Trial Outcomes & Findings for Study of Diazepam Buccal Film Administered in the Interictal and in the Ictal-Periictal States to Adults With Epilepsy (NCT NCT03179891)

NCT ID: NCT03179891

Last Updated: 2020-09-18

Results Overview

Observed time to reach maximum drug concentration (Tmax)

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

35 participants

Primary outcome timeframe

-2 to 0, 0.25, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 192, 240 hours

Results posted on

2020-09-18

Participant Flow

Planned enrollment was 40 adult male and female subjects with a clinical diagnosis of epilepsy requiring admission to an epilepsy monitoring unit, a general clinical research center, or similar facility for evaluation of seizures. A minimum of 30 subjects was expected to complete the study.

A total of 35 subjects were enrolled to receive a 12.5-mg dose of study drug during both the interictal and the ictal/peri-ictal period which occurred in either order as determined by seizure occurrence.

Participant milestones

Participant milestones
Measure
Interictal State First, Then Ictal/Peri-ictal State
Subjects received DBF 12.5 mg during the Interical State first and then during the Ictal/Peri-ictal State with at least 14 days washout between the 2 periods.
Ictal/Peri-Ictal State First, Then Interictal State
Subjects received DBF 12.5 mg during the ictal/peri-ictal state first and then during the interictal state with at least 14 days washout between the 2 periods.
First Intervention
STARTED
13
22
First Intervention
COMPLETED
11
20
First Intervention
NOT COMPLETED
2
2
Second Intervention (After 14 Days)
STARTED
11
20
Second Intervention (After 14 Days)
COMPLETED
11
20
Second Intervention (After 14 Days)
NOT COMPLETED
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Interictal State First, Then Ictal/Peri-ictal State
Subjects received DBF 12.5 mg during the Interical State first and then during the Ictal/Peri-ictal State with at least 14 days washout between the 2 periods.
Ictal/Peri-Ictal State First, Then Interictal State
Subjects received DBF 12.5 mg during the ictal/peri-ictal state first and then during the interictal state with at least 14 days washout between the 2 periods.
First Intervention
Withdrawal by Subject
1
1
First Intervention
Unspecified reason
1
0
First Intervention
Protocol Violation
0
1

Baseline Characteristics

Thirty-five adult subjects with epilepsy received at least 1 dose of study drug and were included in the analysis of safety.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Adult Subjects With Epilepsy
n=35 Participants
Adult subjects with epilepsy admitted to an Epilepsy Monitoring Unit to receive a single 12.5 mg DBF dose during the Ictal Phase and during the Interictal/Peri-ictal Phase with at least 14 days between the 2 periods
Age, Categorical
<=18 years
0 Participants
n=99 Participants
Age, Categorical
Between 18 and 65 years
29 Participants
n=99 Participants
Age, Categorical
>=65 years
6 Participants
n=99 Participants
Sex: Female, Male
Female
18 Participants
n=99 Participants
Sex: Female, Male
Male
17 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
9 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
26 Participants
n=99 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=99 Participants
Race (NIH/OMB)
American Indian or Alaska Native
2 Participants
n=99 Participants
Race (NIH/OMB)
Asian
1 Participants
n=99 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
6 Participants
n=99 Participants
Race (NIH/OMB)
Black or African American
5 Participants
n=99 Participants
Race (NIH/OMB)
White
19 Participants
n=99 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=99 Participants
Race (NIH/OMB)
Unknown or Not Reported
2 Participants
n=99 Participants
Region of Enrollment
United States
35 participants
n=99 Participants • Thirty-five adult subjects with epilepsy received at least 1 dose of study drug and were included in the analysis of safety.
Height
165.64 cm
STANDARD_DEVIATION 10.81 • n=99 Participants
Weight
79.24 kg
STANDARD_DEVIATION 19.58 • n=99 Participants
BMI
27.84 kg/m^2
STANDARD_DEVIATION 7.01 • n=99 Participants

PRIMARY outcome

Timeframe: -2 to 0, 0.25, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 192, 240 hours

Population: The pharmacokinetic analysis set comprised only those subjects who contributed a valid pharmacokinetic profile for the given analysis and whose predose concentration of the relevant analyte did not exceed 5% of the subsequent Cmax.

