MedTrace Logo

Trial Outcomes & Findings for Nicotinic Cholinergic Modulation as a Novel Treatment Strategy for Aggression Associated With Autism (NCT NCT02552147)

NCT ID: NCT02552147

Last Updated: 2022-05-06

Results Overview

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-irritability subscale measures irritability and related symptoms and the subscale score range is 0 (least symptomatic) to 45 (most symptomatic). The ABC is completed by parents/caregivers.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

Baseline and 7 days.

Results posted on

2022-05-06

Participant Flow

Participant milestones

Participant milestones
Measure
Transdermal Placebo First, Nicotine Last
Subject will receive transdermal placebo daily for 7 days, placebo patch for another 7 days, then transdermal nicotine 7 mg daily for a final 7 days. Transdermal nicotine Transdermal placebo
Transdermal Nicotine First, Placebo Last
Subject will receive transdermal nicotine 7 mg daily for 7 days, placebo patch for 7 days, then placebo patch for a final 7 days. Transdermal nicotine Transdermal placebo
First Intervention (7 Days)
STARTED
4
4
First Intervention (7 Days)
COMPLETED
3
4
First Intervention (7 Days)
NOT COMPLETED
1
0
Washout (7 Days)
STARTED
3
4
Washout (7 Days)
COMPLETED
3
4
Washout (7 Days)
NOT COMPLETED
0
0
Second Intervention (7 Days)
STARTED
3
4
Second Intervention (7 Days)
COMPLETED
1
4
Second Intervention (7 Days)
NOT COMPLETED
2
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Transdermal Placebo First, Nicotine Last
Subject will receive transdermal placebo daily for 7 days, placebo patch for another 7 days, then transdermal nicotine 7 mg daily for a final 7 days. Transdermal nicotine Transdermal placebo
Transdermal Nicotine First, Placebo Last
Subject will receive transdermal nicotine 7 mg daily for 7 days, placebo patch for 7 days, then placebo patch for a final 7 days. Transdermal nicotine Transdermal placebo
First Intervention (7 Days)
Protocol Violation
1
0
Second Intervention (7 Days)
Protocol Violation
2
0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
All Study Participants
n=7 Participants
Participants randomized to receive either transdermal nicotine or placebo
Age, Continuous
24 years
STANDARD_DEVIATION 3 • n=7 Participants
Sex: Female, Male
Female
1 Participants
n=7 Participants
Sex: Female, Male
Male
6 Participants
n=7 Participants
Region of Enrollment
United States
7 Participants
n=7 Participants
Aberrant Behavior Checklist-Irritability
25 units on a scale
STANDARD_DEVIATION 8 • n=7 Participants
Aberrant Behavior Checklist-Lethargy/Social withdrawal
20 units on a scale
STANDARD_DEVIATION 13 • n=7 Participants
Aberrant Behavior Checklist-Stereotypic behavior
9 units on a scale
STANDARD_DEVIATION 7 • n=7 Participants
Aberrant Behavior Checklist-Hyperactivity
26 units on a scale
STANDARD_DEVIATION 11 • n=7 Participants
Aberrant Behavior Checklist-Inappropriate speech
6 units on a scale
STANDARD_DEVIATION 4 • n=7 Participants
Social Responsiveness Scale-2
177 units on a scale
STANDARD_DEVIATION 25 • n=7 Participants
Number on antipsychotic medication
5 Participants
n=7 Participants

PRIMARY outcome

Timeframe: Baseline and 7 days.

Population: All participants who completing all study visits and for which ratings scales were completed in accordance with study protocol.

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-irritability subscale measures irritability and related symptoms and the subscale score range is 0 (least symptomatic) to 45 (most symptomatic). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Change From Baseline in Aberrant Behavior Checklist Irritability Subscale (ABC-I)
-12 units on a scale
Standard Deviation 3
-7 units on a scale
Standard Deviation 8

SECONDARY outcome

Timeframe: Baseline and 7 days.

Population: All participants completing all study visits and for which ratings scales were completed in accordance with study protocol.

