Therapeutic Response Evaluation and Adherence Trial (TREAT): A Prospective Study of Hydroxyurea for Children With Sickle Cell Anemia

About the study

The primary objectives of this prospective study of hydroxyurea for children with sickle cell anemia are 1) Develop and prospectively evaluate a population pharmacokinetic/pharmacodynamics model to predict the maximum tolerated dose (MTD); 2) Identify urine biomarkers of hydroxyurea adherence using a novel metabolomics approach; 3) Identify pharmacogenomics modifiers of hydroxyurea MTD; and 4) Longitudinal monitoring of the effect of hydroxyurea upon organ function and quality of life.

Study point of contact

Adriane Hausfeld, RN,BSN,CCRP
(513)803-3236
adriane.hausfeld@cchmc.org
Amanda Pfeiffer, LPC, CCRP
513-803-4977
amanda.pfeiffer@cchmc.org

Locations

1 United States site

Age

6 Months - 21 Years

Genotypes

HbSS

Phase

Not Applicable

Study type

Interventional

Gender

All

Interventions

Drug

participation requirements

Diagnosis of sickle cell anemia (HbSS or Hbβ0-thalassemia)
Age 6 months to 21 years at the time of enrollment
Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy, including patients who are transitioning from chronic transfusions to hydroxyurea therapy

participation restrictions

1. Family unwillingness to sign informed consent or comply with study treatments

Locations

  • Cincinnati, Ohio, United States, Cincinnati Children's Hospital Medical Center, 45229 [Recruiting]
Last updated 2021-12-07