Trial Outcomes & Findings for Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere (NCT NCT03825341)
NCT ID: NCT03825341
Last Updated: 2022-03-18
Results Overview
Summary statistics including mean, standard deviation (SD) will be reported.
TERMINATED
PHASE2
1 participants
1 day
2022-03-18
Participant Flow
1 participant was recruited between March 2019 and Jan. 2022. The participant was randomized but did not receive treatment.
Participant milestones
| Measure |
Arm 1 Liquid Hydroxyurea
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.
|
Arm 2 Hydroxyurea Oral Capsule
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 Study
STARTED
|
0
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
Arm 1 Liquid Hydroxyurea
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.
|
Arm 2 Hydroxyurea Oral Capsule
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 Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
Baseline characteristics by cohort
| Measure |
Arm 1 Liquid Hydroxyurea
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.
|
Arm 2 Hydroxyurea Oral Capsule
n=1 Participants
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
n=1 Participants
Total of all reporting groups
|
|---|---|---|---|
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Age, Categorical
<=18 years
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Age, Categorical
Between 18 and 65 years
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Age, Categorical
>=65 years
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Sex: Female, Male
Female
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Sex: Female, Male
Male
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
Asian
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
Black or African American
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
White
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
More than one race
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
—
|
—
|
0 Participants
Baseline data not entered for the 1 enrollment due to confidentiality concerns.
|
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
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.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: 1 dayPopulation: No participants were enrolled on Arm 1.
Summary statistics including mean, standard deviation (SD) will be reported.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants treated on Arm 2.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 2 daysPopulation: No participants were treated on this study.
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.
Outcome measures
Outcome data not reported
Adverse Events
Arm 1 Liquid Hydroxyurea
Arm 2 Hydroxyurea Oral Capsule
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Jeremie Estepp, MD
St. Jude Children's Research Hospital
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place