Study of Acamprosate in Fragile x Syndrome

Fragile X Syndrome Clinical Trial

Official title:

Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01911455
• Other study ID #: CIN001- Acamprosate in FX
• Status: Completed
• Phase: Phase 1
• Start date: August 27, 2013
• Est. completion date: November 8, 2018

Study information

• Verified date: March 2020
• Source: Children's Hospital Medical Center, Cincinnati
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 46
• Est. completion date: November 8, 2018
• Est. primary completion date: November 8, 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 5 Years to 23 Years

Eligibility

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

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• acamprosate

• Placebo

Locations

Country	Name	City	State
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio

Sponsors (2)

Lead Sponsor	Collaborator
Children's Hospital Medical Center, Cincinnati	Rush University Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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