Trial Outcomes & Findings for Dexmedetomidine in the Treatment of Agitation Associated With Schizophrenia (NCT NCT04268303)

Last Updated: 2023-06-18

Results Overview

The Positive and Negative Syndrome Scale-Excited Component (PEC) includes 5 items-poor impulse control, tension, hostility, uncooperativeness, and excitement-each of which is rated from 1 (minimum) to 7 (maximum); the sum of these 5 items, the PEC total score, ranges from 5 (absence of agitation) to 35 (extremely severe).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

380 participants

Primary outcome timeframe

Baseline and 2 hours

Results posted on

2023-06-18

Participant Flow

Participant milestones

Participant milestones
Measure	120 Micrograms Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo Sublingual placebo film Placebo Film: Placebo Film for BXCL501
Overall Study STARTED	129	125	126
Overall Study COMPLETED	126	123	123
Overall Study NOT COMPLETED	3	2	3

Reasons for withdrawal

Reasons for withdrawal
Measure	120 Micrograms Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo Sublingual placebo film Placebo Film: Placebo Film for BXCL501
Overall Study Lost to Follow-up	1	1	2
Overall Study Withdrawal by Subject	0	1	1
Overall Study Adverse Event	2	0	0

Baseline Characteristics

One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from the first enrollment and treatment (120 μg) were included in the efficacy analyses. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	120 Micrograms n=129 Participants Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms n=125 Participants Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo n=126 Participants Sublingual placebo film Placebo Film: Placebo Film for BXCL501	Total n=380 Participants Total of all reporting groups
Age, Continuous	45.7 years STANDARD_DEVIATION 11.3 • n=5 Participants	46.0 years STANDARD_DEVIATION 11.9 • n=7 Participants	45.1 years STANDARD_DEVIATION 11.1 • n=5 Participants	45.6 years STANDARD_DEVIATION 11.4 • n=4 Participants
Sex: Female, Male Female	52 Participants n=5 Participants	43 Participants n=7 Participants	44 Participants n=5 Participants	139 Participants n=4 Participants
Sex: Female, Male Male	77 Participants n=5 Participants	82 Participants n=7 Participants	82 Participants n=5 Participants	241 Participants n=4 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	17 Participants n=5 Participants	13 Participants n=7 Participants	7 Participants n=5 Participants	37 Participants n=4 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	112 Participants n=5 Participants	112 Participants n=7 Participants	119 Participants n=5 Participants	343 Participants n=4 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race/Ethnicity, Customized Black or African American	92 Participants n=5 Participants	102 Participants n=7 Participants	102 Participants n=5 Participants	296 Participants n=4 Participants
Race/Ethnicity, Customized White	33 Participants n=5 Participants	21 Participants n=7 Participants	21 Participants n=5 Participants	75 Participants n=4 Participants
Race/Ethnicity, Customized Other	4 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	9 Participants n=4 Participants
Race/Ethnicity, Customized Hispanic or Latino	17 Participants n=5 Participants	13 Participants n=7 Participants	7 Participants n=5 Participants	37 Participants n=4 Participants
Positive and Negative Syndrome Scale-Excited Component	17.5 units on a scale STANDARD_DEVIATION 2.5 • n=5 Participants • One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from the first enrollment and treatment (120 μg) were included in the efficacy analyses. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	17.6 units on a scale STANDARD_DEVIATION 2.7 • n=7 Participants • One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from the first enrollment and treatment (120 μg) were included in the efficacy analyses. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	17.6 units on a scale STANDARD_DEVIATION 2.3 • n=5 Participants • One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from the first enrollment and treatment (120 μg) were included in the efficacy analyses. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	17.6 units on a scale STANDARD_DEVIATION 2.5 • n=4 Participants • One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from the first enrollment and treatment (120 μg) were included in the efficacy analyses. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.

PRIMARY outcome

Timeframe: Baseline and 2 hours

Population: Intent-to-Treat population, defined as all participants in the safety population who have a PEC score post-dose.

