Search clinical trials for: Beta Thalassemia

Total 8685 results

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
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, Germany, Canada, Italy
Celgene
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway...

Completed

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia (BEYOND)

Thalassemia

United States, Greece, Italy, Lebanon, Thailand, United Kingdom
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
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
NYU Langone Health

Terminated

Evaluating Postoperative Non-Opioid Pain Management Utilizing Local Anesthetics Coupled With Modulated Coagulation

Opioid Pain Medication

United States
Children's Hospital Karachi

Completed

Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients

Thalassemia, Beta

Pakistan
Sangamo Therapeutics

Enrolling by invitation

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Blood and Lymphatic Diseases

United States
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
Ann & Robert H Lurie Children's Hospital of Chicago
National Heart, Lung, and Blood Institute (NHLBI)

Active, not recruiting

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

Sickle Cell Disease | Sickle B+ Thalassemia | Sickle Beta Zero Thalassemia | Sickle Cell Hemoglobin C

United States
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
Istanbul University

Completed

Cardiac Management of Patients With Thalassemia Minor and Breast Cancer (AQUA)

Heart Diseases | Breast Cancer | Thalassemia Minor

Turkey
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
Cairo University

Completed

Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia

Quality of Life | Fatigue | Cognitive Change | Beta-Thalassemia

Egypt
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
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
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
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
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
Columbia University

Active, not recruiting

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Sickle Cell Disease | Beta Thalassemia

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
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
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
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
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
Vifor (International) Inc.
Labcorp Drug Development Inc

Withdrawn

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

Beta-Thalassemia

United States, Bulgaria, Israel
Bioray Laboratories
PLA 923 Hospital

Terminated

β-globin Restored Autologous HSC in β-thalassemia Major Patients

β-thalassemia Major

China
Case Western Reserve University
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone

Hematologic Diseases | Iron Overload | Hemoglobinopathies | Thalassemia | Beta-Thalassemia | Anemia (Iron-Loading)
Celgene

Completed

Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)

Myelodysplastic Syndromes | Beta-thalassemia

Austria
Prothya Biosolutions

Terminated

Apotransferrin in Patients With β-thalassemia

β-thalassemia Intermedia

Netherlands
Ente Ospedaliero Ospedali Galliera

Completed

Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia (PAH2010)

Pulmonary Arterial Hypertension | Thalassemia Major | Thalassemia Intermedia

Italy
Imara, Inc.

Terminated

A Study of IMR-687 in Subjects With Beta Thalassemia

β Thalassemia

Israel, France, Malaysia, Turkey, Denmark, Georgia, Greece, Italy, Lebanon, Morocco, Netherlands, Tunisia, United Kingdom
bluebird bio

Completed

A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease

Sickle Cell Disease | Beta-Thalassemia Major

France
Nova Laboratories Limited

Completed

Pharmacokinetics of Oral Hydroxyurea Solution (HUPK)

Sickle Cell Disease | Sickle Cell Hemoglobin C | Sickle Cell-beta-thalassemia | Sickle-Cell; Hemoglobin Disease, Thalassemia

United Kingdom, Jamaica
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
Children's Hospital Karachi

Completed

Clinical Experience of Thalidomide in Thalassemic Patients

Thalassemia, Beta

Pakistan
Università degli Studi di Ferrara

Completed

Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients

Thalassemia Major

Italy
EmeraMed

Completed

Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation (EMERA007)

Beta Thalassemia Major

Albania
Northwell Health

Completed

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Sickle Cell Disease | Beta Thalassemia-Major

United States
Fred Hutchinson Cancer Center
National Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaborators

Completed

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

Non-Neoplastic Hematologic and Lymphocytic Disorder

United States
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
Ann & Robert H Lurie Children's Hospital of Chicago

Completed

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

Sickle Cell Disease | Sickle B+ Thalassemia | Sickle Beta Zero Thalassemia | Sickle Cell Hemoglobin C

United States
Protagonist Therapeutics, Inc.

Completed

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia (TRANSCEND)

Chronic Anemia | β-thalassemia | Ineffective Erythropoiesis

United States, Malaysia, Turkey, Thailand, United Kingdom, Italy, Lebanon, Tunisia, Greece
Protagonist Therapeutics, Inc.

Completed

Safety Study for Beta Thalassemia Subjects on PTG-300 (TRANSCEND)

β-thalassemia | Ineffective Erythropoiesis

Malaysia, Thailand, United States, Greece, United Kingdom, Turkey, Lebanon, Tunisia
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
Novartis

Completed

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

Iron Overload | Beta-thalassemia

United States
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
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
Acceleron Pharma Inc. (a wholly owned subsidiary...

Completed

ACE-536 Extension Study - Beta Thalassemia

Beta-Thalassemia

Greece, Italy
AstraZeneca

Completed

PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4) (HESTIA4)

Sickle Cell Disease

Belgium, United Kingdom, Italy, Spain, Kenya, Lebanon

Search clinical trials for: Beta Thalassemia

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