Personalized hydroxyurea dosing using blood-level guidance for children with sickle cell anemia (ENHANCE)
Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)
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 |
| Enrollment | 50 (estimated) |
| Ages | 6 Months and up |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT07177300 on ClinicalTrials.gov |
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.
Who should consider this trial
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.
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.
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 performed: 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.
Eligibility criteria
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 this trial is running
Cincinnati, Ohio
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.