Beta-thalassemia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2023-12-01Clinical Trial Update: Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients [Phase 2, Phase 3]
A phase 2/3 clinical trial evaluating a thalidomide and hydroxyurea combination in patients with beta-thalassemia has completed enrollment a…
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CLINICAL_TRIALS trial_result · 2023-06-18Clinical Trial Update: A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype [Phase 3]
Phase 3 trial evaluating LentiGlobin BB305 for transfusion-dependent beta-thalassemia with non β0/β0 genotype completed; results posted in 2…
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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 · 2022-09-21Clinical Trial Update: Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone [Phase 2]
Phase 2 clinical trial update on chelation therapy using Pyridoxal Isonicotinoyl Hydrazone for iron overload in anemia due to beta-thalassem…
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CLINICAL_TRIALS trial_result · 2022-06-30Clinical Trial Update: A Study of IMR-687 in Subjects With Beta Thalassemia [Phase 2]
Phase 2 study of IMR-687 in Beta-thalassemia was terminated; results have been posted in 2022.
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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 · 2021-11-12Clinical Trial Update: Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP) [Phase 2]
Phase 2 clinical trial update investigating sirolimus for beta-thalassemia in transfusion-dependent patients.
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CLINICAL_TRIALS trial_result · 2021-07-16Clinical Trial Update: Safety Study for Beta Thalassemia Subjects on PTG-300 [Phase 2]
Phase 2 safety study for PTG-300 in beta-thalassemia subjects completed.
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CLINICAL_TRIALS trial_result · 2021-07-16Clinical Trial Update: Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia [Phase 2]
Phase 2 trial update for PTG-300 in beta-thalassemia subjects with chronic anemia (both transfusion-dependent and non-transfusion-dependent)…
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CLINICAL_TRIALS trial_result · 2021-06-07Clinical Trial Update: Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment [Phase 2]
Phase 2 trial update on cardiac T2* in beta-thalassemia patients treated with deferasirox; results posted after completion.
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CLINICAL_TRIALS trial_result · 2020-05-01Clinical Trial Update: Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO [NA]
Clinical trial update for allogeneic stem cell transplantation in severe beta-thalassemia variants; the study is terminated with results pos…
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CLINICAL_TRIALS trial_result · 2020-03-10Clinical Trial Update: A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease [Phase 1, Phase 2]
A completed early-phase study evaluating the safety and efficacy of LentiGlobin BB305 in transfusion-dependent beta-thalassemia and sickle c…
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CLINICAL_TRIALS trial_result · 2020-01-27Clinical Trial Update: An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia [Phase 3]
Phase 3 trial comparing luspatercept to placebo in adults with beta-thalassemia requiring regular red blood cell transfusions; results poste…
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CLINICAL_TRIALS trial_result · 2019-10-23Clinical Trial Update: Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload [Phase 2]
Phase 2 trial assessing safety and efficacy of deferasirox combined with desferoxamine in transfusion-dependent beta-thalassemia patients wi…
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CLINICAL_TRIALS trial_result · 2019-06-28Clinical Trial Update: Gene Therapy for Transfusion Dependent Beta-thalassemia [Phase 1, Phase 2]
Phase 1/2 clinical trial update for a gene therapy targeting transfusion-dependent beta-thalassemia.
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CLINICAL_TRIALS trial_result · 2019-05-23Clinical Trial Update: Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus [Phase 4]
Phase 4 clinical trial evaluating hyalurnic acid levels in β-thalassemic children treated for hepatitis C.
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CLINICAL_TRIALS trial_result · 2018-11-02Clinical Trial Update: the Efficacy and Safety of Sugammadex in Children 0-2 Years Old [Phase 2, Phase 3]
A Phase 2/Phase 3 clinical trial evaluating the efficacy and safety of sugammadex in children aged 0-2 years for anesthesia-related use, wit…
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CLINICAL_TRIALS trial_result · 2018-10-05Clinical Trial Update: A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia [Phase 2]
Phase 2 trial update of Bitopertin (RO4917838) in adults with non-transfusion-dependent beta-thalassemia has completed.
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CLINICAL_TRIALS trial_result · 2018-08-27Clinical Trial Update: L-arginine Versus Sildenafil in Children With Beta Thalassemia Associated With Pulmonary Hypertension [Phase 4]
Phase 4 trial comparing L-arginine to sildenafil in children with beta thalassemia and pulmonary hypertension.
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CLINICAL_TRIALS trial_result · 2018-08-21Clinical Trial Update: CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant [Phase 2]
Phase 2 clinical trial update on CD34+ selection for partially matched family or matched unrelated adult donor transplants in leukemia and l…
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CLINICAL_TRIALS trial_result · 2018-05-25Clinical Trial Update: A Study of EPEG in Beta Thalassemia Patients [Phase 1]
Phase 1 study of EPEG in beta-thalassemia patients has completed; results are not described in this update.
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CLINICAL_TRIALS trial_result · 2017-11-30Clinical Trial Update: Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector [NA]
This document is a clinical trial update for a beta-thalassemia gene therapy using a self-inactivating lentiviral vector, with the trial sta…
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CLINICAL_TRIALS trial_result · 2017-09-20Clinical Trial Update: The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan [Phase 2]
A Phase 2 clinical trial evaluating the efficacy and safety of hydroxyurea in managing beta-thalassemia in Karachi, Pakistan.
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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.