Evaluating hydroxyurea treatment for children with sickle cell anemia
Official title: Therapeutic Response Evaluation and Adherence Trial (TREAT): A Prospective Study of Hydroxyurea for Children With Sickle Cell Anemia
Run by Children's Hospital Medical Center, Cincinnati · NCT02286154 · Data updated 2026-05-14
This study is no longer in our active list. It may have stopped recruiting or finished. The details below are as of 2026-05-14 and are kept for reference. Check ClinicalTrials.gov for its current status, or see similar studies below.
This study tests how well hydroxyurea works for young children with sickle cell anemia and looks at how it affects their health and quality of life over time.
Quick facts
| Study type | Interventional |
|---|---|
| Ages | 6 Months to 21 Years |
| Sex | All |
| Enrollment | 150 (estimated) |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT02286154 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include children aged 6 months to 21 years diagnosed with sickle cell anemia who are beginning hydroxyurea therapy.
✕Probably not a fit
Patients who are not willing to comply with study treatments or sign informed consent may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This prospective study aims to evaluate the pharmacokinetics and pharmacodynamics of hydroxyurea in children with sickle cell anemia. It will develop a model to predict the maximum tolerated dose and identify urine biomarkers for adherence to treatment using metabolomics. Additionally, the study will explore pharmacogenomic factors that may influence the effectiveness of hydroxyurea and monitor its impact on organ function and quality of life over time. The study will include two groups of patients, focusing on young infants who are starting hydroxyurea therapy.
Why it matters
Potential benefit: If successful, this study could lead to improved dosing strategies and adherence monitoring for hydroxyurea, enhancing treatment outcomes for children with sickle cell anemia.
How similar studies have gone: Other studies have shown positive outcomes with hydroxyurea treatment in sickle cell anemia, but this specific approach utilizing pharmacokinetic modeling and adherence biomarkers is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Diagnosis of sickle cell anemia (HbSS or Hbβ0-thalassemia) 2. Age 6 months to 21 years at the time of enrollment 3. Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy, including patients who are transitioning from chronic transfusions to hydroxyurea therapy Exclusion Criteria: 1. Family unwillingness to sign informed consent or comply with study treatments
Where it's running
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States Recruiting
Study contacts
- Principal investigator: Charles Quinn, MD, MS — Children's Hospital Medical Center, Cincinnati
- Study coordinator: Amanda Pfeiffer, LPC, CCRP
- Email: amanda.pfeiffer@cchmc.org
- Phone: 513-803-4977
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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.