Beta Thalassemia Major
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CLINICAL_TRIALS trial_result · 2026-03-20Clinical Trial Update: Iron Accumulation in Erythrocytes in Patients
This completed trial evaluated iron accumulation in erythrocytes in patients with beta-thalassemia major, providing data on iron handling in…
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CLINICAL_TRIALS trial_result · 2026-02-10Clinical Trial Update: A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major [Phase 1]
Phase 1 safety and efficacy study of CS-101 in beta-thalassemia major has completed.
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CLINICAL_TRIALS trial_result · 2025-09-04Clinical Trial Update: Elastography vs Iron Regulators in the Diagnosis of Hepatic Fibrosis- Iron Overload in Children With Beta- Thalassemia
A completed clinical study comparing elastography-based diagnostic approaches to iron-regulation methods for hepatic fibrosis and iron overl…
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CLINICAL_TRIALS trial_result · 2025-06-18Clinical Trial Update: Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major [Phase 4]
Phase 4 trial evaluating Basiliximab to prevent acute graft-versus-host disease after unrelated donor hematopoietic stem cell transplantatio…
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CLINICAL_TRIALS trial_result · 2025-05-18Clinical Trial Update: A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants [NA]
This entry updates the status of a completed clinical trial evaluating the safety and efficacy of the BD211 Drug Product in beta-thalassemia…
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CLINICAL_TRIALS trial_result · 2025-02-11Clinical Trial Update: Long Term Outcomes in β Thalassemia Major
This update summarizes long-term outcomes from a completed clinical trial in beta-thalassemia major sponsored by Ann & Robert H Lurie Childr…
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CLINICAL_TRIALS trial_result · 2024-03-15Clinical Trial Update: Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children [Phase 4]
A Phase 4 trial evaluating safety and efficacy of early deferiprone treatment in infants and young children with beta-thalassemia major anem…
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CLINICAL_TRIALS trial_result · 2023-06-18Clinical Trial Update: Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia. [Phase 2]
A Phase 2 trial assessing the safety and tolerability of sotatercept (ACE-011) in adults with beta-thalassemia was terminated; results were …
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CLINICAL_TRIALS trial_result · 2023-03-06Clinical Trial Update: PK of SOF/LED in HCV - Infected Adolescents With Haematological Disorders [Phase 3]
Phase 3 trial update evaluating the pharmacokinetics of sofosbuvir/ledipasvir in adolescents with hepatitis C infection and beta-thalassemia…
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CLINICAL_TRIALS trial_result · 2021-08-26Clinical Trial Update: Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation [Phase 2]
Phase 2a pilot study update for NBMI treatment in beta-thalassemia major patients requiring iron chelation.
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CLINICAL_TRIALS trial_result · 2020-11-05Clinical Trial Update: HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity [Phase 1, Phase 2]
A phase 1/2 clinical trial evaluating reduced intensity conditioning and hematopoietic stem cell transplantation for high-risk hemoglobinopa…
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CLINICAL_TRIALS trial_result · 2019-02-25Clinical Trial Update: Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus [Phase 3]
Phase 3 trial update on zinc supplementation in patients with beta-thalassemia major complicated with diabetes mellitus has completed.
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CLINICAL_TRIALS trial_result · 2018-07-18Clinical Trial Update: The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major [Phase 4]
This document covers a Phase 4 clinical trial evaluating whether metformin has hepatoprotective effects in patients with beta-thalassemia ma…
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CLINICAL_TRIALS trial_result · 2018-07-12Clinical Trial Update: Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia [NA]
A completed clinical trial compared spirulina supplementation to amlodipine for cardiac iron overload in children with beta-thalassemia majo…
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CLINICAL_TRIALS trial_result · 2017-10-25Clinical Trial Update: the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children [NA]
A completed clinical trial evaluated the potential immunomodulatory effects of Spirulina in children with beta-thalassemia major.
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CLINICAL_TRIALS trial_result · 2017-10-20Clinical Trial Update: Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia [Phase 2]
Phase 2 trial evaluating a reduced-intensity transplant conditioning regimen for severe thalassemia has completed, with results posted in Oc…
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CLINICAL_TRIALS trial_result · 2017-09-08Clinical Trial Update: Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified [Phase 1, Phase 2]
Clinical trial update for autologous gene therapy for beta-thalassemia major in Phase 1/2.
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CLINICAL_TRIALS trial_result · 2016-11-25Clinical Trial Update: Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major [NA]
This completed clinical trial evaluated the feasibility and safety of mobilizing CD34+ hematopoietic progenitor cells using granulocyte colo…
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CLINICAL_TRIALS trial_result · 2016-07-21Clinical Trial Update: Post Hematopoietic Stem Cell Transplantation [Phase 4]
Novartis-sponsored Phase 4 trial evaluating iron overload after hematopoietic stem cell transplantation in patients with beta-thalassemia ma…
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CLINICAL_TRIALS trial_result · 2014-08-29Clinical Trial Update: Beta-thalassemia and Microparticles [NA]
Completed clinical trial update investigating beta-thalassemia (major and intermedia) and microparticles.
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CLINICAL_TRIALS trial_result · 2014-07-23Clinical Trial Update: Clinical Trial of Deferasirox Combination Treatment With Deferiprone In Thalassaemia Patients [NA]
Completed clinical trial evaluating the combination of deferasirox and deferiprone in patients with beta-thalassemia major.
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CLINICAL_TRIALS trial_result · 2014-03-04Clinical Trial Update: Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)
The Thalassemia Clinical Research Network trial has completed its study (status COMPLETED) under sponsor Carelon Research.
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CLINICAL_TRIALS trial_result · 2012-12-20Clinical Trial Update: Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major [NA]
This completed clinical trial evaluated the safety of granulocyte colony-stimulating factor (G-CSF) mobilization in individuals with beta-th…
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CLINICAL_TRIALS trial_result · 2012-12-19Clinical Trial Update: Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major [Phase 1]
Phase 1 clinical trial evaluating the immunomodulatory effects of silymarin in patients with beta-thalassemia major. The study status is com…
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CLINICAL_TRIALS trial_result · 2011-07-29Clinical Trial Update: Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration) [Phase 4]
This Phase 4 extension study evaluates the efficacy and safety of deferasirox in transfusion-dependent beta-thalassemia patients with iron o…