Trial Outcomes & Findings for An Open-Label Trial of Aripiprazole in Autism Spectrum Disorders (NCT NCT00308074)

NCT ID: NCT00308074

Last Updated: 2017-02-15

Results Overview

The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to evaluate how much the patient's illness has improved or worsened compared to their baseline condition at the beginning of the intervention. The ratings are evaluated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

Baseline, Endpoint using last observation carried forward (LOCF) at weeks 3,5,7,9,11 and 13.

Results posted on

2017-02-15

Participant Flow

Recruitment will take place from mainly the Cambridge Health Alliance clinics, and the McLean Hospital affiliated service site for children and adolescents with an autism spectrum disorder (ASD), but also from other healthcare facilities in the vicinity.

All individuals enrolled will have Diagnostic and Statistical Manual Fourth Edition (DSM-IV) diagnoses of Autism, Asperger's Disorder or Pervasive Developmental Disorder, not otherwise specified (PDD NOS) and will be enrolled in this 12-week open-label study

Participant milestones

Participant milestones
Measure
Aripiprazole
aripiprazole monotherapy Aripiprazole will be started at 2.5 or 5mg depending on clinical impression and severity of aggression and agitation. The dose will be evaluated weekly, according to clinical impression and adjusted if deemed appropriate, in not more than 5mg increments . The lowest effective dose will be used.
Overall Study
STARTED
14
Overall Study
COMPLETED
13
Overall Study
NOT COMPLETED
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Aripiprazole
aripiprazole monotherapy Aripiprazole will be started at 2.5 or 5mg depending on clinical impression and severity of aggression and agitation. The dose will be evaluated weekly, according to clinical impression and adjusted if deemed appropriate, in not more than 5mg increments . The lowest effective dose will be used.
Overall Study
Withdrawal by Subject
1

Baseline Characteristics

An Open-Label Trial of Aripiprazole in Autism Spectrum Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Aripiprazole
n=14 Participants
aripiprazole monotherapy
Age, Categorical
<=18 years
14 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Age, Continuous
13 years
STANDARD_DEVIATION 1.66 • n=5 Participants
Gender
Female
5 Participants
n=5 Participants
Gender
Male
9 Participants
n=5 Participants
Region of Enrollment
United States
14 participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Endpoint using last observation carried forward (LOCF) at weeks 3,5,7,9,11 and 13.

The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to evaluate how much the patient's illness has improved or worsened compared to their baseline condition at the beginning of the intervention. The ratings are evaluated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse.

Outcome measures

Outcome measures
Measure
Aripiprazole
n=14 Participants
aripiprazole monotherapy
Clinical Global Impressions-Improvement
1.8 units on a scale
Standard Deviation 1.6

PRIMARY outcome

Timeframe: Baseline, Endpoint using last observation carried forward (LOCF) at weeks 3,5,7,9,11 and 13.

Aberrant Behavior Checklist (ABC) The ABC is a 58 item symptom checklist for assessing problem behaviors in individuals ages 6 to 54 with mental retardation. Items are rated on a 4-point scale (0=no problem to 3=severe problem). A decrease in score indicates improvement. There are five subscales: a) Irritability and Agitation b) Lethargy and Social Withdrawal c) Stereotypic Behavior d) Hyperactivity and Noncompliance and e) Inappropriate Speech.This study uses the Irritability subscale for its outcome. The Irritability subscale is the sum of 15 items. Each item is rated using the scale: 0 = Not at all; 1 = Slight in degree; 2 = Moderately serious; and 3 = Severe in degree. The Irritability subscale total score ranges from 0 to 45. A decrease in score over time indicates improvement.

Outcome measures

Outcome measures
Measure
Aripiprazole
n=14 Participants
aripiprazole monotherapy
Aberrant Behavior Checklist-Irritability Subscale
Baseline irritability score
24.5 units on a scale
Standard Deviation 10.5
Aberrant Behavior Checklist-Irritability Subscale
Endpoint irritability score
8 units on a scale
Standard Deviation 7.9

SECONDARY outcome

Timeframe: Baseline, Endpoint using last observation carried forward (LOCF) at weeks 3,5,7,9,11 and 13.

10-item assessment of obsessive-compulsive symptoms in patients less than 18 years of age. There are 5 items pertaining to compulsions rate symptoms (time spent, interference with functioning, distress, resistance, control) on a 5-point scale ( from 0=no symptoms/minimum severity, to 4=extreme symptoms/maximum severity). Total is the sum of 10 items. The range of possible totals is 0 (no symptoms) to 40 (severe). A decrease in value indicates improvement.

Outcome measures

Outcome measures
Measure
Aripiprazole
n=14 Participants
aripiprazole monotherapy
Yale-Brown Obsessive Compulsive Scale (Y-BOCS)
Baseline score
15.9 units on a scale
Standard Deviation 9.5
Yale-Brown Obsessive Compulsive Scale (Y-BOCS)
Endpoint score
9.9 units on a scale
Standard Deviation 6.9

SECONDARY outcome

Timeframe: Baseline, Endpoint using last observation carried forward (LOCF) at weeks 3,5,7,9,11 and 13.

The Brief Psychiatric Rating Scale for Children is a 21-item rating scale to evaluate psychiatric problems based on the clinician' s interview with the child/adolescent and parents. It has 7 scales: behavioral problems, depression, thought disorders, psychomotor excitation, withdrawal-retardation, anxiety, organicity. Ratings are based on a 7 point scale, from "Not Present" (scores 0) to "Extremely Severe" (scores 6 points). Total is the sum of the 21 items. The range of possible totals is 0 (no symptoms) to 126 (extremely severe).A decrease in score indicates improvement.

Outcome measures

Outcome measures
Measure
Aripiprazole
n=14 Participants
aripiprazole monotherapy
Brief Psychiatric Rating Scale for Children (BPRS-C)
Baseline score
69.0 units on a scale
Standard Deviation 17.4
Brief Psychiatric Rating Scale for Children (BPRS-C)
Endpoint score
36.1 units on a scale
Standard Deviation 14.9

Adverse Events

Aripiprazole

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
Aripiprazole
n=14 participants at risk
aripiprazole monotherapy
Blood and lymphatic system disorders
neutropenia
7.1%
1/14 • Number of events 1 • 2 years, 10 months
Labs, vital signs, ECG and the MOSES were collected at scheduled intervals.

Additional Information

Dr. Jean A. Frazier

University of Massachusetts Medical School

Phone: 508-856-5896

Results disclosure agreements

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