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Combining Lovastatin and a Parent-Implemented Language Intervention for Fragile X Syndrome

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.
 
ClinicalTrials.gov Identifier: NCT02642653
Recruitment Status : Completed
First Posted : December 30, 2015
Results First Posted : September 11, 2019
Last Update Posted : September 11, 2019
Sponsor:
Information provided by (Responsible Party):
University of California, Davis

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Fragile X Syndrome
Genetic Diseases
Interventions Drug: Lovastatin
Other: Placebo
Enrollment 30
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Period Title: Overall Study
Started 14 16
Completed 12 16
Not Completed 2 0
Reason Not Completed
Adverse Event             2             0
Arm/Group Title Lovastatin and PILI Placebo and PILI Total
Hide Arm/Group Description

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Total of all reporting groups
Overall Number of Baseline Participants 14 16 30
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 16 participants 30 participants
<=18 years
14
 100.0%
16
 100.0%
30
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 14 participants 16 participants 30 participants
13.86  (2.14) 13.17  (2.54) 13.52  (2.34)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 16 participants 30 participants
Female
2
  14.3%
0
   0.0%
2
   6.7%
Male
12
  85.7%
16
 100.0%
28
  93.3%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 16 participants 30 participants
Hispanic or Latino
0
   0.0%
4
  25.0%
4
  13.3%
Not Hispanic or Latino
14
 100.0%
12
  75.0%
26
  86.7%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants 16 participants 30 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
   6.3%
1
   3.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
1
   6.3%
1
   3.3%
White
14
 100.0%
10
  62.5%
24
  80.0%
More than one race
0
   0.0%
4
  25.0%
4
  13.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 14 participants 16 participants 30 participants
14 16 30
Intelligence Quotient (IQ)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 14 participants 16 participants 30 participants
44.00  (9.22) 43.13  (6.24) 43.57  (7.73)
[1]
Measure Description: A standardized test designed to measure developmental functioning and IQ for individuals ranging in age from 2 years to adulthood. Minimal verbal directions are used in the administration to allow assessment of cognitive ability independent of language ability. The Brief IQ screener is composed of 4 subtests: figure ground, form completion, sequential order, and repeated patterns subtests. IQ scores are the sum of the scaled scores for the subtests. Scores range from a minimum of 36 to a maximum of 169.
Cognitive growth score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 14 participants 16 participants 30 participants
461.4  (13.32) 462.3  (8.57) 461.85  (10.95)
[1]
Measure Description: Growth Scores are useful for the interpretation of an individual child's performance. Since the child's overall ability level is placed along a linear scale. Raw scores for each Leiter-R subtest are converted into Growth Scale Scores. The score is a three-digit number that can range from 380 to 560. Higher scores represent higher ability levels.
Autism Diagnostic Observation Scale (ADOS) severity score   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 14 participants 16 participants 30 participants
6.85  (2.54) 6.75  (2.05) 6.80  (2.30)
[1]
Measure Description: The Autism Diagnostic Observation Schedule-Second Edition (ADOS-2) The assessment will provide a continuous metric of autism symptom severity for use in characterizing the participant sample and as a potential moderator of treatment response. The ADOS is a gold-standard measure of current symptoms of autism. Total scores will be used to assign a severity score. Scores range from 1 to 10, with higher scores reflecting more severe impairment
1.Primary Outcome
Title Expressive Language Sample Composite Score in the Home
Hide Description The primary outcome reflects the diversity of vocabulary used. Higher scores reflect more skill. The lowest possible value is zero. No theoretical maximum can be defined because the wordless picture books can lead to a large and indeterminate set of options for the amount of talk and the vocabulary used. In a previous study of a nonpharmacological intervention, the range at baseline was 9-177, and at post-treatment, the range for the combined treated and nontreated groups was 23-214, although these values were not corrected for the number of C-units produced (McDuffie at el. 2018 Developmental Neurorehabilitation).
