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

Sponsor
Children's Hospital Medical Center, Cincinnati
Study ID
NCT07708714
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
11 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • hydroxycarbamide — DRUG
    Hydroxyurea at optimized dose

Study Details

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 Dates

First listed
Jul 16, 2026
Start date
May 13, 2026
Status verified
Jul 2026
Primary completion
Nov 30, 2030
Completion
Nov 30, 2032

Study Design

Enrollment
250 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Hydroxyurea: Original NOHARM Cohort
    Original Cohort, continue hydroxyurea at optimized dose
  • Experimental: Hydroxyurea: New Comparator Cohort
    New, untreated cohort, initiate and optimize hydroxyurea dose

Primary Outcome Measure

Composite measure of organ damage [ Time Frame: From enrollment, after 24 months and again after 48 months of study treatment. ]

Central Contacts

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