Study of Acamprosate in Fragile x Syndrome
Primary Purpose
Fragile X Syndrome
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
acamprosate
Placebo
Sponsored by
About this trial
This is an interventional basic science trial for Fragile X Syndrome focused on measuring Fragile X Syndrome
Eligibility 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
Sites / Locations
- Rush University Medical Center
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Placebo Comparator
Arm Label
acamprosate
Placebo
Arm Description
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.
Placebo will be prescribed with the same frequency and duration as the acamprosate group.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
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.
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.
Full Information
NCT ID
NCT01911455
First Posted
July 26, 2013
Last Updated
February 1, 2021
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Rush University Medical Center
1. Study Identification
Unique Protocol Identification Number
NCT01911455
Brief Title
Study of Acamprosate in Fragile x Syndrome
Official Title
Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Completed
Study Start Date
August 27, 2013 (Actual)
Primary Completion Date
November 8, 2018 (Actual)
Study Completion Date
November 8, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Rush University Medical Center
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X Syndrome
7. Study Design
Primary Purpose
Basic Science
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
46 (Actual)
8. Arms, Groups, and Interventions
Arm Title
acamprosate
Arm Type
Active Comparator
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo will be prescribed with the same frequency and duration as the acamprosate group.
Intervention Type
Drug
Intervention Name(s)
acamprosate
Other Intervention Name(s)
Campral
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Aberrant Behavior Checklist-Social Withdrawal subscale
Description
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Time Frame
Change from baseline to week 10
Secondary Outcome Measure Information:
Title
Clinical Global Impressions- Improvement (CGI-I)
Description
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.
Time Frame
week 10
Title
Aberrant Behavior Checklist- Hyperactivity (ABC-H)
Description
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Time Frame
Change from baseline to week 10
Title
Aberrant Behavior Checklist-Social Avoidance (ABC-SA)
Description
The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
Time Frame
Change from baseline to week 10
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
23 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Craig Erickson, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Elizabeth Berry-Kravis, MD
Organizational Affiliation
Rush University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
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Study of Acamprosate in Fragile x Syndrome
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