Study of Acamprosate in Fragile x Syndrome

Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With 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).

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: acamprosate

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 Type: Interventional
• Enrollment (Actual): 46
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 23 years (ADULT, CHILD)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: BASIC_SCIENCE
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.	Drug: acamprosate; Other Names: Campral
PLACEBO_COMPARATOR: Placebo; Placebo will be prescribed with the same frequency and duration as the acamprosate group.	Drug: Placebo

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Children's Hospital Medical Center, Cincinnati
• Collaborators: Rush University Medical Center

Study record dates

Study Major Dates

• Study Start (ACTUAL): August 27, 2013
• Primary Completion (ACTUAL): November 8, 2018
• Study Completion (ACTUAL): November 8, 2018

Study Registration Dates

• First Submitted: July 26, 2013
• First Submitted That Met QC Criteria: July 29, 2013
• First Posted (ESTIMATE): July 30, 2013

Study Record Updates

• Last Update Posted (ACTUAL): February 3, 2021
• Last Update Submitted That Met QC Criteria: February 1, 2021
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Keywords: Fragile X Syndrome
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome; Alcohol Deterrents; Acamprosate
• Other Study ID Numbers: CIN001- Acamprosate in FX