Clinical Trials on Non-Transfusion-dependent Beta-Thalassemia

Total 499 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
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
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
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
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
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
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
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
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.

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
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
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

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
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
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
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
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
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
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
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
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
Tanta University

Unknown

Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major (Al-hijamah)

AL-Hijama in Thalassmia Major

Egypt
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

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
M.D. Anderson Cancer Center

Withdrawn

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Sickle Cell Disease | Sickle Beta Thalassemia | Beta Thalassemia Major | Sickle Cell-SS Disease | Sickle Beta 0 Thalassemia | Sickle Beta Plus Thalassemia

United States
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
University of British Columbia

Completed

Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada

Sickle Cell Disease | Beta-Thalassemia | Sickle Cell Trait | Sickle Cell-Beta Thalassemia | Sickle Cell-SS Disease

Canada, Nepal
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
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
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Bristol-Myers Squibb

Recruiting

A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.

Beta-thalassemia

China
Assiut University

Recruiting

The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients

Beta-Thalassemia

Egypt
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
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
CorrectSequence Therapeutics Co., Ltd
First Affiliated Hospital of Guangxi Medical University

Enrolling by invitation

CS-101 in Patients With β-thalassemia

Beta-Thalassemia

China
Rare Partners srl Impresa Sociale
Università degli Studi di Ferrara

Completed

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

Beta-Thalassemia

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

Completed

ACE-536 Extension Study - Beta Thalassemia

Beta-Thalassemia

Greece, Italy
University Hospital of Patras

Completed

Choroidal Thickness in Beta-thalassemia Patients

Beta-Thalassemia

Greece
IRCCS San Raffaele
Orchard Therapeutics; Fondazione Telethon

Unknown

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

Beta-Thalassemia

Italy
Hoffmann-La Roche

Completed

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

Beta-Thalassemia

Italy, Lebanon, Thailand
Prolong Pharmaceuticals

Completed

A Study of EPEG in Beta Thalassemia Patients

Beta-Thalassemia

Thailand
Shenzhen Geno-Immune Medical Institute

Unknown

Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector

Beta-Thalassemia

China
University of Washington
National Heart, Lung, and Blood Institute (NHLBI); George Papanicolaou Hospital

Completed

Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major

Beta-Thalassemia

Greece

Clinical Trials on Non-Transfusion-dependent Beta-Thalassemia

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