Clinical trial NCT00895752: Riluzole in Fragile X Syndrome
Official title: Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Fragile X Syndrome
Lead sponsor: Indiana University
Sponsor class: OTHER
Collaborators: name: Indiana Clinical and Translational Sciences Institute
First posted: 2009-05-08
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Clinical Global Impression-Improvement (CGI-I)
timeFrame: Obtained at Week 6
measure: Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
timeFrame: Obtained at Baseline and Week 6
Secondary outcomes:
measure: Aberrant Behavior Checklist
timeFrame: Week 6
measure: The ADHD Rating Scale
timeFrame: Week 6
measure: The Clinical Global Impression - Severity Scale
timeFrame: Week 6
measure: The Peabody Picture Vocabulary Test
timeFrame: Week 6
measure: The Social Reciprocity Scale
timeFrame: Week 6
measure: Extra-cellular Signal-relatedness Kinase (ERK)
timeFrame: Screen and Week 6
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children
state: Indiana
Study officials:
affiliation: Indiana University School of Medicine
name: Craig A. Erickson, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.