Thalassemia
📧 Sign up to get email alerts when something new happens for Thalassemia.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: CINC424A2X01B Rollover Protocol [Phase 4]
Phase 4 rollover protocol for CINC424A2X01B is active but not recruiting, spanning Primary Myelofibrosis, Polycythemia Vera, Graft-versus-ho…
-
CLINICAL_TRIALS trial_result · 2026-02-05Clinical Trial Update: Multicenter Observational Study on Myocardial Iron Overload in 3 Multitransfused Populations
A multicenter observational study assessing myocardial iron overload in three transfused populations has completed, focusing on patients wit…
-
CLINICAL_TRIALS trial_result · 2026-01-02Clinical Trial Update: A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants [Phase 1]
Phase 1 study of etavopivat assessing heart rhythm in healthy participants has completed; the trial also lists sickle cell disease and thala…
-
CLINICAL_TRIALS trial_result · 2025-12-29Clinical Trial Update: A Research Study Looking at a Single Dose of Etavopivat in Healthy Chinese Participants [Phase 1]
Phase 1 study evaluating a single dose of etavopivat in healthy Chinese participants has completed.
-
CLINICAL_TRIALS trial_result · 2025-12-17Clinical Trial Update: A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia [Phase 2, Phase 3]
A Phase 2/3 safety and efficacy study of CTX001 in transfusion-dependent beta-thalassemia has completed.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2025-12-16Clinical Trial Update: A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease [Phase 2]
A Phase 2 study of etavopivat in patients with sickle cell disease and thalassemia has completed.
-
CLINICAL_TRIALS trial_result · 2025-10-10Clinical Trial Update: A Research Study of the Effect of Food on Etavopivat in Healthy Participants [Phase 1]
Phase 1 study assessing the effect of food on Etavopivat in healthy participants has been completed; the update focuses on pharmacokinetic i…
-
CLINICAL_TRIALS trial_result · 2025-09-25Clinical Trial Update: A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants [Phase 1]
Completed Phase 1 study evaluating the effect of etavopivat on other drugs in healthy participants, sponsored by Novo Nordisk A/S.
-
CLINICAL_TRIALS trial_result · 2025-03-21Clinical Trial Update: Pregnancy and Childbirth in Women with Thalassemia Yesterday and Today
A completed clinical trial update examining pregnancy and childbirth in women with thalassemia, comparing past and present outcomes.
-
CLINICAL_TRIALS trial_result · 2025-03-21Clinical Trial Update: Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients [NA]
A completed clinical trial evaluating the safety and efficacy of thalidomide in multi-transfused thalassemia patients.
-
CLINICAL_TRIALS trial_result · 2025-03-14Clinical Trial Update: Long-term Clinical Study of CN128 in Thalassemia With Sever Liver Iron Overloaded Patients [Phase 2]
An update on the Phase 2 long-term study of CN128 in patients with thalassemia and severe liver iron overload, which has completed.
-
CLINICAL_TRIALS trial_result · 2025-03-05Clinical Trial Update: Inhibitory Effect of a Polyphenol Supplement on Dietary Iron Absorption in Adults with Thalassemia [NA]
A completed clinical trial evaluating whether a polyphenol supplement inhibits dietary iron absorption in adults with thalassemia and iron o…
-
CLINICAL_TRIALS trial_result · 2024-12-19Clinical Trial Update: Haploidentical Hematopoietic Stem Cell Transplantation [Phase 1]
Phase 1 trial update reports the completion of a haploidentical hematopoietic stem cell transplantation study led by Catherine Bollard, inve…
-
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…
-
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…
-
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…
-
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…
-
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…
-
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…
-
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.
-
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…
-
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…
-
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…
-
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).
-
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.