, S.A.

Completed

Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Participants With Congenital Afibrinogenemia (IG0902)

Congenital Afibrinogenemia

India, United States, Italy, Lebanon
Actelion

Completed

BREATHE 5-OL: Tracleer (Bosentan) in Patients With Pulmonary Arterial Hypertension Related to Eisenmenger Physiology

Pulmonary Arterial Hypertension

Canada, United Kingdom, Italy, United States, Netherlands, Australia, Austria, Belgium, France, Germany, Spain
University of Ulm
University of Pisa; IRCCS Azienda Ospedaliero-Universitaria di Bologna; Medical... and other collaborators

Recruiting

Registry for Patients With Lipodystrophy (ECLip Registry)

Lipodystrophy Acquired | Lipodystrophy Congenital

Austria, France, Germany, Hungary, Italy, Portugal, Spain, Netherlands, Russian Federation, Slovenia, Switzerland, Turkey, United Kingdom

Clinical Trials on Ichthyosiform Erythroderma, Congenital in Italy

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