Beta-thalassemia
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CLINICAL_TRIALS research · 2025-02-18Clinical Trial Update: ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
This clinical trials registry update reports completion of the ERN-EuroBloodNet registry documenting patients with rare red blood cell defec…
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CLINICAL_TRIALS trial_result · 2025-02-11Clinical Trial Update: A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
A clinical trial update indicating recruitment is ongoing for participants with Beta-Thalassemia treated with Betibeglogene Autotemcel.
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CLINICAL_TRIALS research · 2025-01-14Clinical Trial Update: A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia [Phase 1]
A Phase 1 multiple ascending dose study of 9MW3011 in patients with beta-thalassemia is currently recruiting.
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CLINICAL_TRIALS trial_result · 2024-11-29Clinical Trial Update: Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure [Phase 2]
Phase 2 trial update examining a nonmyeloablative peripheral blood stem cell transplantation approach for sickle cell disease and beta-thala…
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CLINICAL_TRIALS research · 2024-11-29Clinical Trial Update: the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia [EARLY_Phase 1]
This document reports an early-phase 1 clinical trial evaluating the safety and efficacy of HGI-001 Injection in transfusion-dependent beta-…
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CLINICAL_TRIALS research · 2024-11-29Clinical Trial Update: Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients [EARLY_Phase 1]
Early-phase clinical trial update evaluating safety and efficacy of β-globin–restored autologous hematopoietic stem cells in β-thalassemia m…
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CLINICAL_TRIALS research · 2024-11-14Clinical Trial Update: FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a longitudinal epidemiology study by xCures to follow outcomes across multiple rare diseases, and the study is currently recruitin…
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CLINICAL_TRIALS trial_result · 2024-11-07Clinical Trial Update: A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia [EARLY_Phase 1]
A pilot Phase 1 trial evaluating the safety and preliminary efficacy of YOLT-204 in transfusion-dependent beta-thalassemia; the study status…
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CLINICAL_TRIALS research · 2024-11-06Clinical Trial Update: A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
A post-marketing surveillance study to assess the safety of luspatercept in Korean patients with myelodysplastic syndrome or beta-thalassemi…
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CLINICAL_TRIALS research · 2024-10-23Clinical Trial Update: Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients. [Phase 1]
Phase 1 clinical trial evaluating safety and efficacy of lentiviral-transduced CD34+ hematopoietic stem cells in beta-thalassemia patients; …
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CLINICAL_TRIALS research · 2024-08-23Clinical Trial Update: Adherence of Beta Thalssemia Patients to Oral Chelation Therapy [NA]
This clinical trial update indicates Sohag University is recruiting Beta-thalassemia patients to study adherence to oral chelation therapy.
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CLINICAL_TRIALS other · 2024-07-24Clinical Trial Update: Plasticizer Exposure and Its Consequences on Health
A clinical trial update notes a study on plasticizer exposure and health outcomes, including beta-thalassemia and glioma, with status NOT_YE…
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CLINICAL_TRIALS trial_result · 2024-07-19Clinical Trial Update: Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen [NA]
This completed clinical trial evaluates Vitamin E supplementation in children with transfusion-dependent beta-thalassemia on different iron …
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CLINICAL_TRIALS trial_result · 2024-07-18Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002) [Phase 2]
A Phase 2 study evaluating the safety and efficacy of luspatercept for beta-thalassemia has completed and results have been posted.
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CLINICAL_TRIALS trial_result · 2024-07-12Clinical Trial Update: Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
A completed clinical trial evaluated additional risk minimisation measures for REBLOZYL among healthcare professionals.
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CLINICAL_TRIALS trial_result · 2024-06-14Clinical Trial Update: Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia [NA]
This entry notes a clinical trial evaluating safety and efficacy of gene-modified autologous hematopoietic stem cells in transfusion-depende…
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CLINICAL_TRIALS trial_result · 2024-06-13Clinical Trial Update: Stem Cell Transplant in Sickle Cell Disease and Thalassemia [Phase 2]
Phase 2 clinical trial update on hematopoietic stem cell transplantation for sickle cell disease and beta-thalassemia; the study is currentl…
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CLINICAL_TRIALS trial_result · 2024-06-04Clinical Trial Update: Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada [NA]
A completed clinical trial evaluated low-cost testing methods for detecting sickle cell disease and beta-thalassemia in Nepal and Canada, wi…
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CLINICAL_TRIALS research · 2024-06-04Clinical Trial Update: EKLF Gene Expression in β-thalassemia
A clinical trial investigating EKLF gene expression in beta-thalassemia; the study is currently not yet recruiting.
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CLINICAL_TRIALS research · 2024-05-29Clinical Trial Update: Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients
This clinical trial update outlines a study to detect KLF1/EKLF DNA mutations in beta-thalassemia patients. The trial status is NOT_YET_RECR…
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CLINICAL_TRIALS research · 2024-05-13Clinical Trial Update: Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia
A clinical trial update describing a study on thyroid function and HbA1c in relation to ferritin levels in adult patients with beta-thalasse…
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CLINICAL_TRIALS research · 2024-05-07Clinical Trial Update: Thrombophilia In Beta Thalassemia
This clinical trial update notes a study investigating thrombophilia in patients with beta-thalassemia. The trial is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2024-03-12Clinical Trial Update: Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study. [Phase 2]
A Phase 2 pilot trial comparing the efficacy and safety of thalidomide versus hydroxyurea in patients with beta-thalassemia was completed at…
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CLINICAL_TRIALS trial_result · 2024-01-23Clinical Trial Update: Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients [Phase 2]
Phase 2 trial update for sirolimus in transfusion-dependent beta-thalassemia patients has completed.
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CLINICAL_TRIALS trial_result · 2023-12-29Clinical Trial Update: A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia [Phase 3]
Phase 3 trial evaluating LentiGlobin BB305 for transfusion-dependent beta-thalassemia has completed with results posted in 2023.