Trial Outcomes & Findings for A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome (NCT NCT00420459)

NCT ID: NCT00420459

Last Updated: 2017-04-18

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Obtained at Baseline and Week 12

Results posted on

2017-04-18

Participant Flow

Participant milestones

Participant milestones
Measure	Open-label Aripiprazole Twelve subjects received open-label aripiprazole, mean final dose was 9.8 mg /day.
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Open-label Aripiprazole n=12 Participants Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Age, Categorical <=18 years	10 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	14.3 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants
Aberrant Behavior Checklist ABC Irritability S	25.3 units on a scale STANDARD_DEVIATION 5.1 • n=5 Participants
Aberrant Behavior Checklist ABC Hyperactivity	31.8 units on a scale STANDARD_DEVIATION 5.8 • n=5 Participants
Aberrant Behavior Checklist ABC Social Withdrawal	16.6 units on a scale STANDARD_DEVIATION 11.8 • n=5 Participants
Aberrant Behavior Checklist ABC Stereotypy	12.7 units on a scale STANDARD_DEVIATION 5.6 • n=5 Participants
Aberrant Behavior Checklist ABC Inappropriate Speech	6.8 units on a scale STANDARD_DEVIATION 5.1 • n=5 Participants
Clinical Global Impressions - Severity	4.5 units on a scale STANDARD_DEVIATION 0.5 • n=5 Participants
Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD	14.2 units on a scale STANDARD_DEVIATION 4.6 • n=5 Participants
Social Responsiveness Scale	124.5 units on a scale STANDARD_DEVIATION 26.7 • n=5 Participants

PRIMARY outcome

Timeframe: Obtained at Baseline and Week 12

Outcome measures

Outcome measures
Measure	Open-label Aripiprazole n=12 Participants Twelve subjects received open-label aripiprazole. Mean final dose was 9.8 mg/day
Aberrant Behavior Checklist ABC Irritablity	7.1 units on a scale Standard Deviation 4.4
Aberrant Behavior Checklist ABC Hyperactivity	15.6 units on a scale Standard Deviation 8.1
Aberrant Behavior Checklist ABC Social Withdrawall	9.6 units on a scale Standard Deviation 10.7
Aberrant Behavior Checklist ABC Stereotypy	7.6 units on a scale Standard Deviation 5.6
Aberrant Behavior Checklist ABC Inappropriate Speech	4.0 units on a scale Standard Deviation 3.2

PRIMARY outcome

Timeframe: Obtained at Baseline and Week 12

The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.

Outcome measures

Outcome measures
Measure	Open-label Aripiprazole n=12 Participants Twelve subjects received open-label aripiprazole. Mean final dose was 9.8 mg/day
Clinical Global Impressions- Severity	3.5 units on a scale Standard Deviation 0.5

SECONDARY outcome

Timeframe: Obtained at Baseline and Week 12

The CY-BOCS PDD has been utilized in a largescale clinical treatment study of repetitive behavior in idiopathic ASDs. CYBOCS-PDD scores range from 0 to 20 and measure repetitive/compulsive behavior and not obsessions. Higher score indicate worse outcome.

Outcome measures

Outcome measures
Measure	Open-label Aripiprazole n=12 Participants Twelve subjects received open-label aripiprazole. Mean final dose was 9.8 mg/day
The Children's Yale-Brown Obsessive Compulsive Scale	10.6 units on a scale Standard Deviation 5.7

SECONDARY outcome

Timeframe: Obtained at Baseline and Week 12

The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment

Outcome measures

Outcome measures
Measure	Open-label Aripiprazole n=12 Participants Twelve subjects received open-label aripiprazole. Mean final dose was 9.8 mg/day
Social Responsiveness Scale	90.1 units on a scale Standard Deviation 31.6

SECONDARY outcome

Timeframe: Screen Visit

Population: Data for this outcome measure are unavailable because the data file was lost during a server migration.

Vineland Adaptive Behavior Scales are a valid and reliable test to measure a person's adaptive level of functioning. Vineland-II forms aid in diagnosing and classifying intellectual and developmental disabilities and other disorders, such as autism spectrum disorders and developmental delays. The content and scales are organized within a 4 domain structure: Communication, Daily Living, Socialization and Motor Skills. The adaptive behavior composite standard score is computed from the sum of standard scores from the domains and converted into the adaptive behavior composite standard score. Higher scores indicate a higher adaptive level of functioning.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Week 12

Population: Data for this outcome measure are unavailable because the data file was lost during a server migration.

The Vineland Adaptive Behavior Scales include an optional Maladaptive Behavior Index with 27 items. The Maladaptive Behavior Index is a composite of Internalizing, Externalizing, and other types of undesirable behavior that may interfere with the individual's adaptive functioning. The Maladaptive Behavior subscale yields raw scores (0-27).

Outcome measures

Outcome data not reported

Adverse Events

Aripiprazole

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Aripiprazole n=12 participants at risk
General disorders Tiredness	58.3% 7/12 • Twelve Weeks
Gastrointestinal disorders Nausea/Vomiting	33.3% 4/12 • Twelve Weeks
General disorders Drooling	25.0% 3/12 • Twelve Weeks
General disorders Slurred Speech	16.7% 2/12 • Twelve Weeks
Psychiatric disorders Increased Anxiety	16.7% 2/12 • Twelve Weeks
Renal and urinary disorders Enuresis	16.7% 2/12 • Twelve Weeks
Gastrointestinal disorders Diarrhea	8.3% 1/12 • Twelve Weeks
Gastrointestinal disorders Constipation	8.3% 1/12 • Twelve Weeks
Nervous system disorders Akathisia	8.3% 1/12 • Twelve Weeks
Gastrointestinal disorders Increased Appetite	8.3% 1/12 • Twelve Weeks

Additional Information

Craig Erickson

Cincinnati Childrens Hospital

Phone: 5136366265

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place