Trial Outcomes & Findings for Study of Paliperidone ER in Adolescents and Young Adults With Autism (NCT NCT00549562)

NCT ID: NCT00549562

Last Updated: 2017-05-16

Results Overview

The CGI Global Improvement (CGI-I) is a clinician-rate scale designed to take into account all factors to arrive at an assessment of severity and response to treatment, including parent report, parent-rated measures, teacher-rated measures, and clinician-rated measures. The CGI-I is rated from 1 to 7 (1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; 7 = very much worse) at a single time-point.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

25 participants

Primary outcome timeframe

Week 8

Results posted on

2017-05-16

Participant Flow

Participant milestones

Participant milestones
Measure
Paliperidone ER
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
Overall Study
STARTED
25
Overall Study
COMPLETED
23
Overall Study
NOT COMPLETED
2

Reasons for withdrawal

Reasons for withdrawal
Measure
Paliperidone ER
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
Overall Study
Adverse Event
1
Overall Study
Lack of Efficacy
1

Baseline Characteristics

Study of Paliperidone ER in Adolescents and Young Adults With Autism

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
Age, Categorical
<=18 years
20 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
5 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
15.36 Years
n=5 Participants
Sex: Female, Male
Female
4 Participants
n=5 Participants
Sex: Female, Male
Male
21 Participants
n=5 Participants
Region of Enrollment
United States
25 participants
n=5 Participants
Clinical Global Impressions-Improvement (CGI-I)
4.0 units on a scale
STANDARD_DEVIATION 0.0 • n=5 Participants
The Aberrant Behavior Checklist
ABC-Irritability
30.3 units on a scale
STANDARD_DEVIATION 6.5 • n=5 Participants
The Aberrant Behavior Checklist
ABC-Hyperactivity
34.7 units on a scale
STANDARD_DEVIATION 8.9 • n=5 Participants
The Aberrant Behavior Checklist
ABC-Social Withdrawal
17.2 units on a scale
STANDARD_DEVIATION 12.5 • n=5 Participants
The Aberrant Behavior Checklist
ABC-Stereotypy
12.5 units on a scale
STANDARD_DEVIATION 6.7 • n=5 Participants
The Aberrant Behavior Checklist
ABC-Inappropriate Speech
7.5 units on a scale
STANDARD_DEVIATION 4.4 • n=5 Participants
Children's Yale-Brown Obsessive Compulsive Scale Modified for Pervasive Developmental Disorders
16 units on a scale
STANDARD_DEVIATION 3 • n=5 Participants
The Social Responsiveness Scale
122.6 units on a scale
STANDARD_DEVIATION 18.4 • n=5 Participants
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subscale Part I
28.9 units on a scale
STANDARD_DEVIATION 6.3 • n=5 Participants
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subscale Part II
8.8 units on a scale
STANDARD_DEVIATION 6.4 • n=5 Participants
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subscale Total
37.4 units on a scale
STANDARD_DEVIATION 8.2 • n=5 Participants

PRIMARY outcome

Timeframe: Week 8

The CGI Global Improvement (CGI-I) is a clinician-rate scale designed to take into account all factors to arrive at an assessment of severity and response to treatment, including parent report, parent-rated measures, teacher-rated measures, and clinician-rated measures. The CGI-I is rated from 1 to 7 (1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; 7 = very much worse) at a single time-point.

Outcome measures

Outcome measures
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
The Clinical Global Impression-Improvement(CGI-I)
1.8 units on a scale
Standard Deviation 1.3

PRIMARY outcome

Timeframe: Week 8

The Aberrant Behavior Checklist (ABC) is a 58-item measure of maladaptive behaviors and is used as a measure of drug effects. Each item is rated from 0 (not at all to 3 (severe). The ABC has 5 subscales:Irritability (15 items) ranging from 0 (not at all) to 45 (severe), Hyperactivity (16 items) ranging from 0 (not at all) to 48 (severe), Social Withdrawal (16 items) ranging from 0 (not at all) to 48 (severe), Stereotypy (7 items) ranging from 0 (not at all) to 21 (severe) and Inappropriate Speech (4 items) ranging from 0 (not at all) to 12 (severe).

