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Safety and Efficacy of AFQ056 in Adolescent Patients With 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: NCT01357239
Recruitment Status : Completed
First Posted : May 20, 2011
Results First Posted : May 12, 2015
Last Update Posted : May 12, 2015
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Fragile X Syndrome
Interventions Drug: AFQ056
Drug: Placebo
Enrollment 139
Recruitment Details  
Pre-assignment Details  
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description 1 capsule of 25 mg and 1 capsule of placebo per intake 2 capsules of 25 mg per intake 1 capsule of 100 mg and 1 capsule of placebo per intake 2 capsules of placebo per intake
Period Title: Stratum: All Patients
Started 31 27 39 42
Completed 31 27 37 40
Not Completed 0 0 2 2
Reason Not Completed
Adverse Event             0             0             1             1
Subject withdrew consent             0             0             1             1
Period Title: Stratum I: Fully-Methylated
Started 8 6 17 22
Completed 8 6 16 22
Not Completed 0 0 1 0
Reason Not Completed
Adverse Event             0             0             1             0
Period Title: Stratum II: Partially-Methylated
Started 23 21 22 20
Completed 23 21 21 18
Not Completed 0 0 1 2
Reason Not Completed
Adverse Event             0             0             0             1
Subject withdrew consent             0             0             1             1
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo Total
Hide Arm/Group Description 1 capsule of 25 mg and 1 capsule of placebo per intake 2 capsules of 25 mg per intake 1 capsule of 100 mg and 1 capsule of placebo per intake 2 capsules of placebo per intake Total of all reporting groups
Overall Number of Baseline Participants 31 27 39 42 139
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 31 participants 27 participants 39 participants 42 participants 139 participants
14.4  (1.70) 14.6  (1.58) 14.6  (1.77) 14.4  (1.85) 14.5  (1.72)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 31 participants 27 participants 39 participants 42 participants 139 participants
Female
5
  16.1%
3
  11.1%
4
  10.3%
3
   7.1%
15
  10.8%
Male
26
  83.9%
24
  88.9%
35
  89.7%
39
  92.9%
124
  89.2%
1.Primary Outcome
Title Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist–Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid
Hide Description The Aberrant Behavior Checklist-Community edition (ABC-C) is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 (“not at all a problem”) to 3 (“problem is severe in degree”) and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum I included patients whose Fragile X Mental Retardation 1 (FMR1) gene was fully methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 16 22
Least Squares Mean (Standard Error)
Unit of Measure: Score
8.6  (4.48) -9.4  (3.88)
2.Secondary Outcome
Title Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 (“not at all a problem”) to 3 (“problem is severe in degree”) and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum II included patients whose FMR1 gene was partially methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 20 18
Least Squares Mean (Standard Error)
Unit of Measure: Score
-6.8  (3.88) -2.8  (4.07) -5.7  (4.06) -3.5  (4.30)
3.Secondary Outcome
Title Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 (“not at all a problem”) to 3 (“problem is severe in degree”) and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales (irritability, lethargy/withdrawal, stereotypic behavior, hyperactivity, inappropriate speech and social avoidance) plus the total score were considered, and for which the total score ranks from 0 to 165. Stratum I included patients whose FMR1 gene was fully methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 22
Least Squares Mean (Standard Error)
Unit of Measure: Score
-11.8  (6.43) -3.4  (7.40) -9.4  (3.88)
4.Secondary Outcome
Title Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients
Hide Description The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum I included patients whose FMR1 gene was fully methylated.
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 15 22
Least Squares Mean (Standard Error)
Unit of Measure: Score
3.3  (0.30) 3.8  (0.34) 3.5  (0.22) 3.1  (0.18)
5.Secondary Outcome
Title Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients
Hide Description The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum II included patients whose FMR1 gene was partially methylated
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 20 18
Least Squares Mean (Standard Error)
Unit of Measure: Score
3.5  (0.19) 3.2  (0.20) 3.5  (0.20) 3.4  (0.21)
6.Secondary Outcome
Title Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I
Hide Description The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum I included patients whose FMR1 gene was fully methylated.
