Trial Outcomes & Findings for Lysteda Pediatric Research Equity Act (PREA) Pharmacokinetic Study in Adolescent Females With Heavy Menstrual Bleeding (NCT NCT01190150)
NCT ID: NCT01190150
Last Updated: 2012-07-16
Results Overview
Cmax is the maximum measured plasma concentration over the time-span specified.
COMPLETED
PHASE4
20 participants
Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)
2012-07-16
Participant Flow
Participant milestones
| Measure |
0.65 g / 1.3 g Tranexamic Acid
Participants received a single dose of 0.65 g tranexamic acid on Day 1 and a single dose of 1.3 g tranexamic acid on Day 8.
|
1.3 g / 0.65 g Tranexamic Acid
Participants received a single dose of 1.3 g tranexamic acid on Day 1 and a single dose of 0.65 g tranexamic acid on Day 8.
|
|---|---|---|
|
First Treatment
STARTED
|
11
|
9
|
|
First Treatment
COMPLETED
|
10
|
9
|
|
First Treatment
NOT COMPLETED
|
1
|
0
|
|
Washout
STARTED
|
10
|
9
|
|
Washout
COMPLETED
|
10
|
7
|
|
Washout
NOT COMPLETED
|
0
|
2
|
|
Second Treatment
STARTED
|
10
|
7
|
|
Second Treatment
COMPLETED
|
10
|
7
|
|
Second Treatment
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
0.65 g / 1.3 g Tranexamic Acid
Participants received a single dose of 0.65 g tranexamic acid on Day 1 and a single dose of 1.3 g tranexamic acid on Day 8.
|
1.3 g / 0.65 g Tranexamic Acid
Participants received a single dose of 1.3 g tranexamic acid on Day 1 and a single dose of 0.65 g tranexamic acid on Day 8.
|
|---|---|---|
|
First Treatment
Withdrawal by Subject
|
1
|
0
|
|
Washout
Withdrawal by Subject
|
0
|
1
|
|
Washout
Difficulty in adherence
|
0
|
1
|
Baseline Characteristics
Lysteda Pediatric Research Equity Act (PREA) Pharmacokinetic Study in Adolescent Females With Heavy Menstrual Bleeding
Baseline characteristics by cohort
| Measure |
0.65 g / 1.3 g Tranexamic Acid
n=11 Participants
Participants received a single dose of 0.65 g tranexamic acid on Day 1 and a single dose of 1.3 g tranexamic acid on Day 8.
|
1.3 g / 0.65 g Tranexamic Acid
n=9 Participants
Participants received a single dose of 1.3 g tranexamic acid on Day 1 and a single dose of 0.65 g tranexamic acid on Day 8.
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
14.5 years
STANDARD_DEVIATION 1.13 • n=5 Participants
|
14.3 years
STANDARD_DEVIATION 1.32 • n=7 Participants
|
14.4 years
STANDARD_DEVIATION 1.19 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
Cmax is the maximum measured plasma concentration over the time-span specified.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Maximum Concentrations Level (Cmax)
|
6.9967 μg/mL
Standard Deviation 2.08058
|
10.9868 μg/mL
Standard Deviation 3.38910
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
Cmax is the maximum measured plasma concentration over the time-span specified and normalized to the 1.3 g dose.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Dose-normalized Maximum Concentrations Level (Cmax)
|
13.9933 μg/mL
Standard Deviation 4.16115
|
10.9868 μg/mL
Standard Deviation 3.38910
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
Time of the maximum measured plasma concentration. If the maximum value occurs at more than one time point, Tmax is defined as the first time point with this value.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Time to Maximum Concentration Level (Tmax)
|
3.17 hours
Standard Deviation 0.642
|
3.11 hours
Standard Deviation 0.679
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
The area under the plasma concentration versus time curve, from time 0 to the last measurable concentration, as calculated by the linear trapezoidal method.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Area Under the Concentration Versus Time Curve From 0 to the Last Time Point (AUC0-t)
|
38.8067 μg*h/mL
Standard Deviation 10.56742
|
56.7935 μg*h/mL
Standard Deviation 19.27448
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
The area under the plasma concentration versus time curve, from time 0 to the last measurable concentration normalized to the 1.3 g dose.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Dose Normalized Area Under the Concentration Versus Time Curve From 0 to the Last Time Point (AUC0-t)
|
77.6134 μg*h/mL
Standard Deviation 21.13483
|
56.7935 μg*h/mL
Standard Deviation 19.27448
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population including all participants with three or more non-zero plasma concentrations. One participant withdrew on Day 7 and did not meet the requisite samples required for this PK parameter.