Observed time to reach maximum drug concentration (Tmax)

Outcome measures

Outcome measures
Measure
Interictal State
n=32 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=24 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Tmax Pharmacokinetic EndPoints
1.000 hours
Interval 0.249 to 4.017
0.525 hours
Interval 0.25 to 2.033

PRIMARY outcome

Timeframe: -2 to 0, 0.25, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 192, 240 hours

Population: The pharmacokinetic analysis set comprised only those subjects who contributed a valid pharmacokinetic profile for the given analysis and whose predose concentration of the relevant analyte did not exceed 5% of the subsequent Cmax.

Observed Peak Drug Concentration (Cmax)

Outcome measures

Outcome measures
Measure
Interictal State
n=32 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=24 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Cmax Pharmacokinetic EndPoints
230.32 ng/mL
Standard Deviation 89.00
209.49 ng/mL
Standard Deviation 120.18

PRIMARY outcome

Timeframe: -2 to 0, 0.25, 0.5, 1, 2, 4, 8, 24, 48, 96, 144, 192, 240 hours

Population: The pharmacokinetic analysis set comprised only those subjects who contributed a valid pharmacokinetic profile for the given analysis and whose predose concentration of the relevant analyte did not exceed 5% of the subsequent Cmax.

Area under the Plasma Concentration -time curve from time zero until the last measured time (AUC0-t)

Outcome measures

Outcome measures
Measure
Interictal State
n=32 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=24 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Area Under the Plasma Concentration Curve Pharmacokinetic EndPoints
560.18 h*ng/mL
Standard Deviation 219.70
498.78 h*ng/mL
Standard Deviation 319.09

SECONDARY outcome

Timeframe: Subject was observed for 15 minutes after initial film placement/adhesion

Population: Safety Population comprising all subjects who received at least 1 dose of study drug (Diazepam Buccal Film).

Number of subjects with unsuccessful insertion/placement of the DBF on first attempt at administration Number of subjects with successful insertion/placement of the DBF on first attempt at administration Placement is judged to be successful when film adheres to the center of buccal mucosa of either right or left cheek. Unsuccessful placements were followed by a subsequent successful insertion/placement of DBF

Outcome measures

Outcome measures
Measure
Interictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Usability Endpoint - Successful Insertion/Placement of the Diazepam Buccal Film (DBF) on First Attempt
Unsuccessful insertion/placement
0 Participants
2 Participants
Usability Endpoint - Successful Insertion/Placement of the Diazepam Buccal Film (DBF) on First Attempt
Successful insertion/placement
33 Participants
31 Participants

SECONDARY outcome

Timeframe: Subject was observed for 15 minutes immediately following DBF placement/adhesion

Population: Safety Population comprising all subjects who received at least 1 dose of study drug (Diazepam Buccal Film).

Was the film noted to have been swallowed by the subject ? Yes No Subjects were instructed to swallow any remnants of film still present in oral cavity 15 minutes after initial film placement. Results include subjects who swallowed film at any point during the 15 minutes immediately after initial film placement.

Outcome measures

Outcome measures
Measure
Interictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Usability Endpoint: Swallowing the Film Before Complete Disintegration/Dissolution
Yes-DBF was Swallowed
4 Participants
2 Participants
Usability Endpoint: Swallowing the Film Before Complete Disintegration/Dissolution
No-DBF was not Swallowed
29 Participants
31 Participants

SECONDARY outcome

Timeframe: Subject was observed for 15 minutes immediately following DBF placement/adhesion

Population: Safety Population comprising all subjects who received at least 1 dose of study drug (Diazepam Buccal Film).

Was the DBF spit out or blown out by the subject after placement on buccal mucosa or did the subject chew, talk, or move the DBF prior to complete disintegration/dissolution? Yes No

Outcome measures

Outcome measures
Measure
Interictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Usability Endpoint: Retention of Diazepam Buccal Film (DBF) From Placement to Complete Disintegration
Yes-DBF was spit out/blown out
0 Participants
3 Participants
Usability Endpoint: Retention of Diazepam Buccal Film (DBF) From Placement to Complete Disintegration
No-DBF was not spit out/blown out
33 Participants
30 Participants

SECONDARY outcome

Timeframe: Subject was observed for 15 minutes immediately following DBF placement/adhesion

Population: Safety Population comprising all subjects who received at least 1 dose of study drug (Diazepam Buccal Film).

