Skip to main content
OpenTrials
Completed

NCT Number: NCT01911455

Study of Acamprosate in Fragile x Syndrome

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Fragile X Syndrome Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Sex Chromosome Disorders X-Linked Intellectual Disability

Age range

5 year–23 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Rush University Medical Center, Chicago, Illinois, United States

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

acamprosate

Drug

Other names: Campral

Placebo

Drug

Primary outcomes

  1. 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 outcomes

  1. 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.

  2. 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.

  3. 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.

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • Rush University Medical Center

Registry information

Official study title

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

Important dates

Study start
2013
Primary completion
2018
Study completion
2018
First posted
Jul 30, 2013
Registry last updated
Feb 3, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.