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: score on a scale
39.63  (32.41) 42.47  (18.60)
2.Primary Outcome
Title Expressive Language Sample Composite Score in the Home
Hide Description The primary outcome reflects the diversity of vocabulary used. Higher scores reflect more skill. The lowest possible value is zero. No theoretical maximum can be defined because the wordless picture books can lead to a large and indeterminate set of options for the amount of talk and the vocabulary used. In a previous study of a nonpharmacological intervention, the range at baseline was 9-177, and at post-treatment, the range for the combined treated and nontreated groups was 23-214, although these values were not corrected for the number of C-units produced (McDuffie at el. 2018 Developmental Neurorehabilitation).
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: score on a scale
59.32  (22.81) 54.81  (16.04)
3.Secondary Outcome
Title FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale
Hide Description The ABC-C is a 58-item caregiver-rated behavior scale used to examine treatment effects on challenging behaviors for individuals with FXS in the following domains: (1) irritability; (2) lethargy/social withdrawal; (3) stereotypic behavior; (4) hyperactivity; and (5) inappropriate speech. Caregiver rates the subject's behavior as follows: 0 = not a problem, l = the behavior is a problem but slight in degree, 2 = the problem is moderately serious, 3 = the problem is severe in degree. Sansone et al. (2012) further subdivided social withdrawal to rate social avoidance in FXS. The subscale includes 4 items which were originally part of the Lethargy/Withdrawal subscale. This subscale captures core aspects of the FXS phenotype related to gaze avoidance, social ''escape'' behaviors, and social anxiety. Subscale score ranges from 0 to 12, higher scores reflect a more problematic behavior.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.5  (2.84) 4.31  (3.66)
4.Secondary Outcome
Title FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale
Hide Description The ABC-C is a 58-item caregiver-rated behavior scale used to examine treatment effects on challenging behaviors for individuals with FXS in the following domains: (1) irritability; (2) lethargy/social withdrawal; (3) stereotypic behavior; (4) hyperactivity; and (5) inappropriate speech. Caregiver rates the subject's behavior as follows: 0 = not a problem, l = the behavior is a problem but slight in degree, 2 = the problem is moderately serious, 3 = the problem is severe in degree. Sansone et al. (2012) further subdivided social withdrawal to rate social avoidance in FXS. The subscale includes 4 items which were originally part of the Lethargy/Withdrawal subscale. This subscale captures core aspects of the FXS phenotype related to gaze avoidance, social ''escape'' behaviors, and social anxiety. Subscale score ranges from 0 to 12, higher scores reflect a more problematic behavior. Shown here are the ABC social avoidance subscale mean scores from the 10-weeks follow-up visit.
Time Frame 10 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.5  (2.11) 3.53  (3.68)
5.Secondary Outcome
Title FXS- Normed Aberrant Behavior Checklist (ABC) Social Avoidance Subscale
Hide Description The ABC-C is a 58-item caregiver-rated behavior scale used to examine treatment effects on challenging behaviors for individuals with FXS in the following domains: (1) irritability; (2) lethargy/social withdrawal; (3) stereotypic behavior; (4) hyperactivity; and (5) inappropriate speech. Caregiver rates the subject's behavior as follows: 0 = not a problem, l = the behavior is a problem but slight in degree, 2 = the problem is moderately serious, 3 = the problem is severe in degree. Sansone et al. (2012) further subdivided social withdrawal to rate social avoidance in FXS. The subscale includes 4 items which were originally part of the Lethargy/Withdrawal subscale. This subscale captures core aspects of the FXS phenotype related to gaze avoidance, social ''escape'' behaviors, and social anxiety. Subscale score ranges from 0 to 12, higher scores reflect a more problematic behavior. Shown are the ABC social avoidance subscale mean scores from the 20-weeks end-of-study visit.
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: score on a scale
2.42  (2.84) 3.38  (3.34)
6.Secondary Outcome
