Clinical Trials on Non-transfusion-dependent Thalassemia

Total 253 results

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
Novartis Pharmaceuticals

Completed

Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet (Jupiter)

Non-transfusion-dependent Thalassemia | Transfusion-dependent Thalassemia

Egypt, Turkey, Thailand, Lebanon, Morocco, Saudi Arabia, Vietnam
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
EdiGene (GuangZhou) Inc.

Active, not recruiting

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia (ET-01)

Transfusion Dependent Beta-Thalassaemia

China
EdiGene (GuangZhou) Inc.
PLA 923 Hospital

Active, not recruiting

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.

Transfusion Dependent Beta-Thalassaemia

China
Institute of Hematology & Blood Diseases Hospital...
EdiGene Inc.; The Affiliated Hospital Of Guizhou Medical University; Zunyi Medical...

Active, not recruiting

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

Transfusion Dependent Beta-Thalassaemia

China
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
Novartis Pharmaceuticals

Completed

Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia (THETIS)

Non-transfusion Dependent Thalassemia

Thailand, Turkey, Italy, Greece, China, United Kingdom, Lebanon, Tunisia
Regeneron Pharmaceuticals

Not yet recruiting

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body (FERVENT-1)

Non-transfusion Dependent Beta-thalassemia (NTDT)
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
First Affiliated Hospital of Guangxi Medical University
Genmedicn Biopharma Ltd.

Recruiting

A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]

Transfusion Dependent Beta-Thalassemia

China
Institute of Hematology & Blood Diseases Hospital...
R&D Kanglin Biotech

Active, not recruiting

Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.

Transfusion-dependent Beta-Thalassemia

China
Kanglin Biotechnology (Hangzhou) Co., Ltd.

Not yet recruiting

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

Transfusion-dependent Beta-Thalassemia

China
Institute of Hematology & Blood Diseases Hospital...
Kanglin Biotech

Recruiting

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Transfusion-dependent Beta-Thalassemia

China
First Affiliated Hospital of Guangxi Medical University
Genmedicn Biopharma Ltd.

Recruiting

A Study Evaluating the Safety and Efficacy of the GMCN-508A Drug Product in Transfusion-dependent α-Thalassemia Participants

Transfusion-dependent α-Thalassemia

China
Sun Yat-sen University

Recruiting

The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Thalassemia Major | Transfusion-dependent Anemia

China
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
Sangamo Therapeutics
Sanofi

Completed

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Transfusion Dependent Beta-thalassemia

United States
Novartis Pharmaceuticals

Completed

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

Beta Thalassemia Transfusion Dependent

Italy
Silence Therapeutics plc

Withdrawn

Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124

Non-transfusion-dependent Thalassemia | Low Risk Myelodysplastic Syndrome

United Kingdom, Bulgaria
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
Editas Medicine, Inc.

Not yet recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
Institute of Hematology & Blood Diseases Hospital

Recruiting

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Gene Therapy | Transfusion-dependent Beta-Thalassemia

China
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Recruiting

Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

Transfusion-dependent Thalassemia

Bangladesh
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Completed

Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia

Transfusion-dependent Thalassemia

Bangladesh
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
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
Novartis Pharmaceuticals

Withdrawn

Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia (CENTAurus)

Thalassemia (Transfusion Delendent)
Novartis Pharmaceuticals

Withdrawn

Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD
Novartis Pharmaceuticals

Terminated

Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload

Cardiac Iron Overload | Transfusion-dependent β-thalassemia Patients

Greece
University of Campania "Luigi Vanvitelli"

Completed

Endocrine Function During Deferasirox Therapy

Iron Overload | Endocrine; Complications | Transfusion-dependent Thalassemia

Italy
Medical University of Vienna
University of Vienna

Not yet recruiting

Plasticizer Exposure and Its Consequences on Health (PEACH)

Glioma | Intracranial Hemorrhages | Toxicity | Beta-thalassemia | Transfusion-dependent Beta-Thalassemia | Healthy Controls

Austria
Kepler University Hospital

Completed

Machine Learning-Based Prediction of Major Perioperative Allogeneic Blood Requirements in Cardiac Surgery (PREMATRICS)

Transfusion-dependent Anemia

Austria
Siriraj Hospital

Completed

Knowledge and Attitude in Perioperative Erythrocyte Transfusion in Medical Personnel in Siriraj Hospital

Transfusion-dependent Anemia

Thailand
Kepler University Hospital

Completed

Prediction of Transfusion-Associated Complications (PRETRACO)

Transfusion-dependent Anemia

Austria
Siriraj Hospital

Completed

An Analysis of Blood Management in Patients Having Hip or Knee Arthroplasty

Transfusion-dependent Anemia

Thailand
Talaris Therapeutics Inc.
Duke University

Withdrawn

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

Anemia, Sickle Cell | Thalassemia | Beta-Thalassemia | Bone Marrow Failure Syndromes | Diamond-Blackfan Anemia | Alpha-Thalassemia | Complex and Transfusion-dependent Hemoglobinopathies

United States
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 Children's Hospital
Novartis

Completed

Pilot Study for Patients With Poor Response to Deferasirox

Sickle Cell Disease | Thalassemia Major | Transfusion-dependent Hemachromatosis

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
Shire

Withdrawn

Treatment of Iron Overload Requiring Chelation Therapy

Metabolic Diseases | Iron Metabolism Disorders | Iron Overload | Beta-thalassemia | Transfusional Hemosiderosis | Transfusional Iron Overload | Iron Chelation
Johann Wolfgang Goethe University Hospital
Wuerzburg University Hospital

Recruiting

Liberal Transfusion Strategy in Elderly Patients (LIBERAL)

Anemia | Surgery | Transfusion-dependent Anemia

Germany
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
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
FerroKin BioSciences, Inc.

Completed

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

Beta-thalassemia | Transfusional Iron Overload

United States, Australia, Thailand
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
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

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
Children's Hospital of Philadelphia
Ann & Robert H Lurie Children's Hospital of Chicago; The Cooley's Anemia Foundation...

Completed

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

Thalassemia Major With Severe Transfusional Iron Overload

United States
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

Clinical Trials on Non-transfusion-dependent Thalassemia

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