Personalized hydroxyurea dosing using blood-level guidance for children with sickle cell anemia (ENHANCE)
Official title: Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
Run by Children's Hospital Medical Center, Cincinnati · NCT07177300 · Data updated 2026-11-12
This trial will test whether starting hydroxyurea based on how each child absorbs the drug (a PK-guided dose) helps babies and children with sickle cell anemia get higher fetal hemoglobin and better organ protection over time.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Ages | 6 Months and up |
| Sex | All |
| Enrollment | 50 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Status | Recruiting |
| Started | December 19, 2024 |
| Main results expected | December 2026 |
| Study ends | December 2028 |
| Registry record updated | September 16, 2025 |
| Trial ID | NCT07177300 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged at least 6 months with sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0) whose family and provider have decided to start hydroxyurea and who are not on regular transfusion programs.
✕Probably not a fit
Patients on regular scheduled blood transfusions or those with other sickle genotypes such as HbSC or HbSβ+ would not be expected to benefit from this protocol and are excluded.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
ENHANCE will enroll infants and children with HbSS or HbSβ0 who are starting hydroxyurea and use a pharmacokinetic (PK)-guided strategy to choose an individualized starting dose and escalate to a maximum tolerated dose (MTD) through long-term follow-up to age 15. The protocol combines clinical monitoring with novel functional organ assessments (brain, heart, kidneys, spleen, eyes), growth and pubertal hormone measurements, and state-of-the-art laboratory assays including single-cell HbF quantitation and single-cell multiome analyses in erythroblasts. Serial genomic DNA sampling will allow monitoring for clonal hematopoiesis and potential leukemic risk, while simplified PK-guided dosing aims to achieve sustained ≥30% HbF with near-pancellular distribution. All participants receive oral PK-optimized hydroxyurea at MTD with systematic long-term monitoring at Cincinnati Children’s Hospital Medical Center.
Why it matters
Potential benefit: If successful, this approach could get children to optimal hydroxyurea doses faster, increase fetal hemoglobin levels, and reduce long-term organ injury and complications.
How similar studies have gone: Hydroxyurea is a well-established therapy that improves outcomes in sickle cell disease, and smaller trials and pilot work have shown promise for individualized or PK-guided dosing, but the comprehensive long-term organ-protection and single-cell genomic approaches here are novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0) * Age 6 months at the time of enrollment * Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy Exclusion Criteria: * Current treatment with regularly scheduled blood transfusions * Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+)
Where it's running
-
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting
Study contacts
- Principal investigator: Charles T. Quinn, Professor of Pediatrics, M.D., M.S. — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Wendi L. Long, Sr. Regulatory Specialist, BS, CCRC
- Email: wendi.long@cchmc.org
- Phone: 513-803-3064
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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.