Riluzole in Fragile X Syndrome

This study has been completed.
Sponsor:
Collaborator:
Indiana Clinical and Translational Science Institute (I-CTSI)
Information provided by (Responsible Party):
Indiana University
ClinicalTrials.gov Identifier:
NCT00895752
First received: May 6, 2009
Last updated: April 16, 2013
Last verified: April 2013
  Purpose

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: Endpoint Classification: Efficacy Study
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 ] [ Designated as safety issue: No ]
  • Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Obtained at Baseline and Week 6 ] [ Designated as safety issue: No ]

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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00895752

Locations
United States, Indiana
Christian Sarkine Autism Clinic
Indianapolis, Indiana, United States, 46202
Sponsors and Collaborators
Indiana University
Indiana Clinical and Translational Science Institute (I-CTSI)
Investigators
Principal Investigator: Craig A. Erickson, MD Indiana University School of Medicine
  More Information

No publications provided

Responsible Party: Indiana University
ClinicalTrials.gov Identifier: NCT00895752     History of Changes
Other Study ID Numbers: 0809-11
Study First Received: May 6, 2009
Last Updated: April 16, 2013
Health Authority: United States: Institutional Review Board

Additional relevant MeSH terms:
Fragile X Syndrome
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
Riluzole
Anticonvulsants
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents

ClinicalTrials.gov processed this record on August 26, 2014