Beta Thalassemia Major
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene [Phase 1]
Phase 1 clinical trial update for beta-thalassemia major using autologous CD34+ cells transduced with TNS9.3.55, a lentiviral vector encodin…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: Risk Factors Affecting Growth in Thalassemic Children at AUCH
A clinical trial at Assiut University is investigating risk factors affecting growth in children with beta-thalassemia major. The study is c…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major [Phase 2]
Phase 2 safety and efficacy study of CS-101 for beta-thalassemia major, with the study not yet recruiting.
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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 research · 2025-12-17Clinical Trial Update: Gonadal Function in Thalassemic Patient
A clinical trial investigates gonadal function in patients with beta-thalassemia major; the study is currently not yet recruiting.
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FDA approval · 2025-12-11FDA Approved: DEFERIPRONE — Original Application (DR. REDDY'S LABORATORIES, INC.)
FDA approved Dr. Reddy's Laboratories' original application for deferiprone, a generic iron chelator, enabling its marketing in the United S…
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CLINICAL_TRIALS research · 2025-10-06Clinical Trial Update: Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With β-TTD [NA]
A clinical trial is currently recruiting to evaluate physiological and psychological factors involved in exercise intolerance in patients wi…
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CLINICAL_TRIALS research · 2025-10-02Clinical Trial Update: Body Composition in Pediatric Patients With Thalassemia Major
This document notes a clinical trial update for a pediatric study on body composition in patients with beta-thalassemia major. It is sponsor…
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FDA approval · 2025-09-08FDA Approved: DEFERASIROX — Original Application (Aurobindo Pharma Limited)
The FDA approved an original ANDA for deferasirox (generic) manufactured by Aurobindo Pharma Limited, expanding access to iron chelation the…
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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 research · 2025-09-02Clinical Trial Update: A Clinical Study of Ultra-transplantation for the Treatment of Major Thalassemia Scheme [NA]
This clinical trial update indicates a study evaluating ultra-transplantation for the treatment of major beta-thalassemia, and the study is …
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FDA approval · 2025-08-15FDA Approved: DEFERIPRONE — Supplemental Application (Hikma Pharmaceuticals USA Inc.)
FDA approved a supplemental application for Deferiprone submitted by Hikma Pharmaceuticals USA Inc.
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CLINICAL_TRIALS research · 2025-08-12Clinical Trial Update: T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias [Phase 1, Phase 2]
This clinical trials update notes that a T-cell depleted alternative donor bone marrow transplant for sickle cell disease and other anemias …
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CLINICAL_TRIALS research · 2025-08-08Clinical Trial Update: Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major [NA]
This clinical trial update reports ongoing recruitment for a study evaluating incentive respiratory training with an incentive spirometer to…
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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 research · 2025-06-04Clinical Trial Update: A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
This is a real-world, Taiwan-based study evaluating luspatercept for beta-thalassemia major, currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-05-30Clinical Trial Update: Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia [Phase 2]
Phase 2 clinical trial evaluating different doses of thalidomide in transfusion-dependent beta-thalassemia is currently recruiting.
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CLINICAL_TRIALS research · 2025-05-20Clinical Trial Update: The Effect of Laughter Yoga in Beta Thalassaemia Major Patients: a Randomised Controlled Trial [NA]
An update on a randomized controlled trial evaluating laughter yoga in beta-thalassemia major patients. The study status is not yet recruiti…
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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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FDA approval · 2025-05-08FDA Approved: DEFEROXAMINE MESYLATE — Supplemental Application (Hospira, Inc.)
FDA approved a supplemental application for Deferoxamine Mesylate (Hospira, Inc.), a generic iron chelator.
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FDA approval · 2025-03-26FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
The FDA approved a supplemental application for Ferriprox (deferiprone) filed by Chiesi USA, Inc.
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FDA approval · 2025-03-26FDA Approved: DEFERASIROX — Supplemental Application (Amneal Pharmaceuticals NY LLC)
The FDA approved a supplemental application for Deferasirox submitted by Amneal Pharmaceuticals NY LLC. The supplemental filing updates the …
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CLINICAL_TRIALS other · 2025-02-25Clinical Trial Update: Pilot Study PBSCT with TCRab Depletion for Hemoglobinopathies [NA]
Pilot study of peripheral blood stem cell transplant with TCRαβ depletion for hemoglobinopathies; the trial is currently active but not recr…
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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…