The Social Responsiveness Scale (SRS) - 2 quantifies impairments in social behaviors seen in autism spectrum disorder and related conditions and their severity. The version used in this study is completed by parents/caregivers. The raw score range is 0 (unaffected) to \> 134 (extremely affected). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Change in Social Responsiveness Scale-Adults (SRS-A)
-1 units on a scale
Standard Deviation 25
-15 units on a scale
Standard Deviation 23

SECONDARY outcome

Timeframe: Baseline and 7 days

Population: Data were not collected.

Brief qualitative reports of the patient's subjective experience will be recorded by asking "In what ways did you find the patch helpful or harmful during the past week, if at all?".

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and one week

Population: Data were not collected.

Subjects will perform a computerized frustration-induction task and complete a rating scale of irritability and frustration levels.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and day 7

Population: Data were not collected.

The State-Trait Anxiety Inventory (STAI) is a measure of trait and state anxiety. It ranges from a score of 20 (least affected) to 80 (most affected).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 7

Caregivers rated nightly sleep quality from 0 (worst) to 10 (best). Average rating for each treatment week is compared.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Nightly Sleep Quality
10 units on a scale
Standard Deviation 0.2
9 units on a scale
Standard Deviation 2

POST_HOC outcome

Timeframe: Baseline and 7 days

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-lethargy/social withdrawal subscale measures lethargy/social withdrawal and related symptoms and the subscale score range is 0 (least symptomatic) to 48 (most symptomatic). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Aberrant Behavior Checklist - Lethargy/Social Withdrawal Subscale Change From Baseline
-2 units on a scale
Standard Deviation 3
-7 units on a scale
Standard Deviation 4

POST_HOC outcome

Timeframe: Baseline and 7 days

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-stereotypic behavior subscale measures stereotypic behaviors and related symptoms and the subscale score range is 0 (least symptomatic) to 21 (most symptomatic). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Aberrant Behavior Checklist - Stereotypic Behavior Subscale Change From Baseline
-2 units on a scale
Standard Deviation 3
-4 units on a scale
Standard Deviation 4

POST_HOC outcome

Timeframe: Baseline and 7 days

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-hyperactivity subscale measures hyperactivity and related symptoms and the subscale score range is 0 (least symptomatic) to 48 (most symptomatic). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Aberrant Behavior Checklist - Hyperactivity Subscale Change From Baseline
-7 units on a scale
Standard Deviation 6
-6 units on a scale
Standard Deviation 6

POST_HOC outcome

Timeframe: Baseline and 7 days

Aberrant Behavior Checklist (ABC) measures symptom subscales for individuals with neurodevelopmental disorders. The ABC-Inappropriate speech subscale measures Inappropriate speech and related symptoms and the subscale score range is 0 (least symptomatic) to 12 (most symptomatic). The ABC is completed by parents/caregivers.

Outcome measures

Outcome measures
Measure
Nicotine
n=5 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=5 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Aberrant Behavior Checklist - Inappropriate Speech Subscale Change From Baseline
-1 units on a scale
Standard Deviation 2
0.2 units on a scale
Standard Deviation 0.8

POST_HOC outcome

Timeframe: Baseline and 7 days

Caregivers asked which treatment week showed the greatest improvement in aggressive or irritable behavior

Outcome measures

Outcome measures
Measure
Nicotine
n=6 Participants
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=6 Participants
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Global Aggressive Behavior Improvement
4 Participants
2 Participants

Adverse Events

Nicotine

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

Placebo

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

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Nicotine
n=7 participants at risk
Subject receiving transdermal nicotine in either the first or last week of the study and completing all study visits.
Placebo
n=7 participants at risk
Subject receiving transdermal placebo in either the first or last week of the study and completing all study visits.
Nervous system disorders
Nightmare
0.00%
0/7 • Duration of trial (3 weeks).
14.3%
1/7 • Number of events 1 • Duration of trial (3 weeks).
Gastrointestinal disorders
Dyspepsia
0.00%
0/7 • Duration of trial (3 weeks).
14.3%
1/7 • Number of events 1 • Duration of trial (3 weeks).

Additional Information

Alan Lewis, MD, PhD

Vanderbilt University Medical Center

Phone: (615) 936-4997

Results disclosure agreements

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