New pill could boost hemoglobin in kids with thalassemia

NCT ID NCT07517133

First seen Apr 22, 2026 · Last updated May 15, 2026 · Updated 5 times

Summary

This study tests whether the drug mitapivat can raise hemoglobin levels in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo daily for 24 weeks. The main goal is to see if more children on mitapivat achieve a meaningful increase in hemoglobin compared to those on placebo.

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