Search clinical trials for: Congenital transfusion dependent anemias

Total 14164 results

Paul Szabolcs

Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT (HSCT+RIC)

Congenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA)

United States
Abfero Pharmaceuticals, Inc

Withdrawn

SP-420 in Subjects With Transfusion-dependent Beta-Thalassemia or Other Rare Anemias

Iron Overload | Beta-Thalassemia

Thailand, Lebanon, Turkey, Canada
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
University Health Network, Toronto

Completed

MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias (MAFIO)

Thalassemia Major | Chronic Blood Transfusion Related Iron Overload

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)
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
Kind Pharmaceuticals LLC

Not yet recruiting

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

β -Thalassemia
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
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
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
Bristol-Myers Squibb

Recruiting

A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India

Anemia

India
Shenzhen Hemogen

Recruiting

the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia

β-thalassemia

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
Shenzhen Hemogen

Recruiting

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

α-thalassemia

China
Shanghai BDgene Co., Ltd.
Ruijin Hospital

Not yet recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

β-thalassemia

China
Shanghai BDgene Co., Ltd.
Shanghai Children's Medical Center

Not yet recruiting

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia

β-thalassemia
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
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
Pharmacosmos A/S
ICON plc

Recruiting

A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia

Beta Thalassemia Major Anemia

Denmark
Bristol-Myers Squibb

Recruiting

A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (α)-Thalassemia

Anemia

Thailand, China, Canada, Turkey, Greece, Taiwan, Italy, Hong Kong
Vertex Pharmaceuticals Incorporated
CRISPR Therapeutics

Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

Hematologic Diseases | Genetic Diseases, Inborn | Sickle Cell Disease | Hemoglobinopathies | Sickle Cell Anemia | Thalassemia | Beta-Thalassemia

United States, Italy, Germany, Saudi Arabia
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
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
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
Forma Therapeutics, Inc.

Recruiting

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

Sickle Cell Disease | Thalassemia

United States, Lebanon, Canada
Phoenicia BioScience
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

The BENeFiTS Trial in Beta Thalassemia Intermedia (PB04-001)

Sickle Cell Disease | Beta Thalassemia Intermedia

United States, Canada
Università degli Studi di Ferrara
Azienda Ospedaliero, Universitaria Pisana; Rare Partners srl Impresa Sociale; Azienda Ospedaliero, Universitaria Meyer

Recruiting

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) (THALA-RAP)

Beta-Thalassemia

Italy
Masonic Cancer Center, University of Minnesota

Recruiting

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Sickle Cell Disease | Severe Congenital Neutropenia | Paroxysmal Nocturnal Hemoglobinuria | Glanzmann Thrombasthenia | Shwachman-Diamond Syndrome | Diamond Blackfan Anemia | Transfusion Dependent Alpha- or Beta- Thalassemia | Non-Malignant Hematologic Disorders

United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

Thalassemia

United States, Canada, United Kingdom
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
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
Bangabandhu Sheikh Mujib Medical University, Dhaka...

Completed

Safety & Efficacy of Thalidomide in Children With Transfusion Dependent Thalassemia

Transfusion-dependent Thalassemia

Bangladesh
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
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
Bioray Laboratories
Xiangya Hospital of Central South University; First Affiliated Hospital of... and other collaborators

Enrolling by invitation

Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia

Beta-Thalassemia

China
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
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
Rare Partners srl Impresa Sociale
Università degli Studi di Ferrara

Completed

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

Beta-Thalassemia

Italy
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 & 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
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
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
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
Northwell Health

Terminated

Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia (ACE-011-DBA)

Diamond Blackfan Anemia

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
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

Search clinical trials for: Congenital transfusion dependent anemias

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