Title Clinical Global Impression- Severity (CGI-S)
Hide Description A clinician rated scale utilizing history from the parents or caregiver and incorporating it into a clinical rating for severity. The CGI-S was used at the pre-treatment assessment to judge symptom severity. The 7-point scale ranges from: 1 = Normal; 2 = Borderline Ill; 3 = Mildly Ill; 4 = Moderately Ill; 5 = Markedly Ill; 6 = Severely Ill; and 7 = Extremely Ill. Therefore, the higher the score, the greater the severity of the patient's illness. Shown here are the CGI-S mean scores from the baseline visit.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: units on a scale
4.58  (0.67) 4.50  (0.63)
7.Secondary Outcome
Title Clinical Global Impression-Improvement (CGI-I) Scale
Hide Description A clinician rated scale utilizing history from the parents or caregiver and incorporating it into a clinical rating to assess for overall therapeutic response. The 7-point scale ranges from: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; and 7 = Very much worse. Therefore, the lower the score, the greater the overall improvement as rated by the clinician. The CGI-I was used at the 10 week and 20 week visits. Shown here are the CGI-I mean scores from the 10-weeks end-of-study visit.
Time Frame 10 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: units on a scale
2.92  (0.79) 3.00  (0.73)
8.Secondary Outcome
Title Clinical Global Impression-Improvement (CGI-I) Scale
Hide Description A clinician rated scale utilizing history from the parents or caregiver and incorporating it into a clinical rating to assess for overall therapeutic response. The 7-point scale ranges from: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = No change; 5 = Minimally worse; 6 = Much worse; and 7 = Very much worse. Therefore, the lower the score, the greater the overall improvement as rated by the clinician. The CGI-I was used at the 10 week and 20 week visits. Shown here are the CGI-I mean scores from the 20-weeks end-of-study visit.
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: units on a scale
2.42  (0.67) 2.38  (0.72)
9.Secondary Outcome
Title Visual Analog Scale (VAS) - Spoken Language Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Spoken Language Impairment scale, at baseline. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
4.06  (2.43) 1.94  (1.41)
10.Secondary Outcome
Title Visual Analog Scale (VAS) - Spoken Language Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Spoken Language Impairment scale, at 10-weeks. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame 10 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
5.28  (2.69) 3.09  (1.14)
11.Secondary Outcome
Title Visual Analog Scale (VAS) - Spoken Language Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Spoken Language Impairment scale, at 20-weeks. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame 20 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
5.66  (1.99) 4.18  (1.53)
12.Secondary Outcome
Title Visual Analog Scale (VAS) - Social Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Social Impairment scale, at baseline. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
3.73  (1.74) 2.55  (1.36)
13.Secondary Outcome
Title Visual Analog Scale (VAS) - Social Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Social Impairment scale, at 10-weeks. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame 10-weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
4.57  (2.15) 3.41  (1.61)
14.Secondary Outcome
Title Visual Analog Scale (VAS) - Social Impairment
Hide Description The measure was used to assess parental impressions of progress in two key symptoms: spoken language impairment and social impairment. The distance of the mark from one end is used as the outcome variable for analysis. Caregivers mark on a visual line measuring 10 cm with "worst behavior" at 0 cm and "best behavior" at 10 cm. For each behavior the caregiver is instructed to mark their impression of the behavior at baseline visit and again at the 10-weeks and 20-weeks visits. The calculated distance in cm between the marks drawn at the baseline and follow-up visits thereby demonstrates whether each behavior improved, worsened, or stayed the same during the study, and by how much. Shown here is the mean distance in cm from the "worst behavior" side for the Social Impairment scale, at 20-weeks. The smaller the value, the worse the behavior. The range is minimum 0 cm to maximum 10 cm.
Time Frame 20-weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description:

