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Riluzole in Fragile X Syndrome

This study has been completed.
Indiana Clinical and Translational Sciences Institute
Information provided by (Responsible Party):
Indiana University Identifier:
First received: May 6, 2009
Last updated: April 16, 2013
Last verified: April 2013
The purpose of this study is to investigate the effectiveness and tolerability of riluzole in adults with Fragile X Syndrome.

Condition Intervention Phase
Fragile X Syndrome
Drug: Riluzole
Phase 4

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay

Resource links provided by NLM:

Further study details as provided by Indiana University:

Primary Outcome Measures:
  • Clinical Global Impression-Improvement (CGI-I) [ Time Frame: Obtained at Baseline and Week 6 ]
  • Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Obtained at Baseline and Week 6 ]

Enrollment: 6
Study Start Date: April 2009
Study Completion Date: July 2010
Primary Completion Date: July 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Riluzole
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Drug: Riluzole
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00895752

United States, Indiana
Christian Sarkine Autism Clinic
Indianapolis, Indiana, United States, 46202
Sponsors and Collaborators
Indiana University
Indiana Clinical and Translational Sciences Institute
Principal Investigator: Craig A. Erickson, MD Indiana University School of Medicine
  More Information

Responsible Party: Indiana University Identifier: NCT00895752     History of Changes
Other Study ID Numbers: 0809-11
Study First Received: May 6, 2009
Last Updated: April 16, 2013

Additional relevant MeSH terms:
Fragile X Syndrome
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents processed this record on March 24, 2017