Trial Outcomes & Findings for A Study to Assess the Efficacy and Safety of AXS-05 in Subjects With Alzheimer's Disease Agitation (NCT NCT05557409)

NCT ID: NCT05557409

Last Updated: 2026-08-13

Results Overview

The Cohen-Mansfield Agitation Inventory (CMAI) is a 29-item caregiver-rated questionnaire that assesses the frequency of agitation-related and disruptive behaviors in subjects with dementia. The scale contains 29 behaviors or items organized into four subscales: physically aggressive, physically non-aggressive, verbally aggressive, and verbally non-aggressive. The CMAI is administered by interviewing the caregiver and asking him or her to rate the frequency with which the subject manifests each behavior using a seven-point scale: 1=never (better outcome), 7=several times an hour (worse outcome). The CMAI total score is the sum of the scores for all of the items in the CMAI. CMAI total scores range from a minimum of 29 (better outcome) to a maximum of 203 (worse outcome).

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

408 participants

Primary outcome timeframe

Up to 5 weeks

Results posted on

2026-08-13

Participant Flow

Participant milestones

Participant milestones
Measure
AXS-05
Up to 5 weeks AXS-05: AXS-05 tablets, taken twice daily
Placebo
Up to 5 weeks Placebo: Placebo tablets, taken twice daily
Overall Study
STARTED
204
204
Overall Study
COMPLETED
194
194
Overall Study
NOT COMPLETED
10
10

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Assess the Efficacy and Safety of AXS-05 in Subjects With Alzheimer's Disease Agitation

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
AXS-05
n=204 Participants
Up to 5 weeks AXS-05: AXS-05 tablets, taken twice daily
Placebo
n=204 Participants
Up to 5 weeks Placebo: Placebo tablets, taken twice daily
Total
n=408 Participants
Total of all reporting groups
Age, Continuous
73.6 years
STANDARD_DEVIATION 5.34 • n=1 Participants
75.0 years
STANDARD_DEVIATION 5.74 • n=1 Participants
74.3 years
STANDARD_DEVIATION 5.58 • n=1 Participants
Sex: Female, Male
Female
130 Participants
n=1 Participants
112 Participants
n=1 Participants
242 Participants
n=1 Participants
Sex: Female, Male
Male
74 Participants
n=1 Participants
92 Participants
n=1 Participants
166 Participants
n=1 Participants
Race/Ethnicity, Customized
Black or African American
18 participants
n=1 Participants
23 participants
n=1 Participants
41 participants
n=1 Participants
Race/Ethnicity, Customized
Asian
2 participants
n=1 Participants
2 participants
n=1 Participants
4 participants
n=1 Participants
Race/Ethnicity, Customized
White
184 participants
n=1 Participants
178 participants
n=1 Participants
362 participants
n=1 Participants
Race/Ethnicity, Customized
Other
0 participants
n=1 Participants
1 participants
n=1 Participants
1 participants
n=1 Participants

PRIMARY outcome

Timeframe: Up to 5 weeks

The Cohen-Mansfield Agitation Inventory (CMAI) is a 29-item caregiver-rated questionnaire that assesses the frequency of agitation-related and disruptive behaviors in subjects with dementia. The scale contains 29 behaviors or items organized into four subscales: physically aggressive, physically non-aggressive, verbally aggressive, and verbally non-aggressive. The CMAI is administered by interviewing the caregiver and asking him or her to rate the frequency with which the subject manifests each behavior using a seven-point scale: 1=never (better outcome), 7=several times an hour (worse outcome). The CMAI total score is the sum of the scores for all of the items in the CMAI. CMAI total scores range from a minimum of 29 (better outcome) to a maximum of 203 (worse outcome).

Outcome measures

Outcome measures
Measure
AXS-05
n=204 Participants
Up to 5 weeks AXS-05: AXS-05 tablets, taken twice daily
Placebo
n=204 Participants
Up to 5 weeks Placebo: Placebo tablets, taken twice daily
Cohen-Mansfield Agitation Inventory (CMAI)
-13.85 score on a scale
Standard Error 0.917
-12.77 score on a scale
Standard Error 0.921

Adverse Events

AXS-05

Serious events: 2 serious events
Other events: 27 other events
Deaths: 0 deaths

Placebo

Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
AXS-05
n=204 participants at risk
Up to 5 weeks AXS-05: AXS-05 tablets, taken twice daily
Placebo
n=204 participants at risk
Up to 5 weeks Placebo: Placebo tablets, taken twice daily
Infections and infestations
Urinary tract infection
0.49%
1/204 • Number of events 1 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
0.00%
0/204 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
General disorders
Asthenia
0.49%
1/204 • Number of events 1 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
0.00%
0/204 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
Nervous system disorders
Cerebrovascular accident
0.49%
1/204 • Number of events 1 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
0.00%
0/204 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).

Other adverse events

Other adverse events
Measure
AXS-05
n=204 participants at risk
Up to 5 weeks AXS-05: AXS-05 tablets, taken twice daily
Placebo
n=204 participants at risk
Up to 5 weeks Placebo: Placebo tablets, taken twice daily
Nervous system disorders
Dizziness
5.9%
12/204 • Number of events 14 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
1.5%
3/204 • Number of events 4 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
Nervous system disorders
Headache
4.4%
9/204 • Number of events 9 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
3.4%
7/204 • Number of events 8 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
Nervous system disorders
Somnolence
2.9%
6/204 • Number of events 6 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).
2.5%
5/204 • Number of events 6 • Treatment emergent adverse events (TEAEs) were collected after administration of study drug through 7 days after the last dose (up to 5 weeks).

Additional Information

Study Director

Axsome Therapeutics

Phone: 212-332-5061

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place