Outcome measures

Outcome measures
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
The Aberrant Behavior Checklist
ABC-Social Withdrawal
7.6 units on a scale
Standard Deviation 8.8
The Aberrant Behavior Checklist
ABC-Stereotypy
6.4 units on a scale
Standard Deviation 5.1
The Aberrant Behavior Checklist
ABC-Inappropriate Speech
3.4 units on a scale
Standard Deviation 3.8
The Aberrant Behavior Checklist
ABC-Irritability
12.6 units on a scale
Standard Deviation 9.1
The Aberrant Behavior Checklist
ABC-Hyperactivity
17.4 units on a scale
Standard Deviation 11.5

SECONDARY outcome

Timeframe: Week 8

The Children's Yale-Brown Obsessive Compulsive Scale Modified for Pervasive Developmental Disorders (CY-BOCS-PDD) is semi-structured clinician rating scale designed to rate the current severity of repetitive behavior in children and adolescents with PDD. The scale consists of 5 items: Time Spent, Interference, Distress, Resistance and Control. Each item is scored from 0 (None) to 4 (Extreme). The scale yields a Total Score from 0 (least symptomatic) to 20 (most symptomatic). Higher scores indicate greater severity of repetitive behavior.

Outcome measures

Outcome measures
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
The Children's Yale-Brown Obsessive Compulsive Scale Modified for Pervasive Developmental Disorders
11.9 units on a scale
Standard Deviation 4.0

SECONDARY outcome

Timeframe: Week 8

The Social Responsiveness Scale (SRS) is a 65-item parent completed scale that assesses social awareness, social cognition, social communication, social motivation and autistic mannerisms. Each item is scored from 1 (not true) to 3 (almost always true). Interpretation in this study is based on a total score that is proportional to the level of impairment in reciprocal social behavior. Scores within 0-53 are within normal limits. Scores within 54-86 indicate mild to moderate impairment. Scores above 87 indicate severe impairment.

Outcome measures

Outcome measures
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
The Social Responsiveness Scale
100.9 units on a scale
Standard Deviation 20.4

SECONDARY outcome

Timeframe: Week 8

The VABS Maladaptive Behavior Domain measures undesirable behaviors that may interfere with an individual's adaptive functioning. The Maladaptive Domain consists two parts. Part I contains 27 minor maladaptive items and Part II contains 9 serious maladaptive behaviors. Each item is scored from 0 (never or seldom engages in the activity) to 2(usually or habitually engages in the activity). Part 1 yields a score of 0 to 54. Part II yields a score of 0 to 18. Both parts are combined to make a Total Score of 0 to 72. High scores of maladaptive behaviors reflect more negative behavior.

Outcome measures

Outcome measures
Measure
Paliperidone ER
n=25 Participants
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subscale-Part I
19.0 units on a scale
Standard Deviation 6.9
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subjscale Part II
6.4 units on a scale
Standard Deviation 3.4
The Vineland Adaptive Behavior Scales (VABS) - Maladaptive Behavior Domain
VABS Maladaptive Behavior Subscale Total
25.1 units on a scale
Standard Deviation 8.8

Adverse Events

Paliperidone ER

Serious events: 0 serious events
Other events: 21 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Paliperidone ER
n=25 participants at risk
8-Week Open-Label Paliperidone ER: Starting dose is 3 mg per day, with the option to increase the dose by 3 mg/day on a weekly basis for 4 weeks to a maximum dose of 12 mg per day.
Gastrointestinal disorders
Excessive Appetite
36.0%
9/25 • Number of events 9 • Eight Weeks
Metabolism and nutrition disorders
Weight Gain
36.0%
9/25 • Number of events 9 • Eight Weeks
General disorders
Tiredness
28.0%
7/25 • Number of events 7 • Eight Weeks
General disorders
Rhinitis/cough
32.0%
8/25 • Number of events 8 • Eight Weeks
Psychiatric disorders
Insomnia
12.0%
3/25 • Number of events 3 • Eight Weeks
Gastrointestinal disorders
Constipation
12.0%
3/25 • Number of events 3 • Eight Weeks
General disorders
Excessive saliva
8.0%
2/25 • Number of events 2 • Eight Weeks
Skin and subcutaneous tissue disorders
Rash
8.0%
2/25 • Number of events 2 • Eight Weeks
Gastrointestinal disorders
Diarrhea
8.0%
2/25 • Number of events 2 • Eight Weeks
Nervous system disorders
Tremor
4.0%
1/25 • Number of events 1 • Eight Weeks
Renal and urinary disorders
Enuresis
4.0%
1/25 • Number of events 1 • Eight Weeks
Nervous system disorders
Headache
4.0%
1/25 • Number of events 1 • Eight Weeks
Cardiac disorders
Palpitations
4.0%
1/25 • Number of events 1 • Eight Weeks
Nervous system disorders
Tic
4.0%
1/25 • Number of events 1 • Eight Weeks
Nervous system disorders
Akathisia
4.0%
1/25 • Number of events 1 • Eight Weeks
General disorders
Dry Mouth
4.0%
1/25 • Number of events 1 • Eight Weeks
Gastrointestinal disorders
Dyspepsia
4.0%
1/25 • Number of events 1 • Eight Weeks

Additional Information

David D. Dunn MD

Indiana University School of Medicine

Phone: 317-944-8164

Results disclosure agreements

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