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 15 22
Measure Type: Number
Unit of Measure: Participants
1 (Very much improved) 1 0 0 0
2 (Much improved) 0 0 3 4
3 (Minimally improved) 2 1 3 11
4 (No change) 5 5 6 6
5 (Minimally worse) 0 0 3 1
6 (Much worse) 0 0 0 0
7 (Very much worse) 0 0 0 0
7.Secondary Outcome
Title Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II
Hide Description The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum II included patients whose FMR1 gene was partially methylated
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 20 18
Measure Type: Number
Unit of Measure: Participants
1 (Very much improved) 0 0 0 0
2 (Much improved) 3 5 3 4
3 (Minimally improved) 7 9 4 5
4 (No change) 12 5 11 8
5 (Minimally worse) 1 2 2 0
6 (Much worse) 0 0 0 1
7 (Very much worse) 0 0 0 0
8.Secondary Outcome
Title Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales: irritability (range 0 to 54); lethargy/withdrawal (range 0 to 39); stereotypic behavior (range 0 to 18); hyperactivity (range 0 to 30); inappropriate speech (range 0 to 12); and social avoidance (range 0 to 12) plus the total score (range 0 to 165) were considered. A negative change represents improvement. Stratum I included patients whose FMR1 gene was fully methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 16 22
Least Squares Mean (Standard Error)
Unit of Measure: Score
Irritability -3.5  (2.74) 2.2  (3.21) 4.2  (1.94) -2.5  (1.67)
Lethargy/withdrawal -1.4  (1.29) -3.5  (1.53) 1.3  (0.92) -1.9  (0.78)
Stereotypic behavior -2.2  (0.96) -2.2  (1.10) 1.2  (0.67) -1.7  (0.58)
Hyperactivity -3.9  (1.57) 1.1  (1.78) 0.7  (1.09) -1.3  (0.95)
Inappropriate speech -0.8  (0.69) 0.3  (0.78) 0.9  (0.48) -1.0  (0.42)
Social avoidance -0.2  (0.71) -1.0  (0.80) -0.1  (0.50) -1.2  (0.42)
9.Secondary Outcome
Title Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 ("not at all a problem") to 3 ("problem is severe in degree") and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to the modified FXS ABC-C algorithm (ABC-CFX) for which 55 items and six subscales: irritability (range 0 to 54); lethargy/withdrawal (range 0 to 39); stereotypic behavior (range 0 to 18); hyperactivity (range 0 to 30); inappropriate speech (range 0 to 12); and social avoidance (range 0 to 12) plus the total score (range 0 to 165) were considered. A negative change represents improvement. Stratum II included patients whose FMR1 gene was partially methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 20 18
Least Squares Mean (Standard Error)
Unit of Measure: Score
Irritability -1.5  (1.58) -1.3  (1.65) -0.8  (1.66) 0.0  (1.75)
Lethargy/withdrawal -0.8  (1.00) -0.4  (1.05) -0.3  (1.05) -0.8  (1.11)
Stereotypic behavior -1.4  (0.57) 0.1  (0.60) -1.0  (0.60) 0.1  (0.64)
Hyperactivity -1.5  (0.87) -0.3  (0.91) -1.8  (0.91) -0.8  (0.96)
Inappropriate speech -0.9  (0.44) -0.1  (0.46) -0.6  (0.47) -0.8  (0.50)
Social avoidance -0.7  (0.40) -0.9  (0.42) -1.1  (0.42) -1.1  (0.45)
10.Secondary Outcome
Title Proportion of Patients With Clinical Response, Where Response is Defined as a Reduction of at Least 25% From Baseline in the ABC-CFX Total Score and a Score of 1 (Very Much Improved) or 2 (Much Improved) on the CGI-I Scale, Stratum I
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 (“not at all a problem”) to 3 (“problem is severe in degree”) and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to ABC-CFX algorithm, for which 55 items and six subscales plus the total score were considered, and for which the total score ranks from 0 to 165. The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum I included patients whose FMR1 gene was fully methylated; Stratum II included patients whose FMR1 gene was partially methylated.