The observer documented if any saliva was seen to exit the mouth during the time the DBF was adhered to buccal mucosa

Outcome measures

Outcome measures
Measure
Interictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Usability Endpoint: Exit of Saliva During the Time the Diazepam Buccal Film (DBF) Was Adhered to Buccal Mucosa
Yes, saliva exited the mouth
0 Participants
2 Participants
Usability Endpoint: Exit of Saliva During the Time the Diazepam Buccal Film (DBF) Was Adhered to Buccal Mucosa
No, saliva did not exit mouth
33 Participants
31 Participants

SECONDARY outcome

Timeframe: Subject was observed for 15 minutes immediately following DBF placement/adhesion

Population: Safety Population comprising all subjects who received at least 1 dose of study drug (Diazepam Buccal Film).

If Yes - saliva exited the mouth during the time, estimate in milliliters of the amount of saliva that exited the mouth after DBF placement on the buccal surface

Outcome measures

Outcome measures
Measure
Interictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state Adult subjects with epilepsy
Ictal/Peri-ictal State
n=33 Participants
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state Adult subjects with epilepsy
Usability Endpoint: Amount of Saliva That Exited the Mouth After Film Placement
Participants with 0 mL saliva exiting the mouth
33 Participants
31 Participants
Usability Endpoint: Amount of Saliva That Exited the Mouth After Film Placement
Participants with 1 mL saliva exiting the mouth
0 Participants
1 Participants
Usability Endpoint: Amount of Saliva That Exited the Mouth After Film Placement
Participants with 2 mL saliva exiting the mouth
0 Participants
1 Participants

Adverse Events

Interictal State (Period A)

Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths

Ictal/Peri-ictal State (Period B)

Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Interictal State (Period A)
n=33 participants at risk
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state. Adult subjects with epilepsy
Ictal/Peri-ictal State (Period B)
n=33 participants at risk
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state. Adult subjects with epilepsy
Nervous system disorders
HOSPITALIZATION DUE TO SEIZURE CLUSTER
0.00%
0/33 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
3.0%
1/33 • Number of events 1 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).

Other adverse events

Other adverse events
Measure
Interictal State (Period A)
n=33 participants at risk
Single 12.5-mg dose of Diazepam Buccal Film administered during the interictal state. Adult subjects with epilepsy
Ictal/Peri-ictal State (Period B)
n=33 participants at risk
Single 12.5-mg dose of Diazepam Buccal Film administered during the ictal/peri-ictal state. Adult subjects with epilepsy
Nervous system disorders
Complex Partial Seizures
27.3%
9/33 • Number of events 9 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
42.4%
14/33 • Number of events 14 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Nervous system disorders
Headache
0.00%
0/33 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
9.1%
3/33 • Number of events 3 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Nervous system disorders
Generalized tonic-clonic seizure
3.0%
1/33 • Number of events 6 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
3.0%
1/33 • Number of events 3 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Nervous system disorders
Seizure
3.0%
1/33 • Number of events 2 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
3.0%
1/33 • Number of events 4 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Nervous system disorders
Somnolence
6.1%
2/33 • Number of events 3 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
0.00%
0/33 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Nervous system disorders
Seizure cluster
0.00%
0/33 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
3.0%
1/33 • Number of events 1 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
Gastrointestinal disorders
Diarrhoea
0.00%
0/33 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).
3.0%
1/33 • Number of events 1 • Adverse events were collected throughout the course of the study for each subject (approximately 1 month for each subject - beginning with the first DBF dose until 14 ± 2 days following the second DBF dose).

Additional Information

Director of Clinical Operations

Aquestive Therapeutics

Phone: 908-941-1900

Results disclosure agreements

  • Principal investigator is a sponsor employee Trial results were included as confidential information and required written sponsor approval.
  • Publication restrictions are in place

Restriction type: OTHER