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Trial record 37 of 74 for:    "fragile x syndrome"

Acamprosate in Youth With Fragile X Syndrome

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ClinicalTrials.gov Identifier: NCT01300923
Recruitment Status : Completed
First Posted : February 23, 2011
Results First Posted : April 28, 2017
Last Update Posted : July 30, 2019
Sponsor:
Information provided by (Responsible Party):
Indiana University

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Fragile X Syndrome
Autism Spectrum Disorders
Intervention Drug: Acamprosate
Enrollment 14
Recruitment Details  
Pre-assignment Details The psychophysiological measures proved non-feasible as subjects could not tolerate the procedures. The paradigms were tried on two subjects in the Autism Spectrum Disorder arm; however, the data that was collected was not usable. Subsequently, neurobiological characterization was halted.
Arm/Group Title Acamprosate Autism Spectrum Disorder (ADS)
Hide Arm/Group Description

The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg.

Acamprosate

This baseline comparison group will participated in only and biomarker portion of subject characterization.
Period Title: Overall Study
Started 12 2
Completed 12 0
Not Completed 0 2
Reason Not Completed
Physician Decision             0             2
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 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
12
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 12 participants
11.9
(6 to 15)
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 9.9  (7.8)
ABC Social Withdrawal 8.8  (5.8)
ABC Stereotypy 6.8  (6.8)
ABC Hyperactivity 16.8  (9.1)
ABC Inappropriate Speech 5.2  (3.5)
ABC Social Avoidance 3.3  (2.6)
[1]
Measure Description: Aberrant Behavior Checklist (ABC) is a 58-item rating scale assessing maladaptive behaviors across 5 original subscales: Irritability(15 items from 0-45), Social Withdrawal(16 items from 0-48), Stereotypy(7 items from 0-21), Hyperactivity(16 items from 0-48), Inappropriate Speech(4 items from 0-12). Also reported is Social Avoidance, a newly developed 4-item subscale (from 0-12) that captures core social avoidance aspects of Fragile X Syndrome. Items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors.
Clinical Global Impression -Severity   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
4.25  (0.45)
[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
11.1  (2.6)
[1]
Measure Description: The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change.
Social Responsiveness Scale   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
91.3  (27.4)
[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
ADHD Rating Scale 4th Edition   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
23.6  (10.6)
[1]
Measure Description: The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity.
Peabody Picture Vocabulary Test   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
85.2  (32.0)
[1]
Measure Description: The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank.
Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 12 participants
Communication Domain 63.4  (10.0)
Expressive Communication Domain 69.8  (23.0)
[1]
Measure Description: The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest to this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is rated from 0 (never performs the behavior) to 2 (usually performs behavior independently). Higher scores indicate higher adaptive functioning.
Brain-derived neurotropic factor   [1] 
Mean (Standard Deviation)
Unit of measure:  pg/mL
Number Analyzed 12 participants
790.4  (1.3050)
[1]
Measure Description: Brain-derived neurotrophic factor (BDNF) is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses.
1.Primary Outcome
Title Clinical Global Impression- Severity Scale (CGI-S)
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 Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day).
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
7.0  (8.9)
2.Secondary Outcome
Title The Aberrant Behavior Checklist (ABC)
Hide Description The Aberrant Behavior Checklist (ABC) is a 58-item rating scale used to assess maladaptive behaviors across five original subscales: Irritability (15 items from 0-45), Social Withdrawal (16 items from 0-48), Stereotypy (7 items from 0-21), Hyperactivity (16 items from 0-48), Inappropriate Speech (4 items from 0-12). Additionally, Social Avoidance, a newly developed four-item subscale (from 0-12) of the ABC that captures core social avoidance aspects of Fragile X Syndrome is reported. All items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors. Differences between Baseline and Week 10 are used as an indicator of change.
Time Frame Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
ABC Irritability 7.0  (8.9)
ABC Social Withdrawal 4.1  (6.5)
ABC Stereotypy 6.0  (6.3)
ABC Hyperactivity 11.0  (8.6)
ABC Inappropriate Speech 4.8  (3.4)
ABC Social Avoidance 1.6  (2.7)
3.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 Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
76.4  (26.8)
4.Secondary Outcome
Title Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD
Hide Description The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change.
Time Frame Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
9.8  (4.1)
5.Secondary Outcome
Title ADHD Rating Scale 4th Edition
Hide Description The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity.
Time Frame Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
16.7  (8.0)
6.Secondary Outcome
Title Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain
Hide Description The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest in this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is scored from 0 (never performs the behavior) to 3 (usually performs the behavior independently). Higher scores indicate higher adaptive functioning. Differences between Baseline and Week 10 are used as an indicator of change.
Time Frame Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day).
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
Communication Domain 66.6  (11.2)
Expressive Communication 78.9  (21.2)
7.Secondary Outcome
Title Peabody Picture Vocabulary
Hide Description The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank.
Time Frame Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 12
Mean (Standard Deviation)
Unit of Measure: units on a scale
83.3  (32.0)
8.Secondary Outcome
Title Brain-derived Neurotrophic Factor (BDNF)
Hide Description BDNF is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses.
Time Frame Screen and Week 10
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Acamprosate Treatment Group
Hide Arm/Group Description:
Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: pg/mL
1,007.6  (1,493.2)
Time Frame Ten Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Acamprosate
Hide Arm/Group Description The maximum dose of acamprosate to be used in this study is 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg.
All-Cause Mortality
Acamprosate
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Acamprosate
Affected / at Risk (%) # Events
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%
Acamprosate
Affected / at Risk (%) # Events
Total   9/12 (75.00%)    
Gastrointestinal disorders   
Diarrhea  1/12 (8.33%)  1
mild constipation  1/12 (8.33%)  1
Infections and infestations   
Rhinitis  1/12 (8.33%)  1
Psychiatric disorders   
Irritability  4/12 (33.33%)  4
Increased repetitive behavior  2/12 (16.67%)  2
Increased anxiety  1/12 (8.33%)  1
Insomnia  1/12 (8.33%)  1
Nightmares  1/12 (8.33%)  1
Renal and urinary disorders   
Urinary Urgency  1/12 (8.33%)  1
Psychophysiological measures proved non-feasible as subjects could not tolerate the procedures. The paradigms were tried on 2 subjects in the ADS arm, as data collected was not usable. Subsequently, neurobiological characterization was halted.
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
ClinicalTrials.gov Identifier: NCT01300923     History of Changes
Other Study ID Numbers: 1003-26
First Submitted: August 25, 2010
First Posted: February 23, 2011
Results First Submitted: July 29, 2015
Results First Posted: April 28, 2017
Last Update Posted: July 30, 2019