Clinical Trials on Non-Transfusion-dependent Alpha-Thalassemia

Total 276 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
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
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
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
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
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
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
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Withdrawn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
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
University of California, San Francisco

Recruiting

International Registry of Patients With Alpha Thalassemia (ATM Registry)

Alpha-Thalassemia | Alpha Thalassemia Major | Alpha Thalassemia Minor

United States
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
University of California, San Francisco
California Institute for Regenerative Medicine (CIRM)

Enrolling by invitation

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Hemoglobinopathies | Alpha Thalassemia Major | Thalassemia Major | Fetal Hydrops | Hemoglobinopathy; With Thalassemia | Fetal Anemia | Alpha; Thalassemia | Thalassemia Alpha | A-Thalassemia

United States
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
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)
Institute of Hematology & Blood Diseases Hospital
R&D Kanglin Biotech

Recruiting

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

Transfusion-dependent Beta-Thalassemia

China
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
HaEmek Medical Center, Israel

Completed

Demographic, Clinical and Laboratory Characteristics of Children With Alpha Thalassemia in Northern Israel

Hemolytic Anemia | Thalassemia Alpha

Israel
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
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
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
The 923rd Hospital of Joint Logistics Support Force...
Guangzhou Reforgene Medicine Co., Ltd.

Recruiting

Safety and Efficacy of RM-004 Cells for Hemoglobin H-Constant Spring Disease

Hemoglobinopathies | Hereditary Diseases | Alpha Thalassemia Hemoglobin H Constant Spring

China
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...

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
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
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)
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
National Institute of Allergy and Infectious Diseases...

Recruiting

Screening for Alpha Thalassemia in Healthy Volunteers

Alpha Thalassemia

United States
Deborah Rund

Completed

Genetics of Alpha Thalassemia in Israeli Ethnic Groups

Alpha Thalassemia

Israel
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
Shenzhen Hemogen

Recruiting

the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia

α-thalassemia

China
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
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
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
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
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
National Institute of Allergy and Infectious Diseases...

Recruiting

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

Malaria | Sickle Cell Disease | Alpha and Beta Thalassemia | Human Physiology

United States

Clinical Trials on Non-Transfusion-dependent Alpha-Thalassemia

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