Clinical Trials on Transfusion Dependent Beta Thalassemia in New Zealand

Total 40 results

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
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
Editas Medicine, Inc.

Recruiting

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

Hemoglobinopathies | Thalassemia Major | Thalassemia Intermedia | Transfusion Dependent Beta Thalassemia

United States, Canada
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
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
Boston University

Completed

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

Beta-Thalassemia

United States, United Kingdom
Memorial Sloan Kettering Cancer Center

Completed

Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major

Beta Thalassemia Major | Congenital Anemias

United States
Northwell Health

Completed

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Sickle Cell Disease | Beta Thalassemia-Major

United States
Ionis Pharmaceuticals, Inc.

Terminated

Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)

Beta Thalassemia Intermedia

Australia, Thailand, Greece, Lebanon, Turkey
Phoenicia BioScience
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)

Sickle Cell Disease | Beta Thalassemia Intermedia

United States, Canada
Genzyme, a Sanofi Company

Completed

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

Beta-Thalassemia

United States
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
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

Hematologic Diseases | Anemia, Sickle Cell | Beta-Thalassemia | Hematopoietic Stem Cell Transplantation

United States, Canada
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
Terumo BCTbio
United States Department of Defense; U.S. Army Medical Research and Development... and other collaborators

Terminated

Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE) (PRAISE)

Transfusion Dependent Thalassemia

United States, Israel, Italy, 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
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
HealthCore-NERI
National Heart, Lung, and Blood Institute (NHLBI)

Terminated

Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases

Heart Diseases | Cardiovascular Diseases | Beta-Thalassemia

United States
Novartis Pharmaceuticals

Completed

Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

Beta-Thalassemia

United States
Sideris Pharmaceuticals

Terminated

Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

Iron Overload | Beta-Thalassemia

United States, Canada, Lebanon, Thailand, Turkey
Columbia University

Active, not recruiting

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Sickle Cell Disease | Beta Thalassemia

United States
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
Memorial Sloan Kettering Cancer Center

Recruiting

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Sickle Cell Disease | Thalassemia | Thalassemia, Beta

United States
Memorial Sloan Kettering Cancer Center

Active, not recruiting

ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene

Confirmed Diagnosis of ß-thalassemia Major

United States
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
Bristol-Myers Squibb

Recruiting

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

Anemia

India
Weill Medical College of Cornell University

Unknown

The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study

Thalassemia Major

United States
DisperSol Technologies, LLC

Completed

Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation

Thalassemia Major

Thailand, United States
Columbia University

Completed

High-Tc Susceptometer to Monitor Transfusional Iron Overload

Myelodysplastic Syndromes | Sickle Cell Disease | Aplastic Anemia | Transfusional Iron Overload | Thalassemia Major

United States
HealthCore-NERI
National Heart, Lung, and Blood Institute (NHLBI); Thalassemia Clinical Research...

Completed

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

Hematologic Diseases | Iron Overload | Hypertension, Pulmonary | Osteoporosis | Thalassemia | Beta-Thalassemia | Anemia, Cooley's

United States, Canada
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, Israel, United Kingdom, Turkey, Greece, France, Australia, Belgium, Bulgaria, Canada, Germany, Italy, Malaysia, Spain, Sweden, Taiwan, Thailand, Tunisia, China, Lebanon, Netherlands
Mitchell Cairo

Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Hodgkin Lymphoma | Sickle Cell Disease | Acute Leukemia | Beta-Thalassemia | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Diamond Blackfan Anemia | Amegakaryocytic Thrombocytopenia | Kostmann

United States
New York Medical College

Completed

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

Lymphoma | Leukemia | Sickle Cell Disease | Histiocytosis | Immunodeficiencies | Inborn Errors of Metabolism | Bone Marrow Failure | Beta Thalassemia

United States
Novartis Pharmaceuticals

Completed

Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias

Myelodysplastic Syndromes | Fanconi Syndrome | Anemia, Aplastic | Beta-thalassemia | Anemia, Diamond-Blackfan

United States
Invenux, LLC
State University of New York - Downstate Medical Center

Active, not recruiting

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

Sickle Cell Disease | Sickle-Beta Zero Thalassemia

United States
Novartis Pharmaceuticals

Completed

An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)

Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia

Australia
Novartis Pharmaceuticals

Completed

Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload

Transfusion-dependent Anemia

Egypt, Hungary, Turkey, United States, Bulgaria, Italy, Belgium, Russian Federation, Philippines, France, Malaysia, India, Oman, Panama, Lebanon, Thailand, Tunisia
Novartis Pharmaceuticals

Completed

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Anemia | Hemosiderosis

United States, Germany, Belgium, United Kingdom, Italy, Canada, France
Diane George

Active, not recruiting

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sickle Cell Disease | Hemoglobinopathies | Severe Congenital Neutropenia | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Macrophage Activation Syndrome | Bone Marrow Failure Syndrome | Amegakaryocytic Thrombocytopenia | Schwachman Diamond Syndrome | Primary Immunodeficiency Syndromes | Acquired Immunodeficiency... and other conditions

United States
Novartis Pharmaceuticals

Completed

Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Chronic Iron Overload Due to Transfusion-dependant Anemias

Italy, Austria, Thailand, Spain, Germany, Mexico, Malaysia, Greece, United Kingdom, France, Saudi Arabia, United Arab Emirates, Lebanon, Russian Federation, United States, Argentina

Clinical Trials on Transfusion Dependent Beta Thalassemia in New Zealand

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