Transfusion-dependent Beta-thalassemia
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia [Phase 4]
This clinical trial update describes a Phase 4 study evaluating hydroxyurea and metformin in transfusion-dependent beta-thalassemia, which h…
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CLINICAL_TRIALS trial_result · 2026-02-11Clinical Trial Update: Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia [NA]
The document reports the completion of a clinical trial evaluating ET-01 transplantation for safety and efficacy in transfusion-dependent be…
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CLINICAL_TRIALS research · 2026-02-03Clinical Trial Update: Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies [Phase 1]
Phase 1 clinical trial evaluating hematopoietic stem cell BCL11A enhancer gene editing for severe sickle cell disease and beta-thalassemia, …
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CLINICAL_TRIALS trial_result · 2026-01-27Clinical Trial Update: Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia [Phase 2]
A Phase 2 clinical trial evaluating a glucagon-like peptide-1 receptor agonist for diabetes management in children and adolescents with tran…
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CLINICAL_TRIALS research · 2025-12-17Clinical Trial Update: Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients
This clinical trial evaluates the use of the Hemanext One Hypoxic Red Blood Cell Storage System for transfusion in transfusion-dependent bet…
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CLINICAL_TRIALS trial_result · 2025-04-09Clinical Trial Update: Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This update provides long-term follow-up data for subjects with transfusion-dependent beta-thalassemia who were treated with an ex vivo gene…
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CLINICAL_TRIALS trial_result · 2025-04-02Clinical Trial Update: EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) [Phase 1, Phase 2]
Editas Medicine reports an update for EDIT-301, an autologous hematopoietic stem cell gene-editing therapy, currently in Phase 1/Phase 2 for…
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CLINICAL_TRIALS research · 2025-04-02Clinical Trial Update: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
A long-term follow-up study is ongoing to monitor participants with sickle cell disease or transfusion-dependent beta-thalassemia who receiv…
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CLINICAL_TRIALS research · 2025-03-10Clinical Trial Update: Β-Thalassemia Treatment with KL003 Cell Injection [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating KL003 cell injection for transfusion-dependent beta-thalassemia is currently recruiting.
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CLINICAL_TRIALS research · 2024-10-29Clinical Trial Update: Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
A clinical trial update discusses decision-making and quality-of-life considerations for hematologic diseases undergoing gene therapy, with …
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CLINICAL_TRIALS trial_result · 2023-12-13Clinical Trial Update: A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT) [Phase 1, Phase 2]
A Phase 1/2 trial evaluating the safety, tolerability, and efficacy of ST-400 for transfusion-dependent beta-thalassemia has completed with …
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CLINICAL_TRIALS trial_result · 2016-12-12Clinical Trial Update: Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec [Phase 2]
Phase 2 trial evaluating Deferasirox in transfusion-dependent beta-thalassemia patients with myocardial iron overload (cardiac MRI T2* < 20 …