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

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ClinicalTrials.gov Identifier: NCT00420459
Recruitment Status : Completed
First Posted : January 11, 2007
Results First Posted : April 18, 2017
Last Update Posted : April 18, 2017
Sponsor:
Information provided by (Responsible Party):
Indiana University ( Indiana University School of Medicine )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Intervention Drug: Aripiprazole
Enrollment 12
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description Twelve subjects received open-label aripiprazole, mean final dose was 9.8 mg /day.
Period Title: Overall Study
Started 12
Completed 12
Not Completed 0
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Overall Number of Baseline Participants 12
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
<=18 years
10
  83.3%
Between 18 and 65 years
2
  16.7%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 12 participants
14.3
(6 to 25)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 12 participants
Female
2
  16.7%
Male
10
  83.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 12 participants
12
Aberrant Behavior Checklist   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
ABC Irritability S 25.3  (5.1)
ABC Hyperactivity 31.8  (5.8)
ABC Social Withdrawal 16.6  (11.8)
ABC Stereotypy 12.7  (5.6)
ABC Inappropriate Speech 6.8  (5.1)
[1]
Measure Description: 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).
Clinical Global Impressions - Severity   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
4.5  (0.5)
[1]
Measure Description: 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.
Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
14.2  (4.6)
[1]
Measure Description: 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.
Social Responsiveness Scale   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
124.5  (26.7)
[1]
Measure Description: 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
1.Primary Outcome
Title Aberrant Behavior Checklist
Hide Description 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).
Time Frame Obtained at Baseline and Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole. Mean final dose was 9.8 mg/day
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
ABC Irritablity 7.1  (4.4)
ABC Hyperactivity 15.6  (8.1)
ABC Social Withdrawall 9.6  (10.7)
ABC Stereotypy 7.6  (5.6)
ABC Inappropriate Speech 4.0  (3.2)
2.Primary Outcome
Title Clinical Global Impressions- Severity
Hide Description 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.
Time Frame Obtained at Baseline and Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole, mean final dose was 9.8 mg /day.
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
3.5  (0.5)
3.Secondary Outcome
Title The Children's Yale-Brown Obsessive Compulsive Scale
Hide Description 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.
Time Frame Obtained at Baseline and Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
10.6  (5.7)
4.Secondary Outcome
Title Social Responsiveness Scale
Hide Description 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
Time Frame Obtained at Baseline and Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
90.1  (31.6)
5.Secondary Outcome
Title The Vineland Adaptive Behavior Scales
Hide Description 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.
Time Frame Screen Visit
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data for this outcome measure are unavailable because the data file was lost during a server migration.
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
6.Secondary Outcome
Title The Vineland Maladaptive Behavior Subscales
Hide Description 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).
Time Frame Week 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data for this outcome measure are unavailable because the data file was lost during a server migration.
Arm/Group Title Open-label Aripiprazole
Hide Arm/Group Description:
Twelve subjects received open-label aripiprazole for 12 weeks. Mean dose, 9.8 mg/day
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Twelve Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Aripiprazole
Hide Arm/Group Description [Not Specified]
All-Cause Mortality
Aripiprazole
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Aripiprazole
Affected / at Risk (%)
Total   0/12 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Aripiprazole
Affected / at Risk (%)
Total   12/12 (100.00%) 
Gastrointestinal disorders   
Nausea/Vomiting  4/12 (33.33%) 
Diarrhea  1/12 (8.33%) 
Constipation  1/12 (8.33%) 
Increased Appetite  1/12 (8.33%) 
General disorders   
Tiredness  7/12 (58.33%) 
Drooling  3/12 (25.00%) 
Slurred Speech  2/12 (16.67%) 
Nervous system disorders   
Akathisia  1/12 (8.33%) 
Psychiatric disorders   
Increased Anxiety  2/12 (16.67%) 
Renal and urinary disorders   
Enuresis  2/12 (16.67%) 
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Craig Erickson
Organization: Cincinnati Childrens Hospital
Phone: 5136366265
Responsible Party: Indiana University ( Indiana University School of Medicine )
ClinicalTrials.gov Identifier: NCT00420459     History of Changes
Other Study ID Numbers: 0609-22
First Submitted: January 9, 2007
First Posted: January 11, 2007
Results First Submitted: July 29, 2015
Results First Posted: April 18, 2017
Last Update Posted: April 18, 2017