Trial Outcomes & Findings for Riluzole in Fragile X Syndrome (NCT NCT00895752)
NCT ID: NCT00895752
Last Updated: 2017-04-18
Results Overview
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
COMPLETED
PHASE4
6 participants
Obtained at Week 6
2017-04-18
Participant Flow
Participant milestones
| Measure |
Riluzole
6 subjects received open label riluzole, maximum dose of 50mg bid.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Riluzole in Fragile X Syndrome
Baseline characteristics by cohort
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
22.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
|
Aberrant Behavior Checklist
ABC Irritability
|
15.2 units on a scale
STANDARD_DEVIATION 11.4 • n=5 Participants
|
|
Aberrant Behavior Checklist
ABC Social Withdrawal
|
15.8 units on a scale
STANDARD_DEVIATION 11.0 • n=5 Participants
|
|
Aberrant Behavior Checklist
ABC Stereotypy
|
8.3 units on a scale
STANDARD_DEVIATION 7.8 • n=5 Participants
|
|
Aberrant Behavior Checklist
ABC Hyperactivity
|
19.5 units on a scale
STANDARD_DEVIATION 8.3 • n=5 Participants
|
|
Aberrant Behavior Checklist
ABC Inappropriate Speech
|
8.5 units on a scale
STANDARD_DEVIATION 2.7 • n=5 Participants
|
|
ADHD rating scale
|
24.3 units on a scale
STANDARD_DEVIATION 8.4 • n=5 Participants
|
|
Clinical Global Improvement-Severity
|
4.3 units on a scale
STANDARD_DEVIATION 0.5 • n=5 Participants
|
|
Peabody Picture Vocabulary Test
|
116.8 units on a scale
STANDARD_DEVIATION 40.2 • n=5 Participants
|
|
Social Reciprocity Scale
|
114.7 units on a scale
STANDARD_DEVIATION 23.6 • n=5 Participants
|
|
Children's Yale-Brown Obsessive Compulsive Scale modified for pervasive developmental disorders
|
11.2 units on a scale
STANDARD_DEVIATION 3.2 • n=5 Participants
|
PRIMARY outcome
Timeframe: Obtained at Week 6The 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
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
Clinical Global Impression-Improvement (CGI-I)
|
3.5 units on a scale
Interval 2.0 to 4.0
|
PRIMARY outcome
Timeframe: Obtained at Baseline and Week 6The 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.
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Baseline
|
11.2 Units on a Scale
Standard Deviation 3.2
|
|
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)
Six Weeks
|
9.7 Units on a Scale
Standard Deviation 3.3
|
SECONDARY outcome
Timeframe: Week 6The 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).
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
Aberrant Behavior Checklist
Irritablity
|
8.3 units on a scale
Standard Deviation 7.7
|
|
Aberrant Behavior Checklist
Social Withdrawal
|
9.7 units on a scale
Standard Deviation 8.0
|
|
Aberrant Behavior Checklist
Stereotypy
|
5.3 units on a scale
Standard Deviation 6.4
|
|
Aberrant Behavior Checklist
Hyperactivity
|
15.0 units on a scale
Standard Deviation 9.2
|
|
Aberrant Behavior Checklist
Inappropriate Speech
|
6.2 units on a scale
Standard Deviation 4.0
|
SECONDARY outcome
Timeframe: Week 6The 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
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
The ADHD Rating Scale
|
19.0 units on a scale
Standard Deviation 9.7
|
SECONDARY outcome
Timeframe: Week 6The 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
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
The Clinical Global Impression - Severity Scale
|
4.2 units on a scale
Standard Deviation 0.4
|
SECONDARY outcome
Timeframe: Week 6The 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.
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
The Peabody Picture Vocabulary Test
|
123.2 units on a scale
Standard Deviation 36.6
|
SECONDARY outcome
Timeframe: Week 6The 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.
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
The Social Reciprocity Scale
|
103.5 units on a scale
Standard Deviation 24.4
|
SECONDARY outcome
Timeframe: Screen and Week 6ERK activations times, as defined as the time in minutes for ERK phosphorylation to reach the half maximal level.
Outcome measures
| Measure |
Riluzole
n=6 Participants
Six subjects received 6-weeks of open-label riluzole with maximum dose of 50mg bid.
|
|---|---|
|
Extra-cellular Signal-relatedness Kinase (ERK)
|
2.99 minutes
Standard Deviation 0.3
|
Adverse Events
Riluzole
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Riluzole
n=6 participants at risk
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.
|
|---|---|
|
General disorders
Tiredness
|
33.3%
2/6 • Number of events 3 • Six Weeks
|
|
General disorders
Irritability
|
50.0%
3/6 • Number of events 3 • Six Weeks
|
|
Psychiatric disorders
Worsening of Inattention
|
16.7%
1/6 • Number of events 1 • Six Weeks
|
|
Metabolism and nutrition disorders
Appetite Reduction
|
16.7%
1/6 • Number of events 1 • Six Weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place