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Study of Acamprosate in Fragile x Syndrome

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ClinicalTrials.gov Identifier: NCT01911455
Recruitment Status : Active, not recruiting
First Posted : July 30, 2013
Last Update Posted : February 11, 2019
Sponsor:
Collaborator:
Rush University Medical Center
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Tracking Information
First Submitted Date  ICMJE July 26, 2013
First Posted Date  ICMJE July 30, 2013
Last Update Posted Date February 11, 2019
Study Start Date  ICMJE June 2013
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 18, 2017)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: July 29, 2013)
Aberrant Behavior Checklist-Social Withdrawal subscale [ Time Frame: Change from baseline to week 10 ]
Change History Complete list of historical versions of study NCT01911455 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: July 18, 2017)
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: July 29, 2013)
  • Clinical Global Impressions- Improvement (CGI-I) [ Time Frame: week 10 ]
  • Aberrant Behavior Checklist- Hyperactivity (ABC-H) [ Time Frame: Change from baseline to week 10 ]
  • Aberrant Behavior Checklist-Social Avoidance (ABC-SA) [ Time Frame: Change from baseline to week 10 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Acamprosate in Fragile x Syndrome
Official Title  ICMJE Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE
  • Drug: acamprosate
    Other Name: Campral
  • Drug: Placebo
Study Arms  ICMJE
  • 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.
    Intervention: Drug: acamprosate
  • Placebo Comparator: Placebo
    Placebo will be prescribed with the same frequency and duration as the acamprosate group.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 29, 2013)
48
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2020
Estimated Primary Completion Date June 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 23 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01911455
Other Study ID Numbers  ICMJE CIN001- Acamprosate in FX
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Children's Hospital Medical Center, Cincinnati
Study Sponsor  ICMJE Children's Hospital Medical Center, Cincinnati
Collaborators  ICMJE Rush University Medical Center
Investigators  ICMJE
Principal Investigator: Craig Erickson, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Elizabeth Berry-Kravis, MD Rush University Medical Center
PRS Account Children's Hospital Medical Center, Cincinnati
Verification Date February 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP