Clinical Trials on Alpha and Beta Thalassemia

Total 713 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
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
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
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
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Withdrawn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
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
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
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
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
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
HaEmek Medical Center, Israel

Completed

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

Hemolytic Anemia | Thalassemia Alpha

Israel
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
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
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
Aga Khan University

Completed

Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia

Beta Thalassemia Major
China Medical University Hospital

Completed

Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients

Beta-thalassemia Major
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
Lantu Biopharma

Not yet recruiting

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Beta-Thalassemia
bluebird bio
Center for International Blood and Marrow Transplant Research

Recruiting

A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Beta-Thalassemia

United States
Children's Hospital of Fudan University
CorrectSequence Therapeutics Co., Ltd

Recruiting

Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major

Beta-Thalassemia

China
Assiut University

Not yet recruiting

The Prevelence of IVS 1-6 (T-C) [HBB:c.92 +6 T-C] Gene Mutation in Suspected Cases of β Thalassemia in Assiut University Hospitals

Beta-Thalassemia
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
Assiut University

Not yet recruiting

Iron Status in BTM With Blood Transfusion

Beta-Thalassemia
CorrectSequence Therapeutics Co., Ltd
First Affiliated Hospital of Guangxi Medical University

Active, not recruiting

A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

Beta-Thalassemia

China
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
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
Assiut University

Unknown

Dermatological Abnormalities in Beta-thalassemia Major

Beta-Thalassemia
University of Washington
National Heart, Lung, and Blood Institute (NHLBI); Genzyme, a Sanofi Company; George Papanicolaou Hospital

Completed

Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major

Beta-Thalassemia

Greece
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
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
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
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

Clinical Trials on Alpha and Beta Thalassemia

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