Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study

Recruiting now · Phase 1/Phase 2

Conditions studied: Sickle Cell Disease, Sickle Cell Anemia in Children

In brief

NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study. All children enrolled in NOHARM received hydroxyurea treatment at a fixed daily dose of 20 mg/kg/day. This dose was selected as a likely safe dose, but does not escalate hydroxyurea to maximum tolerated dose "MTD" as is commonly done in the US. Without this information, we cannot know whether hydroxyurea treatment at the MTD would be feasible (since it requires closer monitoring to avoid hematological toxicities), safe (since adverse events may be greater with MTD, risk of malaria may be altered by MTD, and risk of infections as a result of neutropenia could also be greater with MTD) or beneficial (MTD is associated with higher hemoglobin and fetal hemoglobin concentration).

Key facts

Study ID
NCT07708714
Run by
Children's Hospital Medical Center, Cincinnati
People needed
250
Starts
2026-05-13
Expected to finish
2032-11-30
Last updated by the study team
2026-07-16

Who can join

Age: 11 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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