Trial Outcomes & Findings for Study Evaluating Betrixaban in Pediatric Participants (NCT NCT03346083)
NCT ID: NCT03346083
Last Updated: 2024-01-12
Results Overview
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
TERMINATED
PHASE1
21 participants
Up to 6 days post dose
2024-01-12
Participant Flow
After a screening period of up to 30 days, eligible participants who had provided assent and for whom a parent or legal guardian had provided signed informed consent entered the hospital, clinical research unit, or Phase 1 unit on Day -1. Those who were already inpatients remained hospitalized.
Participant milestones
| Measure |
Cohort 1: Betrixaban 40 mg
Participants received a single, oral dose of betrixaban at 40 milligrams (mg) in a fed state, and had 10 pharmacokinetic (PK) blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
18
|
|
Overall Study
Received At Least 1 Dose Of Study Drug
|
3
|
18
|
|
Overall Study
Evaluated Through Day 7 Follow-up
|
3
|
18
|
|
Overall Study
COMPLETED
|
3
|
18
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study Evaluating Betrixaban in Pediatric Participants
Baseline characteristics by cohort
| Measure |
Cohort 1: Betrixaban 40 mg
n=3 Participants
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
n=18 Participants
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
Age Categorical · In utero
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · Preterm newborn - gestational age < 37 wk
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · Newborns (0-27 days)
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · Children (2-11 years)
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · Adolescents (12-17 years)
|
3 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · Adults (18-64 years)
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · From 65 to 84 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Customized
Age Categorical · 85 years and over
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 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
|
0 Participants
n=7 Participants
|
0 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
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
18 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
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Ukraine
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Russia
|
3 participants
n=5 Participants
|
11 participants
n=7 Participants
|
14 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
0 participants
n=5 Participants
|
4 participants
n=7 Participants
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 6 days post dosePopulation: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-inf) were not collected.
Outcome measures
Outcome data not reported
PRIMARY outcome
Timeframe: Up to 6 days post dosePopulation: The evaluable PK population included all participants who received the study drug and had sufficient blood samples through Day 3 to compute either Cmax or total AUC assessments with the extrapolated portion of the AUC(0-inf) less than 30%.
Data reported as "0.200" indicates that the data are below the lower limit of quantification. Note that the Measure of Central Tendency could not be determined for Cohort 1 or Cohort 2 due to the values that are below the lower limit of quantification.
Outcome measures
| Measure |
Cohort 1: Betrixaban 40 mg
n=3 Participants
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
n=18 Participants
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
|---|---|---|
|
Maximum Observed Plasma Concentration (Cmax) Of Betrixaban
|
NA nanograms (ng)/milliliters (mL)
Interval 0.2 to 2.57
The Lower Limit and Upper Limit Cmax data are reported here as only individual participant data are available.
|
NA nanograms (ng)/milliliters (mL)
Interval 0.2 to 48.5
The Lower Limit and Upper Limit Cmax data are reported here as only individual participant data are available.
|
SECONDARY outcome
Timeframe: Up to 6 days post dosePopulation: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for AUC(0-last) were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 6 days post dosePopulation: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for t½ were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 6 days post dosePopulation: The evaluable PK population included all participants who received the study drug and had sufficient blood samples through Day 3 to compute either Cmax or total AUC assessments with the extrapolated portion of the AUC(0-inf) less than 30%.
The Tmax that the highest (maximum) Cmax of betrixaban was observed per group up to Day 6 (120 hours) post dosing is reported.
Outcome measures
| Measure |
Cohort 1: Betrixaban 40 mg
n=3 Participants
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
n=18 Participants
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
|---|---|---|
|
Time To Maximum Observed Plasma Concentration (Tmax) Of Betrixaban
|
2 hours
Interval 1.0 to 6.0
|
2 hours
Interval 1.0 to 12.0
|
SECONDARY outcome
Timeframe: Up to 6 days post dosePopulation: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for CL were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 6 days post dosePopulation: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for Vd were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Day 6Population: After completion of Part 1 of the study and prior to initiating Part 2, the Sponsor decided to stop developing betrixaban and closed the study early. Therefore, data for this Outcome Measure were not collected.
Following the Sponsor's decision to cease developing betrixaban, data for thrombin levels were not collected.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 7 days post dosePopulation: Safety population: all participants enrolled in Part 1 of the study who received study drug.
A treatment-related adverse event was any undesirable event or any untoward medical occurrence that occurs to a participant during the course of a study, or the protocol-defined time after study termination. An Investigator qualified in medicine made the determination of relationship to the investigational product for each adverse event (Unrelated, Unlikely Related, Possibly Related, or Probably Related). If the relationship between the adverse event and the investigational product was determined to be "possible" or "probable", the event was considered to be related to the investigational product for the purposes of expedited regulatory reporting. One participant experienced a mild study-drug-related headache that resolved in less than 2 hours. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Cohort 1: Betrixaban 40 mg
n=3 Participants
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
n=18 Participants
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
|---|---|---|
|
Count Of Participants With Treatment-related Adverse Events
|
0 Participants
|
1 Participants
|
Adverse Events
Cohort 1: Betrixaban 40 mg
Cohort 2: Betrixaban 80 mg
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort 1: Betrixaban 40 mg
n=3 participants at risk
Participants received a single, oral dose of betrixaban at 40 mg in a fed state, and had 10 PK blood sampling time points.
|
Cohort 2: Betrixaban 80 mg
n=18 participants at risk
Participants received a single, oral dose of betrixaban at 80 mg in a fed state, and had 5 PK sampling time points.
|
|---|---|---|
|
Vascular disorders
Hypertension
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Vascular disorders
Hypotension
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Cardiac disorders
Bradycardia
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/3 • Up to 7 days post dose.
|
5.6%
1/18 • Number of events 1 • Up to 7 days post dose.
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place