Evaluating hydroxyurea treatment for children with sickle cell anemia
Therapeutic Response Evaluation and Adherence Trial (TREAT): A Prospective Study of Hydroxyurea for Children With Sickle Cell Anemia
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
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | 6 Months to 21 Years |
| Sex | All |
| Sponsor | Children's Hospital Medical Center, Cincinnati Academic / other |
| Locations | 1 site (Cincinnati, Ohio) |
| Trial ID | NCT02286154 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include children aged 6 months to 21 years diagnosed with sickle cell anemia who are beginning hydroxyurea therapy.
Not a fit: Patients who are not willing to comply with study treatments or sign informed consent may not benefit from this study.
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 performed: 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.
Eligibility criteria
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 this trial is running
Cincinnati, Ohio
- 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
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.