Study of Acamprosate in Fragile x Syndrome

Study Overview
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Brief Summary
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In this research study we want to understand the effectiveness of a drug treatment, acamprosate, for interfering symptoms (i.e., inattention/hyperactivity, social impairment) associated with Fragile X Syndrome (FXS).

Detailed Description
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Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.

Official Title
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Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome

Conditions
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Fragile X Syndrome

Intervention / Treatment
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* Drug: acamprosate
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnostic confirmation of full mutation FXS Age ≥5 years and <23 years General good health as determined by physical exam, medical history and laboratory work up. Exclusion Criteria: Use of more than two psychotropic medications (medications affecting behavior). Unstable dosing of any psychotropic medication (medication affecting behavior) Problems with kidney functioning Unstable seizure disorder Change in any anti-convulsant drug dosing in the 60 days prior to study entry Prior adequate treatment trial with acamprosate as determined by the study doctor Pregnant or lactating females

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 23 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: acamprosate<br>The maximum dose of acamprosate to be used in this study is 666 mg three times daily (total 1998 mg/day) for subjects weight ≥ 50 kg and 1332mg for subjects that weigh < 50 kg. | Drug: acamprosate<br> <br> * Other names: Campral;|
| Placebo Comparator: Placebo<br>Placebo will be prescribed with the same frequency and duration as the acamprosate group. | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant Behavior Checklist-Social Withdrawal subscale | The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. | Change from baseline to week 10 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinical Global Impressions- Improvement (CGI-I) | The CGI-I global improvement item is a 7-point Likert scale designed to measure symptomatic change at a specific time as compared to baseline. CGI-I is a gold standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities. | week 10 | 
| Aberrant Behavior Checklist- Hyperactivity (ABC-H) | The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. | Change from baseline to week 10 | 
| Aberrant Behavior Checklist-Social Avoidance (ABC-SA) | The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials. | Change from baseline to week 10 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fragile X Syndrome