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Riluzole in Fragile X Syndrome

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00895752
First Posted: May 8, 2009
Last Update Posted: April 18, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Indiana Clinical and Translational Sciences Institute
Information provided by (Responsible Party):
Indiana University
Results First Submitted: July 29, 2015  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Fragile X Syndrome
Intervention: Drug: Riluzole

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Riluzole 6 subjects received open label riluzole, maximum dose of 50mg bid.

Participant Flow:   Overall Study
    Riluzole
STARTED   6 
COMPLETED   6 
NOT COMPLETED   0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Riluzole Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.

Baseline Measures
   Riluzole 
Overall Participants Analyzed 
[Units: Participants]
 6 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      6 100.0% 
>=65 years      0   0.0% 
Age 
[Units: Years]
Mean (Full Range)
 22.5 
 (19 to 24) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      0   0.0% 
Male      6 100.0% 
Region of Enrollment 
[Units: Participants]
 
United States   6 
Aberrant Behavior Checklist [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 
ABC Irritability   15.2  (11.4) 
ABC Social Withdrawal   15.8  (11.0) 
ABC Stereotypy   8.3  (7.8) 
ABC Hyperactivity   19.5  (8.3) 
ABC Inappropriate Speech   8.5  (2.7) 
[1] 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).
ADHD rating scale [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 24.3  (8.4) 
[1] 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.
Clinical Global Improvement-Severity [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 4.3  (0.5) 
[1] 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.
Peabody Picture Vocabulary Test [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 116.8  (40.2) 
[1] 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.
Social Reciprocity Scale [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 114.7  (23.6) 
[1] 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
Children's Yale-Brown Obsessive Compulsive Scale modified for pervasive developmental disorders [1] 
[Units: Units on a scale]
Mean (Standard Deviation)
 11.2  (3.2) 
[1] The Children’s Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change.


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Clinical Global Impression-Improvement (CGI-I)   [ Time Frame: Obtained at Week 6 ]

2.  Primary:   Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)   [ Time Frame: Obtained at Baseline and Week 6 ]

3.  Secondary:   Aberrant Behavior Checklist   [ Time Frame: Week 6 ]

4.  Secondary:   The ADHD Rating Scale   [ Time Frame: Week 6 ]

5.  Secondary:   The Clinical Global Impression - Severity Scale   [ Time Frame: Week 6 ]

6.  Secondary:   The Peabody Picture Vocabulary Test   [ Time Frame: Week 6 ]

7.  Secondary:   The Social Reciprocity Scale   [ Time Frame: Week 6 ]

8.  Secondary:   Extra-cellular Signal-relatedness Kinase (ERK)   [ Time Frame: Screen and Week 6 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Craig Erickson MD
Organization: Cincinnati Childrens Hospital
phone: 5136366265
e-mail: craig.erickson@cchmc.org


Publications of Results:

Responsible Party: Indiana University
ClinicalTrials.gov Identifier: NCT00895752     History of Changes
Other Study ID Numbers: 0809-11
First Submitted: May 6, 2009
First Posted: May 8, 2009
Results First Submitted: July 29, 2015
Results First Posted: April 18, 2017
Last Update Posted: April 18, 2017