Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

  • STATUS
    Recruiting
  • End date
    Jun 30, 2022
  • participants needed
    65
  • sponsor
    St. Jude Children's Research Hospital
Updated on 19 March 2021
body mass index
anemia
hydroxyurea
schwartz
hydroxyurea oral capsule

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.

Description

HOPE18 will be an open label, 2-arm study of HU disposition in 48 children with SCD. In Arm 1, n=18 infants ages 9 months to 2 years will be administered an extemporaneous oral liquid formulation of HU on a single occasion followed by PK sampling. The dose administered will be ~20 mg/kg/day or the infant's usual daily dose. In Arm 2, n=30 children who range in age from 2 to 18 years will be administered HU, both a sprinkle formulation and capsules (Droxia 200 mg), on two separate occasions separated by at least 1 day but no more than 30 days in a randomized, crossover fashion. The doses of HU on each occasion will be rounded to the nearest 200 mg and will not exceed 35 mg/kg or 2000 mg. We hypothesize that the PK profile of the sprinkle formulation will not differ significantly from the PK profile of Droxia capsules in children and adolescents ages 2 - 18 years of age. Participants in both arms will be followed up to 30 days from receiving last HU dose.

Details
Condition Thalassemia, SICKLE CELL ANEMIA, Sickle Cell Disease
Treatment hydroxyurea, Hydroxyurea Oral Capsule
Clinical Study IdentifierNCT03825341
SponsorSt. Jude Children's Research Hospital
Last Modified on19 March 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Participants will be eligible for this study if only if all of the following
inclusion criteria apply
Laboratory (i.e. electrophoretic, chromatographic or DNA) confirmation of HbSS or HbS0thalassemia
Participants may or may not be currently receiving HU. If participants are taking HU, then their most recent dose must be 24 hours prior to the start of the study
Participant is in the "well" state (defined by 2 weeks since the last SCD-related complication)
Clinical evidence of normal gastrointestinal function and structure
No clinical evidence of hepatic compromise, including transaminases < 3 times the upper limit of normal
Estimated glomerular filtration rate (Schwartz equation) > 70 ml/min/1.73m2
Body mass index (BMI) 5th and 95th percentile as per CDC growth charts
In addition
For the Pharmacokinetic Study (Arm 1)
Age 9 months and < 2 years
Able to consume a minimum of 30 ml of water following ingestion of the study article
For the Bioavailability Study (Arm 2)
Age 2 years and 18 years
Weight of 10 kg
Females of child-bearing potential must have a negative pregnancy test prior to dosing and be willing to practice appropriate contraceptive measures, including abstinence, from the time of the initial pregnancy testing through the remainder of the study (30 days after last administration of investigational agents)
Males of child-bearing potential must be willing to practice appropriate contraceptive measures, including abstinence, during study participation (30 days after last administration of investigational agents)
Able to ingest both sprinkles and capsule study articles and consume a minimum of 30 ml of water following ingestion of each agent

Exclusion Criteria

Chronic transfusion therapy, or transfused within 3 months of study participation
Known renal impairment (creatinine >1.5x the upper limit of normal for age)
Known hepatic impairment or Grade 2 or higher transaminases and bilirubin levels
Diagnoses other than sickle cell anemia or sickle beta-zero thalassemia (i.e., other sickle cell variants or sickle/ hereditary persistence of fetal hemoglobin)
Blood count parameters as follows: hemoglobin <6.0 gm/dL, absolute reticulocyte count <80,000/mm3, absolute neutrophil count <1000/mm3, or platelet count <80,000/mm3
The participant has used opiates, H2 blockers, proton pump inhibitors, antacids, other GI motility agents or any other medication that, in the opinion of the investigator, will interfere with the study procedures or affect the interpretation of the results of the study for 3 days prior to the first dose of study
Participants taking antiretroviral drugs (including didanosine and stavudine) due to increased risk of toxicity with concomitant use
Participation in another clinical intervention trial utilizing an IND/IDE agent, but can participate in HUGKISS since same drug agent
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