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

Overall Number of Participants Analyzed 12 16
Mean (Standard Deviation)
Unit of Measure: centimeters
5.18  (1.70) 3.99  (2.06)
Time Frame From baseline to follow-up (20-weeks)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Lovastatin and PILI Placebo and PILI
Hide Arm/Group Description

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with study medication Lovastatin.

Lovastatin: Once per day dosing

Subjects will receive the Parent Implemented Language Intervention (PILI) in combination with placebo.

Placebo: Once per day dosing

All-Cause Mortality
Lovastatin and PILI Placebo and PILI
Affected / at Risk (%) Affected / at Risk (%)
Total   0/12 (0.00%)      0/16 (0.00%)    
Hide Serious Adverse Events
Lovastatin and PILI Placebo and PILI
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/12 (0.00%)      0/16 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Lovastatin and PILI Placebo and PILI
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   11/12 (91.67%)      13/16 (81.25%)    
Blood and lymphatic system disorders     
Epistaxis   1/12 (8.33%)  1 1/16 (6.25%)  1
Eye disorders     
Eye Infection   0/12 (0.00%)  0 1/16 (6.25%)  2
Gastrointestinal disorders     
Decreased Appetite   0/12 (0.00%)  0 3/16 (18.75%)  3
Diarrhea   1/12 (8.33%)  1 2/16 (12.50%)  3
Gastrointestinal issues   2/12 (16.67%)  2 4/16 (25.00%)  5
Vomiting   1/12 (8.33%)  1 0/16 (0.00%)  0
General disorders     
Fatigue   0/12 (0.00%)  0 1/16 (6.25%)  1
Fever   1/12 (8.33%)  2 0/16 (0.00%)  0
Hepatobiliary disorders     
Elevated Bilirubin   0/12 (0.00%)  0 1/16 (6.25%)  1
Infections and infestations     
Ear infection   0/12 (0.00%)  0 1/16 (6.25%)  1
Sinus Infection   0/12 (0.00%)  0 1/16 (6.25%)  1
Skin_Infection   1/12 (8.33%)  2 2/16 (12.50%)  2
Upper Respiratory Infection   7/12 (58.33%)  10 8/16 (50.00%)  12
Injury, poisoning and procedural complications     
Musculoskeletal injury   0/12 (0.00%)  0 1/16 (6.25%)  1
Sprain   0/12 (0.00%)  0 1/16 (6.25%)  1
Skin abrasion   1/12 (8.33%)  1 0/16 (0.00%)  0
Metabolism and nutrition disorders     
Weight Loss   0/12 (0.00%)  0 1/16 (6.25%)  1
Nervous system disorders     
Dizziness   0/12 (0.00%)  0 1/16 (6.25%)  1
Dry Mouth   0/12 (0.00%)  0 1/16 (6.25%)  1
Headache   1/12 (8.33%)  1 2/16 (12.50%)  2
Sedation   0/12 (0.00%)  0 1/16 (6.25%)  1
Psychiatric disorders     
Abnormal vocalizations   1/12 (8.33%)  1 1/16 (6.25%)  1
Agitation   0/12 (0.00%)  0 1/16 (6.25%)  1
Anxiety   1/12 (8.33%)  1 1/16 (6.25%)  1
Disruptive behavior   1/12 (8.33%)  1 0/16 (0.00%)  0
Emotional Lability   0/12 (0.00%)  0 2/16 (12.50%)  2
Hyperactivity   2/12 (16.67%)  2 0/16 (0.00%)  0
Insomnia   0/12 (0.00%)  0 1/16 (6.25%)  1
Irritability   0/12 (0.00%)  0 1/16 (6.25%)  1
Obsessive compulsive behavior   0/12 (0.00%)  0 1/16 (6.25%)  1
Self-injurious behavior   0/12 (0.00%)  0 1/16 (6.25%)  1
Sleep Disturbance   1/12 (8.33%)  1 1/16 (6.25%)  1
Renal and urinary disorders     
Incontinence   1/12 (8.33%)  1 0/16 (0.00%)  0
Reproductive system and breast disorders     
Menstrual Cramps   1/12 (8.33%)  1 0/16 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Rhinorrhea   0/12 (0.00%)  0 1/16 (6.25%)  1
Skin and subcutaneous tissue disorders     
Itchiness   0/12 (0.00%)  0 2/16 (12.50%)  3
Rash   3/12 (25.00%)  3 1/16 (6.25%)  1
Indicates events were collected by systematic assessment
The study did not have a cohort treated only with lovastatin without the parent-implemented language intervention (PILI). The efficacy of the study medication alone compared to placebo can't be evaluated.
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
Layout table for Results Point of Contact information
Name/Title: Dr. Randi Hagerman
Organization: University of California Davis
Phone: 9167030247
EMail: rjhagerman@ucdavis.edu
Layout table for additonal information
Responsible Party: University of California, Davis
ClinicalTrials.gov Identifier: NCT02642653    
Other Study ID Numbers: 520144
First Submitted: December 21, 2015
First Posted: December 30, 2015
Results First Submitted: July 23, 2019
Results First Posted: September 11, 2019
Last Update Posted: September 11, 2019