Clinical Trials on Sickle Cell-Beta0-Thalassemia in Italy

Total 85 results

Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

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

United States, Italy, Germany, Saudi Arabia
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
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
IRCCS San Raffaele
Telethon Institute for Gene Therapy (OSR-TIGET)

Active, not recruiting

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Beta Thalassaemia

Italy
Rare Partners srl Impresa Sociale
Università degli Studi di Ferrara

Completed

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (SIRTHALACLIN)

Beta-Thalassemia

Italy
IRCCS San Raffaele
Orchard Therapeutics; Fondazione Telethon

Unknown

Gene Therapy for Transfusion Dependent Beta-thalassemia (TIGET-BTHAL)

Beta-Thalassemia

Italy
Royal Brompton & Harefield NHS Foundation Trust
CORDA, The Heart Charity; Apotex Inc.; The Cooley's Anemia Foundation,; The UK...

Unknown

Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

Beta-Thalassemia

Italy
Novartis Pharmaceuticals

Completed

Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec

Beta Thalassemia Transfusion Dependent

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

Completed

ACE-536 Extension Study - Beta Thalassemia

Beta-Thalassemia

Greece, Italy
Hoffmann-La Roche

Completed

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

Beta-Thalassemia

Italy, Lebanon, Thailand
Università degli Studi di Ferrara
Azienda Ospedaliero, Universitaria Pisana; Rare Partners srl Impresa Sociale; Azienda Ospedaliero, Universitaria Meyer

Recruiting

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) (THALA-RAP)

Beta-Thalassemia

Italy
Azienda Ospedaliera V. Cervello

Completed

Efficacy Study of the Use of Sequential DFP-DFO Versus DFP (SEQDFPDFO)

Beta-Thalassemia | Thalassemia Major

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
Federico II University

Completed

The Role of OCTA in Patients Affected by Beta Thalassemia

Thalassemia

Italy
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

Beta-Thalassemia

France, United Kingdom, United States, Thailand, Italy, Germany
bluebird bio

Completed

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

Beta-Thalassemia

United States, Germany, United Kingdom, France, Greece, Italy
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Active, not recruiting

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

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

United States, United Kingdom, Canada, Germany, 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
Vifor (International) Inc.
Labcorp Corporation of America Holdings, Inc

Completed

Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia (VITHAL)

Beta-Thalassemia | Non-transfusion-dependent Thalassemia

Greece, Israel, Italy, Lebanon, Thailand
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
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
bluebird bio

Active, not recruiting

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Transfusion-dependent Beta-Thalassemia

United States, France, United Kingdom, Australia, Thailand, Italy, Germany, Greece
Shire

Terminated

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

Beta-Thalassemia | Transfusional Iron Overload

United States, Lebanon, Canada, Italy, Turkey
University Hospital of Ferrara

Recruiting

Atrial Fibrillation in Beta-Thalassemia

Atrial Fibrillation | Thalassemia

Italy
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (ENERGIZE)

Non-Transfusion-dependent Alpha-Thalassemia | Non-Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United Kingdom, Malaysia, United States, Netherlands, Bulgaria, Turkey, Italy, Canada, Brazil, France, United Arab Emirates, Denmark, Greece, Lebanon, Saudi Arabia
Shire

Completed

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

Beta-thalassemia | Transfusional Iron Overload

United States, United Kingdom, Italy, Thailand, Turkey
Università degli Studi di Ferrara

Completed

Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients

Thalassemia Major

Italy
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (ENERGIZE-T)

Transfusion-dependent Alpha-Thalassemia | Transfusion-dependent Beta-Thalassemia

Spain, Taiwan, Thailand, United States, France, Canada, Malaysia, Germany, Netherlands, Bulgaria, United Kingdom, Turkey, Italy, Greece, United Arab Emirates, Brazil, Denmark, Lebanon, Saudi Arabia
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
Cerus Corporation

Completed

Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion

Thalassemia Major

Turkey, Italy
Celgene
Acceleron Pharma Inc. (a wholly owned subsidiary of Merck Sharp and Dohme...

Completed

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia (BELIEVE)

Beta-Thalassemia | Erythrocyte Transfusion

United States, Israel, Australia, Turkey, Taiwan, Malaysia, United Kingdom, Canada, Thailand, France, Lebanon, Italy, Bulgaria, Greece, Tunisia
Imara, Inc.

Terminated

A Study of IMR-687 in Subjects With Beta Thalassemia

β Thalassemia

Israel, France, Malaysia, Turkey, Denmark, Georgia, Greece, Italy, Lebanon, Morocco, Netherlands, Tunisia, United Kingdom
Società Italiana Talassemie ed Emoglobinopatie

Completed

Vitamin D and Bisphosphonates in the Treatment of Sickle Cell Disease

Sickle Cells Disease

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
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
Azienda Ospedaliero-Universitaria di Modena

Recruiting

"Iron Overload and Endocrinological Diseases"

Iron Overload | Hemochromatosis | Thalassemia Major | Thalassemia Intermedia | Endocrinopathy

Italy
Novartis Pharmaceuticals

Completed

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

Thalassemia Major

Thailand, Turkey, Italy, Lebanon, Greece
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
Ospedale Microcitemico
Azienda Sanitaria Locale di Cagliari

Completed

Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major (DFODFPTM)

Iron Overload | Cardiomyopathy

Italy
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
ApoPharma

Completed

Efficacy Study in Removing Excess Iron From the Heart

Hemosiderosis | Thalassemia Major

Greece, Italy
Ente Ospedaliero Ospedali Galliera

Completed

Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia (PAH2010)

Pulmonary Arterial Hypertension | Thalassemia Major | Thalassemia Intermedia

Italy
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
Celgene

Recruiting

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

Beta-thalassemia | Myelodysplastic Syndromes (MDS) | Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis

United States, Japan, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Israel, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, Lebanon, China, Netherlands
University of Milano Bicocca

Completed

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

Covid19 | Thalassemia Major

Italy
San Luigi Gonzaga Hospital

Completed

Avascular Bone Necrosis in Sickle Cell Disease: a Pediatric Study. (OsteoSCD)

Sickle Cell Disease

Italy
Novartis Pharmaceuticals

Completed

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

Transfusional Iron Overload in β-thalassemia

United States, Greece, Argentina, Germany, Belgium, Italy, United Kingdom, France, Tunisia, Canada, Turkey, Brazil
University of Milano Bicocca
Comitato Maria Letizia Verga,Italy

Recruiting

Precision Exercise in Children With Malignant Hemopathies (SportTherapy)

Lymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | Drepanocytosis

Italy
Pfizer

Completed

Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease

Phase 1 Sickle Cell

United States, United Kingdom, Belgium, Netherlands, Italy

Clinical Trials on Sickle Cell-Beta0-Thalassemia in Italy

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