Riluzole in Fragile X Syndrome

NCT ID: NCT00895752

Last Updated: 2017-04-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-04-30
• Study Completion Date: 2010-07-31

Brief Summary

The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.

Detailed Description

Fragile X Syndrome (FXS) represents the most common inherited form of intellectual disability. FXS is more common in males and the symptoms associated with the disorder are more marked in males. FXS is associated with characteristic physical features, behaviors, and comorbidities. Those with FXS often suffer from behavioral difficulties that include anxiety-related symptoms (shyness, social phobia, obsessive-compulsive disorder (OCD) symptoms), attention deficit hyperactivity symptoms (overarousal, hyperactivity, distractibility, impulsivity) and aggressive/self-injurious behaviors.

Riluzole is approved by the FDA for use in treating amyotrophic lateral sclerosis (ALS) in adults. Recently, riluzole has been the subject of several open-label studies describing the use of the drug in treatment-resistant depression and OCD.

Given the overlap between repetitive behavior in FXS and symptoms of OCD, it is logical to study riluzole in FXS given the compound's promise in ameliorating treatment-refractory symptoms of OCD.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Riluzole

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Group Type: EXPERIMENTAL

Riluzole

Intervention Type: DRUG

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Interventions

Riluzole

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Males and females age 18 years or older.
• Confirmed molecular diagnosis of Fragile X Syndrome.
• Clinical Global Impression Severity (CGI-S) score of 3 or greater.
• Significant interfering repetitive behavior as determined by the principal investigator.
• Must be in good health as determined by screening procedures including a detailed medical history, and complete physical and neurological examination.
• Dosing of concomitant medications during the study must remain stable.

Exclusion Criteria

• Pregnancy.
• Concomitant use of another glutamatergic agent (memantine, topiramate, amantadine, among others.
• Evidence of prior trial of riluzole and/or hypersensitivity/allergic reaction to riluzole.
• Abnormal baseline liver function tests at screen or by history; or complete blood count abnormalities at screen or by history.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Indiana Clinical and Translational Sciences Institute

OTHER

Sponsor Role: collaborator

Indiana University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Craig A. Erickson, MD

Role: PRINCIPAL_INVESTIGATOR

Indiana University School of Medicine

Locations

Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children

Indianapolis, Indiana, United States

Countries

United States

References

Erickson CA, Weng N, Weiler IJ, Greenough WT, Stigler KA, Wink LK, McDougle CJ. Open-label riluzole in fragile X syndrome. Brain Res. 2011 Mar 22;1380:264-70. doi: 10.1016/j.brainres.2010.10.108. Epub 2010 Nov 5.

Reference Type: RESULT

PMID: 21059347 (View on PubMed)

Other Identifiers

0809-11

Identifier Type: -

Identifier Source: org_study_id