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Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03825341
Recruitment Status : Terminated (The study terminated early due to challenges with recruitment during the COVID pandemic.)
First Posted : January 31, 2019
Results First Posted : March 18, 2022
Last Update Posted : March 18, 2022
Sponsor:
Information provided by (Responsible Party):
St. Jude Children's Research Hospital

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Crossover Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Sickle Cell Disease
Thalassemia
Interventions Drug: Hydroxyurea
Drug: Hydroxyurea Oral Capsule
Enrollment 1
Recruitment Details 1 participant was recruited between March 2019 and Jan. 2022. The participant was randomized but did not receive treatment.
Pre-assignment Details  
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Period Title: Overall Study
Started 0 1
Completed 0 0
Not Completed 0 1
Reason Not Completed
Withdrawal by Subject             0             1
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule Total
Hide Arm/Group Description

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.

 Total of all reporting groups
Overall Number of Baseline Participants 0 1 1
Hide Baseline Analysis Population Description
No enrollments on Arm 1.
Age, Categorical   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants
<=18 years 0
Between 18 and 65 years 0
>=65 years 0
[1]
Measure Analysis Population Description: Baseline data not entered for the 1 enrollment due to confidentiality concerns.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants
Female 0
Male 0
[1]
Measure Analysis Population Description: Baseline data not entered for the 1 enrollment due to confidentiality concerns.
Ethnicity (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants
Hispanic or Latino 0
Not Hispanic or Latino 0
Unknown or Not Reported 0
[1]
Measure Analysis Population Description: Baseline data not entered for the 1 enrollment due to confidentiality concerns.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants 0 participants 0 participants
American Indian or Alaska Native 0
Asian 0
Native Hawaiian or Other Pacific Islander 0
Black or African American 0
White 0
More than one race 0
Unknown or Not Reported 0
[1]
Measure Analysis Population Description: Baseline data not entered for the 1 enrollment due to confidentiality concerns.
1.Primary Outcome
Title The Maximum Concentration Observed After Dosing (Cmax) for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
2.Primary Outcome
Title The Time of Maximum Observed Concentration (Cmax) Relative to Time of Dosing for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Primary Outcome
Title AUClast for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description The area under the concentration-time curve from time of dosing of the drug to the time of the last measurable concentration or when concentrations were Below the Limit of Quantitation (BLQ) were calculated using either the linear (concentration before Cmax) or log trapezoidal rule (concentrations after Cmax). Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Primary Outcome
Title AUCinfinity for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description The AUC extrapolated from the last measured concentration (Clast) to time infinity using the formula AUClast + Clast / λz. Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Primary Outcome
Title Mean Residence Time as Generated by WinNonlin (AUMC/AUC) for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
6.Primary Outcome
Title Apparent Clearance Calculated From Dose/ AUCINF for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Primary Outcome
Title Apparent Clearance Normalized for Body Weight (BW) for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
8.Primary Outcome
Title Elimination Slope for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description The first-order linear slope associated with the terminal (log-linear) portion of the curve and estimated via linear regression of log concentrations vs. time. Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
9.Primary Outcome
Title Terminal Elimination Half-life Obtained From: t½ = ln(2)/ λz for HU Liquid Formulation in Infants (9 Months to <2 Years)
Hide Description Summary statistics including mean, standard deviation (SD) will be reported.
Time Frame 1 day
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were enrolled on Arm 1.
Arm/Group Title Arm 1 Liquid Hydroxyurea
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
10.Secondary Outcome
Title The Maximum Concentration Observed After Dosing (Cmax) for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
11.Secondary Outcome
Title The Time of Maximum Observed Concentration (Cmax) Relative to Time of Dosing for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
12.Secondary Outcome
Title AUClast for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description The AUC extrapolated from the last measured concentration (Clast) to time infinity using the formula AUClast + Clast / λz. Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title AUCinfinity for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description The AUC extrapolated from the last measured concentration (Clast) to time infinity using the formula AUClast + Clast / λz. Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Mean Residence Time as Generated by WinNonlin (AUMC/AUC) for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
15.Secondary Outcome
Title Apparent Clearance Calculated From Dose/ AUCINF for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
16.Secondary Outcome
Title Apparent Clearance Normalized for Body Weight (BW) for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
17.Secondary Outcome
Title Elimination Slope for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description The first-order linear slope associated with the terminal (log-linear) portion of the curve and estimated via linear regression of log concentrations vs. time. Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
18.Secondary Outcome
Title Terminal Elimination Half-life Obtained From: t½ = ln(2)/ λz for HU "Sprinkles" Compared to Capsules in Children and Adolescents (≥2 to 18 Years)
Hide Description Summary statistics including mean and SD will be reported for "sprinkles" and capsules and will be compared using two-sample t-test or Wilcoxon rank sum test depending on the normality of the data at a significance level of 0.05 per study design above. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants treated on Arm 2.
Arm/Group Title Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
19.Secondary Outcome
Title The Maximum Concentration Observed After Dosing (Cmax) for Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:
In Arm 1 of this study, 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 oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
20.Secondary Outcome
Title The Time of Maximum Observed Concentration (Cmax) Relative to Time of Dosing for Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
21.Secondary Outcome
Title AUClast for Infants Versus Older Children
Hide Description The area under the concentration-time curve from time of dosing of the drug to the time of the last measurable concentration or when concentrations were Below the Limit of Quantitation (BLQ) were calculated using either the linear (concentration before Cmax) or log trapezoidal rule (concentrations after Cmax). The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
22.Secondary Outcome
Title AUCinfinity for Infants Versus Older Children
Hide Description The AUC extrapolated from the last measured concentration (Clast) to time infinity using the formula AUClast + Clast / λz. The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
23.Secondary Outcome
Title Mean Residence Time as Generated by WinNonlin (AUMC/AUC) for Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
24.Secondary Outcome
Title Apparent Clearance Calculated From Dose/ AUCINF for In Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
25.Secondary Outcome
Title Apparent Clearance Normalized for Body Weight (BW) for Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
26.Secondary Outcome
Title Elimination Slope for In Infants Versus Older Children
Hide Description The first-order linear slope associated with the terminal (log-linear) portion of the curve and estimated via linear regression of log concentrations vs. time. The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
27.Secondary Outcome
Title Terminal Elimination Half-life Obtained From: t½ = ln(2)/ λz for Infants Versus Older Children
Hide Description The older children will include children on arm 2 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. Summary statistics will be reported for the infants and older children and will be compared using two sample t-test or Wilcoxon rank sum test depending on the normality of the data. Logarithmic transformation will be applied if data do not follow normal.
Time Frame 2 days
Hide Outcome Measure Data
Hide Analysis Population Description
No participants were treated on this study.
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description:

