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Phase 3 Completed N=208 Randomized Quadruple-blind Treatment

Novel Use Of Hydroxyurea in an African Region With Malaria

Source: ClinicalTrials.gov NCT01976416 ↗
Enrolled (actual)
208
Serious AEs
5.8%
Results posted
Oct 2018
Primary outcomePrimary: Number of Malaria Episodes — 5; 7 malaria episodes
◆ Published Evidence
Highly cited
150citations · ~17 / year
Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM): a trial for children with sickle cell anemia.
Blood · 2017 · Open access · High-confidence link

Summary

Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in malaria endemic regions of sub-Saharan Africa, the areas with the most children with SCA. The primary goal of this study is to investigate the safety and efficacy of hydroxyurea for children with SCA in a malaria endemic region within sub-Saharan Africa.

Linked Publications (5)

  • Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM): a trial for children with sickle cell anemia.
    Blood · 2017 · 150 citations · Open access · High-confidence link
  • Perceived benefits and risks of participation in a clinical trial for Ugandan children with sickle cell anemia.
    Pediatric blood & cancer · 2020 · 34 citations · Likely link
  • Novel Use of Hydroxyurea in an African Region With Malaria: Protocol for a Randomized Controlled Clinical Trial.
    JMIR research protocols · 2016 · 24 citations · Open access · Likely link
  • Hydroxyurea pharmacokinetics in children with sickle cell anemia across different global populations.
    Blood advances · 2026 · 4 citations · Open access · Likely link
  • Angiopoietin-2 is associated with sickle cell complications, including stroke risk, and decreases with hydroxyurea therapy.
    Blood vessels, thrombosis & hemostasis · 2024 · 3 citations · Open access · Likely link

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Malaria Episodes
5; 7

Eligibility Criteria

Inclusion Criteria

  • Pediatric subjects with documented sickle cell anemia (HbSS supported by hemoglobin electrophoresis or by peripheral blood smear showing sickled red blood cells)
  • Age range of 1.00-3.99 years, inclusive, at the time of enrollment
  • Weight at least 5.0 kg at the time of enrollment
  • Willingness to comply with all study-related treatments, evaluations, and follow up

Exclusion Criteria

  • Known chronic medical condition (e.g., HIV, malignancy, active clinical tuberculosis)
  • Severe malnutrition determined by impaired growth parameters as defined by WHO (weight for length/height or weight-for-length/height > 3 z-scores below the median WHO growth standards)
  • Pre-existing severe hematological toxicity:
  • Hb 2 times the upper limit of normal for age
  • Blood transfusion within 30 days prior to enrollment
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01976416) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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