Outcome measures

Outcome measures
Measure	120 Micrograms n=129 Participants Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms n=125 Participants Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo n=126 Participants Sublingual placebo film Placebo Film: Placebo Film for BXCL501
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Baseline	17.5 score on a scale Standard Deviation 2.46	17.6 score on a scale Standard Deviation 2.69	17.6 score on a scale Standard Deviation 2.26
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score 2 Hours	-8.4 score on a scale Standard Deviation 4.83	-10.4 score on a scale Standard Deviation 4.34	-4.7 score on a scale Standard Deviation 4.69

SECONDARY outcome

Timeframe: Baseline and 10, 20, 30, 45, 60, 90 minutes post-dose

Population: Intent-to-Treat population, defined as all participants in the safety population who have a PEC score post-dose.

Effect on agitation onset was measured by change from baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) total score. The Positive and Negative Syndrome Scale-Excited Component (PEC) includes 5 items-poor impulse control, tension, hostility, uncooperativeness, and excitement-each of which is rated from 1 (minimum) to 7 (maximum); the sum of these 5 items, the PEC total score, ranges from 5 (absence of agitation) to 35 (extremely severe).

Outcome measures

Outcome measures
Measure	120 Micrograms n=129 Participants Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms n=125 Participants Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo n=126 Participants Sublingual placebo film Placebo Film: Placebo Film for BXCL501
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time Baseline	17.5 score on a scale Standard Deviation 2.46	17.6 score on a scale Standard Deviation 2.69	17.6 score on a scale Standard Deviation 2.26
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 10 minutes	-1.3 score on a scale Standard Deviation 2.50	-2.0 score on a scale Standard Deviation 3.19	-1.3 score on a scale Standard Deviation 2.17
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 20 minutes	-2.8 score on a scale Standard Deviation 4.03	-3.9 score on a scale Standard Deviation 4.54	-2.5 score on a scale Standard Deviation 3.20
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 30 minutes	-4.4 score on a scale Standard Deviation 4.90	-5.7 score on a scale Standard Deviation 5.27	-3.1 score on a scale Standard Deviation 3.56
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 45 minutes	-5.7 score on a scale Standard Deviation 5.18	-7.6 score on a scale Standard Deviation 5.32	-3.6 score on a scale Standard Deviation 3.78
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 60 minutes	-6.9 score on a scale Standard Deviation 5.06	-8.8 score on a scale Standard Deviation 4.96	-4.1 score on a scale Standard Deviation 4.16
Change From Baseline in the Positive and Negative Syndrome Scale- Excited Component (PEC) Total Score Over Time 90 minutes	-8.0 score on a scale Standard Deviation 5.00	-9.8 score on a scale Standard Deviation 4.60	-4.6 score on a scale Standard Deviation 4.38

Adverse Events

120 Micrograms

Serious events: 0 serious events

Other events: 51 other events

Deaths: 0 deaths

180 Micrograms

Serious events: 0 serious events

Other events: 47 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	120 Micrograms n=129 participants at risk Sublingual film containing 120 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	180 Micrograms n=126 participants at risk Sublingual film containing 180 Micrograms dexmedetomidine Sublingual film containing dexmedetomidine (BXCL501): Sublingual film containing dexmedetomidine (BXCL501)	Placebo n=126 participants at risk Sublingual placebo film Placebo Film: Placebo Film for BXCL501
Nervous system disorders Dizziness	2.3% 3/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	6.3% 8/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	0.79% 1/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Gastrointestinal disorders Dry mouth	7.8% 10/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	4.0% 5/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	1.6% 2/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Nervous system disorders Headache	4.7% 6/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	3.2% 4/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	4.8% 6/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Nervous system disorders Hypoesthesia oral	3.9% 5/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	5.6% 7/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	0.00% 0/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Vascular disorders Hypotension	6.2% 8/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	4.0% 5/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	0.00% 0/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Vascular disorders Orthostatic hypotension	1.6% 2/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	5.6% 7/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	0.00% 0/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Nervous system disorders Paresthesia oral	3.9% 5/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	2.4% 3/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	0.79% 1/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.
Nervous system disorders Somnolence	21.7% 28/129 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	23.0% 29/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.	7.9% 10/126 • 30 days Adverse Events: The Safety Population included all participants in the Randomized Population who received at least 1 dose of double-blind investigational product. One participant enrolled and randomized to the 120 μg group was enrolled a second time at a different site and randomized to the 180 μg group. The data from both enrollments (120 μg and 180 μg) were included in the safety analyses and counted separately in each treatment group; they were counted once when dose groups were combined.

Additional Information

Vice President Head of Clinical Operations

BioXcel Therapeutics

Phone: (475) 355 5177

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place