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame No participants were treated on this study.
Adverse Event Reporting Description No participants were treated on this study.
 
Arm/Group Title Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Hide Arm/Group Description

In Arm 1 of this study, 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.

Hydroxyurea: Drug: Hydroxyurea oral liquid dose administered will be 20mg/kg/day or infants's usual daily dose.

In Arm 2, n=30 children who range in age from 2 to 18 years will be administered oral capsule HU, both a sprinkle formulation and capsules (Droxia® 200 mg), on two separate occasions separated by at least 1 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

Hydroxyurea Oral Capsule: Drug: Hydroxyurea both a sprinkle formulation and capsules (Droxia 200mg) administered on 2 separate occasions.
All-Cause Mortality
Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
Hide Serious Adverse Events
Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Arm 1 Liquid Hydroxyurea Arm 2 Hydroxyurea Oral Capsule
Affected / at Risk (%) Affected / at Risk (%)
Total   0/0   0/0 
No participants were treated on this study.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jeremie Estepp, MD
Organization: St. Jude Children's Research Hospital
Phone: (901) 595-5703
EMail: jeremie.estepp@stjude.org
Layout table for additonal information
Responsible Party: St. Jude Children's Research Hospital
ClinicalTrials.gov Identifier: NCT03825341    
Other Study ID Numbers: HOPE18
First Submitted: January 23, 2019
First Posted: January 31, 2019
Results First Submitted: February 18, 2022
Results First Posted: March 18, 2022
Last Update Posted: March 18, 2022