Study of Acamprosate in Fragile x Syndrome

Phase 1

Completed

• Conditions: Fragile X Syndrome
• Interventions: Drug: Placebo; Drug: acamprosate

• Registration Number: NCT01911455
• Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary

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

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.

Study Timeline

• Study First Submitted: 2013-07-26
• Study First Submitted QC: 2013-07-29
• Study First Posted: 2013-07-30
• Study Start: 2013-08-27
• Primary Completion: 2018-11-08
• Study Completion: 2018-11-08
• Status Verified: 2020-03
• Last Update Submitted: 2021-02-01
• Last Update Posted: 2021-02-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 46

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo will be prescribed with the same frequency and duration as the acamprosate group.
acamprosate	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.

Primary Outcome Measures

Name	Time	Method
Aberrant Behavior Checklist-Social Withdrawal subscale	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 Outcome Measures

Name	Time	Method
Clinical Global Impressions- Improvement (CGI-I)	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)	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)	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.

Trial Locations

Locations (2)

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Rush University Medical Center; 🇺🇸 Chicago, Illinois, United States