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Acamprosate in Youth With Fragile X Syndrome

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Indiana University
ClinicalTrials.gov Identifier:
NCT01300923
First received: August 25, 2010
Last updated: April 27, 2017
Last verified: April 2017
Results First Received: July 29, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: No masking;   Primary Purpose: Treatment
Conditions: Fragile X Syndrome
Autism Spectrum Disorders
Intervention: Drug: Acamprosate

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
Acamprosate

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

Autism Spectrum Disorder (ADS) This baseline comparison group will participated in only and biomarker portion of subject characterization.

Participant Flow:   Overall Study
    Acamprosate   Autism Spectrum Disorder (ADS)
STARTED   12   2 
COMPLETED   12   0 
NOT COMPLETED   0   2 
Physician Decision                0                2 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Acamprosate Treatment Group Twelve subjects received open-label acamprosate, mean final of 1,054 mg/day (range: 666-1,998 mg/day)

Baseline Measures
   Acamprosate Treatment Group 
Overall Participants Analyzed 
[Units: Participants]
 12 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      12 100.0% 
Between 18 and 65 years      0   0.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Full Range)
 11.9 
 (6 to 15) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      2  16.7% 
Male      10  83.3% 
Region of Enrollment 
[Units: Participants]
 
United States   12 
Aberrant Behavior Checklist [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 
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] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 4.25  (0.45) 
[1] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 11.1  (2.6) 
[1] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 91.3  (27.4) 
[1] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 23.6  (10.6) 
[1] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 85.2  (32.0) 
[1] 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] 
[Units: Units on a scale]
Mean (Standard Deviation)
 
Communication Domain   63.4  (10.0) 
Expressive Communication Domain   69.8  (23.0) 
[1] 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] 
[Units: pg/mL]
Mean (Standard Deviation)
 790.4  (1.3050) 
[1] Brain-derived neurotrophic factor (BDNF) is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses.


  Outcome Measures
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1.  Primary:   Clinical Global Impression- Severity Scale (CGI-S)   [ Time Frame: Week 10 ]

2.  Secondary:   The Aberrant Behavior Checklist (ABC)   [ Time Frame: Week 10 ]

3.  Secondary:   Social Responsiveness Scale   [ Time Frame: Week 10 ]

4.  Secondary:   Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD   [ Time Frame: Week 10 ]

5.  Secondary:   ADHD Rating Scale 4th Edition   [ Time Frame: Week 10 ]

6.  Secondary:   Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain   [ Time Frame: Week 10 ]

7.  Secondary:   Peabody Picture Vocabulary   [ Time Frame: Week 10 ]

8.  Secondary:   Brain-derived Neurotrophic Factor (BDNF)   [ Time Frame: Screen and Week 10 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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.


  More Information
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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
e-mail: craig.erickson@cchmc.org


Publications:

Responsible Party: Indiana University
ClinicalTrials.gov Identifier: NCT01300923     History of Changes
Other Study ID Numbers: 1003-26
Study First Received: August 25, 2010
Results First Received: July 29, 2015
Last Updated: April 27, 2017