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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

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Intervention Drug: Riluzole
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Riluzole
Hide Arm/Group Description 6 subjects received open label riluzole, maximum dose of 50mg bid.
Period Title: Overall Study
Started 6
Completed 6
Not Completed 0
Arm/Group Title Riluzole
Hide Arm/Group Description Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
0
   0.0%
Between 18 and 65 years
6
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 6 participants
22.5
(19 to 24)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
0
   0.0%
Male
6
 100.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 6 participants
6
Aberrant Behavior Checklist   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
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]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
24.3  (8.4)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
4.3  (0.5)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
116.8  (40.2)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
114.7  (23.6)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 6 participants
11.2  (3.2)
[1]
Measure Description: 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.
1.Primary Outcome
Title Clinical Global Impression-Improvement (CGI-I)
Hide Description 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
Time Frame Obtained at Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
Overall Number of Participants Analyzed 6
Mean (Full Range)
Unit of Measure: units on a scale
3.5
(2 to 4)
2.Primary Outcome
Title Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Hide Description 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.
Time Frame Obtained at Baseline and Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
6 subjects received open label riluzole, maximum dose of 50mg bid.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: Units on a Scale
Baseline 11.2  (3.2)
Six Weeks 9.7  (3.3)
3.Secondary Outcome
Title Aberrant Behavior Checklist
Hide Description 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).
Time Frame Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
6 subjects received open label riluzole, maximum dose of 50mg bid.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
Irritablity 8.3  (7.7)
Social Withdrawal 9.7  (8.0)
Stereotypy 5.3  (6.4)
Hyperactivity 15.0  (9.2)
Inappropriate Speech 6.2  (4.0)
4.Secondary Outcome
Title The ADHD Rating Scale
Hide Description 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
Time Frame Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
19.0  (9.7)
5.Secondary Outcome
Title The Clinical Global Impression - Severity Scale
Hide Description 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
Time Frame Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
4.2  (0.4)
6.Secondary Outcome
Title The Peabody Picture Vocabulary Test
Hide Description 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.
Time Frame Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
123.2  (36.6)
7.Secondary Outcome
Title The Social Reciprocity Scale
Hide Description 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.
Time Frame Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: units on a scale
103.5  (24.4)
8.Secondary Outcome
Title Extra-cellular Signal-relatedness Kinase (ERK)
Hide Description ERK activations times, as defined as the time in minutes for ERK phosphorylation to reach the half maximal level.
Time Frame Screen and Week 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Riluzole
Hide Arm/Group Description:
Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: minutes
2.99  (0.3)
Time Frame Six Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Riluzole
Hide Arm/Group Description

Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

Riluzole: Six week open-label treatment with riluzole, maximum dose of 50 mg twice a day.

All-Cause Mortality
Riluzole
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Riluzole
Affected / at Risk (%) # Events
Total   0/6 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Riluzole
Affected / at Risk (%) # Events
Total   3/6 (50.00%)    
General disorders   
Tiredness  2/6 (33.33%)  3
Irritability  3/6 (50.00%)  3
Metabolism and nutrition disorders   
Appetite Reduction  1/6 (16.67%)  1
Psychiatric disorders   
Worsening of Inattention  1/6 (16.67%)  1
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
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