Fragile X Syndrome
Conditions
Keywords
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.
Interventions
DRUGacamprosate
DRUGPlacebo
Sponsors
Rush University Medical Center
Children's Hospital Medical Center, Cincinnati
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
BASIC_SCIENCE
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to 23 Years
Healthy volunteers
Yes
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Aberrant Behavior Checklist-Social Withdrawal subscale | 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
| Measure | Time frame | Description |
|---|---|---|
| Clinical Global Impressions- Improvement (CGI-I) | 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) | 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) | 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. |
Countries
United States
Outcome results
None listed