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Study of Acamprosate in Fragile x Syndrome

This study is currently recruiting participants.
Verified July 2017 by Children's Hospital Medical Center, Cincinnati
Sponsor:
ClinicalTrials.gov Identifier:
NCT01911455
First Posted: July 30, 2013
Last Update Posted: July 21, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Rush University Medical Center
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
  Purpose
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).

Condition Intervention Phase
Fragile X Syndrome Drug: acamprosate Drug: Placebo Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome 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 Outcome 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.


Estimated Enrollment: 48
Study Start Date: June 2013
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: February 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Campral
Placebo Comparator: Placebo
Placebo will be prescribed with the same frequency and duration as the acamprosate group.
Drug: Placebo

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 23 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01911455


Contacts
Contact: Katie Friedmann, RN 513-636-0523 fragilex@cchmc.org

Locations
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Contact: Jamie Chin    312-942-0025    jamie_a_chin@rush.edu   
Principal Investigator: Elizabeth Berry-Kravis, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Katie Friedmann, RN    513-636-0523    fragilex@cchmc.org   
Principal Investigator: Craig Erickson, MD         
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Rush University Medical Center
Investigators
Principal Investigator: Craig Erickson, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Elizabeth Berry-Kravis, MD Rush University Medical Center
  More Information

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01911455     History of Changes
Other Study ID Numbers: CIN001- Acamprosate in FX
First Submitted: July 26, 2013
First Posted: July 30, 2013
Last Update Posted: July 21, 2017
Last Verified: July 2017

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fragile X Syndrome

Additional relevant MeSH terms:
Fragile X Syndrome
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Acamprosate
Alcohol Deterrents