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Riluzole in Fragile X Syndrome

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ClinicalTrials.gov Identifier: NCT00895752
Recruitment Status : Completed
First Posted : May 8, 2009
Results First Posted : April 18, 2017
Last Update Posted : April 18, 2017
Sponsor:
Collaborator:
Indiana Clinical and Translational Sciences Institute
Information provided by (Responsible Party):
Indiana University

Tracking Information
First Submitted Date  ICMJE May 6, 2009
First Posted Date  ICMJE May 8, 2009
Results First Submitted Date  ICMJE July 29, 2015
Results First Posted Date  ICMJE April 18, 2017
Last Update Posted Date April 18, 2017
Study Start Date  ICMJE April 2009
Actual Primary Completion Date July 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 7, 2017)
  • Clinical Global Impression-Improvement (CGI-I) [ Time Frame: Obtained at Week 6 ]
    The Clinical Global Impression - Improvement scale (CGI-I) is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. and rated as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse
  • Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS) [ Time Frame: Obtained at Baseline and Week 6 ]
    The CY-BOCS PDD has been utilized in a largescale clinical treatment study of repetitive behavior in idiopathic ASDs. CYBOCS-PDD scores range from 0 to 20 and measure repetitive/compulsive behavior and not obsessions. Higher score indicate worse outcome.
Original Primary Outcome Measures  ICMJE
 (submitted: May 6, 2009)
  • 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 ]
Change History Complete list of historical versions of study NCT00895752 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2017)
  • Aberrant Behavior Checklist [ Time Frame: Week 6 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item measure of maladaptive behaviors and is used as a measure of drug effects. The ABC has 5 subscales: Social Withdrawal (16 items) ranging from 0 (not at all) to 48 (severe), Irritability (15 items) ranging from 0 (not at all) to 45 (severe), Inappropriate Speech (4 items) ranging from 0 (not at all) to 12 (severe), Hyperactivity (16 items) ranging from 0 (not at all) to 48 (severe), and Stereotypy (7 items) ranging from 0 (not at all) to 21 (severe). Items are rated from 0 (not at all) to 3 (severe).
  • The ADHD Rating Scale [ Time Frame: Week 6 ]
    The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity
  • The Clinical Global Impression - Severity Scale [ Time Frame: Week 6 ]
    The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill
  • The Peabody Picture Vocabulary Test [ Time Frame: Week 6 ]
    The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank.
  • The Social Reciprocity Scale [ Time Frame: Week 6 ]
    The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment.
  • Extra-cellular Signal-relatedness Kinase (ERK) [ Time Frame: Screen and Week 6 ]
    ERK activations times, as defined as the time in minutes for ERK phosphorylation to reach the half maximal level.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Riluzole in Fragile X Syndrome
Official Title  ICMJE Riluzole in Fragile X Syndrome: A Pilot Study Incorporating Biomarker Assay
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Fragile X Syndrome
Intervention  ICMJE Drug: Riluzole
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Study Arms  ICMJE Experimental: Riluzole
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Intervention: Drug: Riluzole
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 6, 2009)
6
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 2010
Actual Primary Completion Date July 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00895752
Other Study ID Numbers  ICMJE 0809-11
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Indiana University
Study Sponsor  ICMJE Indiana University
Collaborators  ICMJE Indiana Clinical and Translational Sciences Institute
Investigators  ICMJE
Principal Investigator: Craig A. Erickson, MD Indiana University School of Medicine
PRS Account Indiana University
Verification Date March 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP