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 : April 28, 2017
|Condition or disease||Intervention/treatment||Phase|
|Fragile X Syndrome Autism Spectrum Disorders||Drug: Acamprosate||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||14 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Study of Acamprosate in Youth With Fragile X Syndrome|
|Study Start Date :||August 2010|
|Actual Primary Completion Date :||September 2011|
|Actual Study Completion Date :||September 2011|
U.S. FDA Resources
Active Comparator: 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.
Other Name: Campral
No Intervention: Autism Spectrum Disorder
This baseline comparison group will participated in only the psychophysiological and biomarker portion of subject characterization.
- Clinical Global Impression- Severity Scale (CGI-S) [ Time Frame: Week 10 ]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.
- The Aberrant Behavior Checklist (ABC) [ Time Frame: Week 10 ]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.
- Social Responsiveness Scale [ Time Frame: Week 10 ]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
- Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD [ Time Frame: Week 10 ]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.
- ADHD Rating Scale 4th Edition [ Time Frame: Week 10 ]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.
- Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain [ Time Frame: Week 10 ]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.
- Peabody Picture Vocabulary [ Time Frame: Week 10 ]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.
- Brain-derived Neurotrophic Factor (BDNF) [ Time Frame: Screen and Week 10 ]BDNF is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01300923
|United States, Indiana|
|Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children|
|Indianapolis, Indiana, United States, 46202|
|Principal Investigator:||Craig A. Erickson, M.D.||Indiana University School of Medicine - Department of Psychiatry|