Trial Outcomes & Findings for Safety and Tolerability of Dabigatran Etexilate in Adolescents (NCT NCT00844415)

Results Overview

Patients were carefully assessed for signs and symptoms of bleeding. Bleeding was to be classified as major or minor. Major bleeding had to satisfy one or more of the following criteria: Overt bleeding associated with a decrease in haemoglobin of at least 2 g/dL in 24 hours, Overt bleeding requiring a transfusion of red blood cells, Overt bleeding which was retroperitoneal, intracranial, intraocular, or intraarticular, any overt bleeding deemed by the attending physician to require discontinuation of study medication. Minor bleeds were clinical bleeds that did not fulfill the criteria for major bleeds.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

9 participants

Primary outcome timeframe

From Screening until 30 days after first drug administration (end of trial visit)

Results posted on

2025-02-12

Participant Flow

Participant milestones

Participant milestones
Measure	All Patients (Pat.) Dabigatran was administered twice daily for three consecutive days (total 6 doses). All patients received an initial oral dose of 1.71mg/kg of dabigatran (80 percent of the adult dose of 150 mg/70 kg adjusted for the patient's weight). Based on thrombin time (TT) and clinical assessment, the dose was adjusted to the target dose of 2.14 mg/kg of dabigatran (100 percent of the adult dose adjusted for the patient's weight). Three patients received 75 mg dabigatran (first dose) followed by 100 mg twice daily (BID). Three patients took dabigatran 100 mg (first dose) followed by 125 mg BID. Two patients received a dose of 125 mg dabigatran followed by 150 mg BID. One patient received only a single dose of dabigatran (75 mg).
Overall Study STARTED	9
Overall Study Pat. Received 75mg Followed by 100mg	3
Overall Study Pat. Received 100mg Followed by 125mg	3
Overall Study Pat. Received 125mg Followed by 150mg	2
Overall Study Pat. Received Single Dose 75mg	1
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients (Pat.) Dabigatran was administered twice daily for three consecutive days (total 6 doses). All patients received an initial oral dose of 1.71mg/kg of dabigatran (80 percent of the adult dose of 150 mg/70 kg adjusted for the patient's weight). Based on thrombin time (TT) and clinical assessment, the dose was adjusted to the target dose of 2.14 mg/kg of dabigatran (100 percent of the adult dose adjusted for the patient's weight). Three patients received 75 mg dabigatran (first dose) followed by 100 mg twice daily (BID). Three patients took dabigatran 100 mg (first dose) followed by 125 mg BID. Two patients received a dose of 125 mg dabigatran followed by 150 mg BID. One patient received only a single dose of dabigatran (75 mg).
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Safety and Tolerability of Dabigatran Etexilate in Adolescents

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=9 Participants Dabigatran was administered twice daily for three consecutive days (total 6 doses). All patients received an initial oral dose of 1.71mg/kg of dabigatran (80 percent of the adult dose of 150 mg/70 kg adjusted for the patient's weight). Based on thrombin time (TT) and clinical assessment, the dose was adjusted to the target dose of 2.14 mg/kg of dabigatran (100 percent of the adult dose adjusted for the patient's weight). Three patients received 75 mg dabigatran (first dose) followed by 100 mg twice daily (BID). Three patients took dabigatran 100 mg (first dose) followed by 125 mg BID. Two patients received a dose of 125 mg dabigatran followed by 150 mg BID. One patient received only a single dose of dabigatran (75 mg).
Age, Continuous	15.7 years STANDARD_DEVIATION 1.3 • n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: From Screening until 30 days after first drug administration (end of trial visit)

Population: Treated set (TS). This patient set includes all patients who were dispensed study medication and were documented to have taken at least one dose of investigational treatment.

Patients were carefully assessed for signs and symptoms of bleeding. Bleeding was to be classified as major or minor. Major bleeding had to satisfy one or more of the following criteria: Overt bleeding associated with a decrease in haemoglobin of at least 2 g/dL in 24 hours, Overt bleeding requiring a transfusion of red blood cells, Overt bleeding which was retroperitoneal, intracranial, intraocular, or intraarticular, any overt bleeding deemed by the attending physician to require discontinuation of study medication. Minor bleeds were clinical bleeds that did not fulfill the criteria for major bleeds.

Outcome measures

Outcome measures
Measure	All Patients n=9 Participants Dabigatran was administered twice daily for three consecutive days (total 6 doses). All patients received an initial oral dose of 1.71mg/kg of dabigatran (80 percent of the adult dose of 150 mg/70 kg adjusted for the patient's weight). Based on thrombin time (TT) and clinical assessment, the dose was adjusted to the target dose of 2.14 mg/kg of dabigatran (100 percent of the adult dose adjusted for the patient's weight). Three patients received 75 mg dabigatran (first dose) followed by 100 mg twice daily (BID). Three patients took dabigatran 100 mg (first dose) followed by 125 mg BID. Two patients received a dose of 125 mg dabigatran followed by 150 mg BID. One patient received only a single dose of dabigatran (75 mg).
Number of Patients With Bleeding Events (Major and Minor)	0 Participants

PRIMARY outcome

Timeframe: From Screening until 30 days after first drug administration (end of trial visit)

Population: TS

Patients with treatment drug related adverse events (DRAEs) and serious adverse events (SAEs) are reported separately for on-treatment and post-treatment period. Events were considered "on-treatment" if occurring within 72 hours after last drug administration.

Outcome measures

Outcome measures
Measure	All Patients n=9 Participants Dabigatran was administered twice daily for three consecutive days (total 6 doses). All patients received an initial oral dose of 1.71mg/kg of dabigatran (80 percent of the adult dose of 150 mg/70 kg adjusted for the patient's weight). Based on thrombin time (TT) and clinical assessment, the dose was adjusted to the target dose of 2.14 mg/kg of dabigatran (100 percent of the adult dose adjusted for the patient's weight). Three patients received 75 mg dabigatran (first dose) followed by 100 mg twice daily (BID). Three patients took dabigatran 100 mg (first dose) followed by 125 mg BID. Two patients received a dose of 125 mg dabigatran followed by 150 mg BID. One patient received only a single dose of dabigatran (75 mg).
Number of Patients With Adverse Events DRAEs on-treatment	2 Participants
Number of Patients With Adverse Events SAEs on-treatment	0 Participants
Number of Patients With Adverse Events DRAEs post-treatment	0 Participants
Number of Patients With Adverse Events SAEs post-treatment	1 Participants

PRIMARY outcome

Timeframe: 3 days

Population: Treated Set. Descriptive statistics for the concentration measurement could only be calculated for the subgroups of patients receiving the same sequence of doses. Statistics were only reported for groups with at least 3 patients (non-sparse data).

Plasma concentration of free dabigatran measured at 72 hours after first dose