Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere
Status:
Recruiting
Trial end date:
2022-06-30
Target enrollment:
Participant gender:
Summary
Primary Objective
1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years)
2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children
and adolescents (≥2 to 18 years).
Secondary Objective:
Compare PK parameters in infants versus older children on this study and those from our
previous "Pharmacokinetics and Bioavailability of a Liquid Formulation of Hydroxyurea in
Pediatric Patients with Sickle Cell Anemia" (NCT01506544) trial.
Exploratory Objectives:
Capture information regarding the taste of HU sprinkles using palatability questionnaire.
This trial is an open label, single center assessment of the pharmacokinetics of two
formulations of hydroxyurea (HU) designed to (1) determine the pharmacokinetic profile of a
liquid formulation in infants and to (2) determine the bioavailability of "sprinkles", a
novel method of administration for older children. The study aims to generate data to
facilitate FDA approval for HU in children and potentially validate a new mode of
administration ("sprinkles") that will optimize access and adherence for children in the US
and globally.