Study of Acamprosate in Fragile x Syndrome

Overview

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).

Full Title of Study: “Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: June 2020

Detailed Description

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.

Interventions

  • Drug: acamprosate
  • Drug: Placebo

Arms, Groups and Cohorts

  • Active Comparator: acamprosate
    • 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.
  • Placebo Comparator: Placebo
    • Placebo will be prescribed with the same frequency and duration as the acamprosate group.

Clinical Trial Outcome Measures

Primary Measures

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

Secondary Measures

  • Clinical Global Impressions- Improvement (CGI-I)
    • Time Frame: week 10
    • 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.
  • Aberrant Behavior Checklist- Hyperactivity (ABC-H)
    • Time Frame: Change from baseline to week 10
    • The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
  • Aberrant Behavior Checklist-Social Avoidance (ABC-SA)
    • Time Frame: Change from baseline to week 10
    • The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 5 Years

Maximum Age: 23 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

  • Lead Sponsor
    • Children’s Hospital Medical Center, Cincinnati
  • Collaborator
    • Rush University Medical Center
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Craig Erickson, MD, Principal Investigator, Children’s Hospital Medical Center, Cincinnati
    • Elizabeth Berry-Kravis, MD, Principal Investigator, Rush University Medical Center

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