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β -Thalassemia
Clinical trial pipeline · Data from ClinicalTrials.gov
See which β -Thalassemia trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which β -Thalassemia trials may be worth asking aboutNorth America
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The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion…
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusio…
Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia. The study will be condu…
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in part…
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001…
The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion…