Optimizing Hydroxyurea in Children with Sickle Cell in Africa
Official title: Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study
Run by Children's Hospital Medical Center, Cincinnati · NCT07708714 · Data updated 2026-11-12
This study tests whether raising hydroxyurea to a maximum tolerated dose is safe and beneficial for Ugandan children with sickle cell anemia already taking a fixed daily dose.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 11 Years to 18 Years |
| Sex | All |
| Enrollment | 250 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Kampala) |
| Status | Recruiting |
| Started | May 13, 2026 |
| Main results expected | November 30, 2030 |
| Study ends | November 30, 2032 |
| Registry record updated | July 16, 2026 |
| Trial ID | NCT07708714 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Adolescents aged 11-18 with confirmed sickle cell anemia, enrolled in the NOHARM MTD study or hydroxyurea-naïve controls in Kampala, Uganda.
✕Probably not a fit
Patients who do not respond to higher hydroxyurea doses or who experience intolerable side effects may not benefit from dose escalation.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This interventional study in Uganda aims to determine the long-term safety and potential benefits of dose-escalated hydroxyurea for children with sickle cell anemia. It compares a maximum tolerated dose strategy to a fixed 20 mg/kg/day regimen, while exploring pharmacokinetics, pharmacodynamics, and underlying biology through genomic and multiomics analyses. Participants are adolescents already enrolled in a prior NOHARM MTD study or hydroxyurea-naïve controls, and will undergo quarterly visits to monitor health and study outcomes. The trial is led by Cincinnati Children's Hospital Medical Center with collaboration from Makerere University, Indiana University, and the Indian Institute of Science, at Mulago Hospital Sickle Cell Clinic in Kampala. The investigation seeks to clarify long-term organ safety, dosing strategies, and potential biomarkers that predict response to hydroxyurea in this population.
Why it matters
Potential benefit: If successful, participants could receive a more effective hydroxyurea dosing strategy with better pain control and biological markers, potentially improving long-term health outcomes.
How similar studies have gone: Prior studies have explored MTD approaches in other populations, but this African cohort with pharmacogenomic and multiomics analyses represents a novel extension.
Who can join: the full rules
Show full inclusion / exclusion criteria
Adolescents with confirmed SCA who are currently enrolled in the NOHARM MTD Study of hydroxyurea at the Mulago Hospital Sickle Cell Clinic (MHSCC), will be eligible for the NOHARM MTD LT version 2.0 extension study after completing re-consent. An age-matched untreated (hydroxyurea-naïve) group of adolescents will also be enrolled as a Comparator Cohort. This will comprise 75 children from Kampala and surrounding districts, ages 11 - 18 years of age, with confirmed SCA.
Where it's running
-
Mulago Hospital Sickle Cell Clinic
Kampala, Uganda Recruiting
Study contacts
- Principal investigator: Russell E. Ware, MD, PhD — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Teresa Latham, DrPH, PhD
- Email: teresa.latham@cchmc.org
- Phone: 5138037922
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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This study is testing if hydroxyurea can safely help children with sickle cell anemia in Africa feel better and improve their treatment options.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.