Thalassemia
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CLINICAL_TRIALS research · 2024-12-06Clinical Trial Update: Thalassaemia Severity
An update on a clinical trial investigating thalassemia severity, currently not yet recruiting.
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CLINICAL_TRIALS research · 2024-11-29Clinical Trial Update: the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia [EARLY_Phase 1]
An ongoing early-phase trial evaluating the safety and efficacy of HGI-002 Injection in transfusion-dependent α-thalassemia, which is curren…
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CLINICAL_TRIALS trial_result · 2024-10-30Clinical Trial Update: Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
Completed clinical trial assessing cerebrovascular reserve and white matter disease in patients with chronic anemia due to thalassemia or si…
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CLINICAL_TRIALS trial_result · 2024-10-28Clinical Trial Update: Pharmacokinetics of Oral Hydroxyurea Solution [Phase 1, Phase 2]
Completed Phase 1/2 pharmacokinetics study of an oral hydroxyurea solution in sickle cell disease and related hemoglobin disorders, with res…
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Psychiatric Problems in Thalassemic Children
Clinical trial investigating psychiatric problems in children with thalassemia, currently not yet recruiting.
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CLINICAL_TRIALS trial_result · 2024-07-23Clinical Trial Update: Pharmacotherapy Prescribing Pattern and the Adherence Level to Iron Chelation Therapy in Thalassemia
This completed clinical trial update analyzes prescribing patterns and patient adherence to iron chelation therapy in individuals with thala…
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CLINICAL_TRIALS trial_result · 2024-07-18Clinical Trial Update: Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004) [Phase 2]
Extension phase 2 study update evaluating the long-term safety and efficacy of luspatercept in β-thalassemia patients previously enrolled in…
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CLINICAL_TRIALS trial_result · 2024-07-12Clinical Trial Update: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases [Phase 2]
This Phase 2 clinical trial update reports the termination of a haploidentical hematopoietic stem cell transplantation study using reduced-i…
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CLINICAL_TRIALS trial_result · 2024-05-17Clinical Trial Update: Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
A completed clinical trial evaluating strategies to improve red blood cell transfusions for chronically transfused patients with sickle cell…
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CLINICAL_TRIALS trial_result · 2023-11-22Clinical Trial Update: Long-term Clinical Study of CN128 in Thalassemia Patients [Phase 2]
Phase 2 long-term clinical study update for CN128 in thalassemia and iron overload conditions has completed.
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FDA approval · 2023-09-26FDA Approved: DEFERASIROX — Supplemental Application (Bionpharma Inc.)
FDA approved a supplemental application for Deferasirox from Bionpharma Inc. under an ANDA, authorizing an additional regulatory action for …
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CLINICAL_TRIALS trial_result · 2023-09-26Clinical Trial Update: Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study [Phase 1, Phase 2]
Long-term follow-up update from BP-004 study (Phase 1/Phase 2) by Bellicum Pharmaceuticals; study status TERMINATED with results posted in 2…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
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CLINICAL_TRIALS trial_result · 2023-01-04Clinical Trial Update: Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant [Phase 1, Phase 2]
A phase 1/2 safety study evaluating gene-modified donor T-cells after TCRαβ+ depleted stem cell transplant was terminated; results were post…
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CLINICAL_TRIALS trial_result · 2022-04-27Clinical Trial Update: Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies [Phase 1, Phase 2]
Phase 1/2 trial investigating NiCord allogeneic stem and progenitor cells for hemoglobinopathies (sickle cell disease and thalassemia).
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CLINICAL_TRIALS trial_result · 2022-03-25Clinical Trial Update: Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia [Phase 2, Phase 3]
A completed Phase 2/Phase 3 trial evaluating the efficacy and safety of low-dose thalidomide in transfusion-dependent thalassemia.
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CLINICAL_TRIALS trial_result · 2022-03-18Clinical Trial Update: Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere [Phase 2]
Phase 2 trial update on hydroxyurea therapy aimed at optimizing access in pediatric patients; study terminated with results posted.
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FDA approval · 2021-11-30FDA Approved: FERRIPROX — Supplemental Application (Chiesi USA, Inc.)
FDA approved a supplemental application for Ferriprox (deferiprone) submitted by Chiesi USA, Inc.
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CLINICAL_TRIALS trial_result · 2021-11-24Clinical Trial Update: Clinical Experience of Thalidomide in Thalassemic Patients [Phase 2, Phase 3]
Clinical trial update reporting experience with thalidomide in thalassemia patients (Phase 2 and Phase 3; completed).
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CLINICAL_TRIALS trial_result · 2021-11-18Clinical Trial Update: A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia [Phase 2]
Phase 2 trial assessing efficacy and safety of luspatercept in adults with non transfusion dependent beta-thalassemia has completed with res…
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CLINICAL_TRIALS trial_result · 2021-11-15Clinical Trial Update: A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia [Phase 2]
Phase 2 clinical trial evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of AG-348 (mitapivat) in adults with non-tran…
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CLINICAL_TRIALS trial_result · 2021-08-12Clinical Trial Update: Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload [Phase 2]
Phase 2 trial update on combined chelation therapy for transfusion-dependent thalassemia with iron overload; study completed with posted res…
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EMA safety_alert · 2021-02-17Precautionary marketing suspension of thalassaemia medicine Zynteglo
EMA announces a precautionary marketing suspension of Zynteglo, a gene therapy for thalassemia, pending further investigation.
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CLINICAL_TRIALS trial_result · 2021-02-02Clinical Trial Update: L-Glutamine Therapy for Sickle Cell Anemia [Phase 2]
Phase 2 clinical trial of L-Glutamine therapy for sickle cell anemia has been terminated; results posted in 2021.
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CLINICAL_TRIALS trial_result · 2021-01-29Clinical Trial Update: L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia [Phase 2]
Phase 2 clinical trial of L-Glutamine therapy for sickle cell anemia and sickle beta-zero thalassemia completed with results posted.