The area under the plasma concentration versus time curve from time 0 to infinity. AUCinf is calculated as the sum of AUC0-t plus the ratio of the last measurable plasma concentration to the elimination rate constant.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Area Under the Concentration Versus Time Curve From 0 to Infinity (AUCinf)
|
39.8016 μg*h/mL
Standard Deviation 10.71237
|
61.2591 μg*h/mL
Standard Deviation 15.45770
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population including all participants with three or more non-zero plasma concentrations. One participant withdrew on Day 7 and did not meet the requisite samples required for this PK parameter.
Dose-normalized AUCinf is calculated as the sum of AUC0-t plus the ratio of the last measurable plasma concentration to the elimination rate constant, normalized to the 1.3 g dose.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Dose Normalized Area Under the Concentration Versus Time Curve From 0 to Infinity (AUCinf)
|
79.6032 μg*h/mL
Standard Deviation 21.42474
|
61.2591 μg*h/mL
Standard Deviation 15.45770
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population including all participants with three or more non-zero plasma concentrations. One participant withdrew on Day 7 and did not meet the requisite samples required for this PK parameter.
Comparison of AUC0-t to AUCinf by creating a ratio.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
The Ratio of AUC0-t to AUCinf
|
0.9741 ratio of AUC0-t / AUCinf
Standard Deviation 0.00862
|
0.9715 ratio of AUC0-t / AUCinf
Standard Deviation 0.01158
|
PRIMARY outcome
Timeframe: Day 1 or Day 8 (before dosing and at the following times thereafter: 0.5, 0.75, 1, 2, 2.5, 3.0, 3.5, 4, 5, 6, 10, 14, and 24 hours post-dose)Population: Plasma PK population includes all participants with at least one quantifiable PK concentration.
Apparent first-order terminal elimination half life
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Elimination Half-life (t ½)
|
5.06 hours
Standard Deviation 1.194
|
5.42 hours
Standard Deviation 1.463
|
SECONDARY outcome
Timeframe: Day 1 up to week 4Population: The Safety Population consisted of all randomized participants who received at least one dose of tranexamic acid.
Treatment-emergent AEs are summarized by total participants with TEAEs, participants with serious TEAEs, participants with TEAEs deemed by the investigator to be related to treatment, and participants who experienced TEAEs that caused permanent discontinuation from the study.
Outcome measures
| Measure |
0.65 g Tranexamic Acid
n=18 Participants
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 Participants
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Participants With Treatment-emergent Adverse Events (TEAEs)
Participants with TEAEs
|
1 participants
|
2 participants
|
|
Participants With Treatment-emergent Adverse Events (TEAEs)
Participants with serious TEAEs
|
0 participants
|
0 participants
|
|
Participants With Treatment-emergent Adverse Events (TEAEs)
Participants with treatment-related TEAEs
|
0 participants
|
1 participants
|
|
Participants With Treatment-emergent Adverse Events (TEAEs)
Participants with TEAEs causing discontinuation
|
0 participants
|
0 participants
|
Adverse Events
0.65 g Tranexamic Acid
1.3 g Tranexamic Acid
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
0.65 g Tranexamic Acid
n=18 participants at risk
Participants were treated with a single dose of 0.65 g tranexamic acid.
|
1.3 g Tranexamic Acid
n=19 participants at risk
Participants were treated with a single dose of 1.3 g tranexamic acid.
|
|---|---|---|
|
Nervous system disorders
Dizziness
|
0.00%
0/18 • Day 1 up to week 4
|
10.5%
2/19 • Day 1 up to week 4
|
|
Nervous system disorders
Headache
|
0.00%
0/18 • Day 1 up to week 4
|
5.3%
1/19 • Day 1 up to week 4
|
|
Skin and subcutaneous tissue disorders
Blister
|
5.6%
1/18 • Day 1 up to week 4
|
0.00%
0/19 • Day 1 up to week 4
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The only disclosure restriction on the PI is that the sponsor can review the draft manuscript prior to publication and can request delay of publication where any contents are deemed patentable by the sponsor or confidential to the sponsor. Comments will be given within four weeks from receipt of the draft manuscript. Additional time may be required to allow Ferring to seek patent protection of the invention.
- Publication restrictions are in place
Restriction type: OTHER