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Total is the number of patients with non-missing baseline ABC-CFX total score and at least one nonmissing post-baseline ABC-CFX total score and CGI-I assessment
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 17 22
Measure Type: Number
Unit of Measure: Number of participants
1 0 1 2
11.Secondary Outcome
Title Proportion of Patients With Clinical Response, Where Response is Defined as a Reduction of at Least 25% From Baseline in the ABC-CFX Total Score and a Score of 1 (Very Much Improved) or 2 (Much Improved) on the CGI-I Scale, Stratum II
Hide Description The ABC-C is a 58-item, caregiver-rated symptom checklist for assessing problem behaviors of children and adults with mental retardation at home, in residential facilities, and work training centers. The assessment was done by attributing to each item a score from 0 (“not at all a problem”) to 3 (“problem is severe in degree”) and the total score ranks from 0 to 174. The data collected from the full, 58-item ABC-C were analyzed according to ABC-CFX algorithm, for which 55 items and six subscales plus the total score were considered, and for which the total score ranks from 0 to 165. The Clinical Global Impression-Improvement (CGI-I) scale is a clinician rated scale used to assess treatment response in psychiatric patients, and the score ranges from 1 to 7 (with 1 being “very much improved”, 4 being “no change” to 7 being “very much worse”). Stratum I included patients whose FMR1 gene was fully methylated; Stratum II included patients whose FMR1 gene was partially methylated.
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Total is the number of patients with non-missing baseline ABC-CFX total score and at least one nonmissing post-baseline ABC-CFX total score and CGI-I assessment
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 22 19
Measure Type: Number
Unit of Measure: Number of participants
1 4 1 2
12.Secondary Outcome
Title Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum I
Hide Description The Repetitive Behavior Scale - Revised (RBS-R) is a rating tool that captures the breadth of repetitive behavior. It is a 43-item questionnaire filled by the caregivers. Each behavior assessed is rated from 0 (behavior does not occur) to 3 (behavior occurs and it is a severe problem). The total score ranks from 0 to 129. The behaviors are grouped into six domains: ritualistic behavior (range 0 to 18); sameness behavior (range 0 to 33); stereotypic behavior (range 0 to 18); self-injurious behavior (range 0 to 24); compulsive behavior (range 0 to 24); and restricted interests (range 0 to 12). A negative change represents improvement. Stratum I included patients whose FMR1 gene was fully methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 8 6 16 22
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Total score -2.3  (3.25) -8.5  (3.95) 1.5  (2.31) -6.2  (2.03)
Stereotyped behavior -1.0  (0.91) -1.5  (1.03) 0.5  (0.64) -1.0  (0.56)
Self-injurious behavior 0.0  (0.46) 0.8  (0.53) -0.2  (0.33) -1.1  (0.28)
Compulsive behavior -0.1  (0.84) -1.2  (1.06) 0.2  (0.60) -0.8  (0.51)
Ritualistic behavior -0.2  (0.93) -2.2  (1.12) 0.8  (0.66) -0.6  (0.57)
Sameness behavior -0.8  (1.19) -2.8  (1.42) 0.3  (0.85) -1.8  (0.73)
Restricted behavior 0.0  (0.65) -1.2  (0.74) -0.2  (0.46) -1.1  (0.39)
13.Secondary Outcome
Title Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum II
Hide Description The Repetitive Behavior Scale - Revised (RBS-R) is a rating tool that captures the breadth of repetitive behavior. It is a 43-item questionnaire filled by the caregivers. Each behavior assessed is rated from 0 (behavior does not occur) to 3 (behavior occurs and it is a severe problem). The total score ranks from 0 to 129. The behaviors are grouped into six domains: ritualistic behavior (range 0 to 18); sameness behavior (range 0 to 33); stereotypic behavior (range 0 to 18); self-injurious behavior (range 0 to 24); compulsive behavior (range 0 to 24); and restricted interests (range 0 to 12). A negative change represents improvement. Stratum II included patients whose FMR1 gene was partially methylated
Time Frame Baseline to week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS):all randomized patients who received at least 1 dose of study drug, had a baseline and at least 1 post-baseline assessment for the primary efficacy parameter. Only participants who had a value at the given time and the assessment was within the window for analysis were included
Arm/Group Title AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid Placebo
Hide Arm/Group Description:
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Overall Number of Participants Analyzed 23 21 20 18
Least Squares Mean (Standard Error)
Unit of Measure: Score on a scale
Total score -4.3  (2.36) -5.9  (2.51) -2.4  (2.47) -5.0  (2.67)
Stereotyped behavior 0.1  (0.53) 0.1  (0.56) 0.4  (0.56) 0.2  (0.59)
Self-injurious behavior -0.6  (0.53) -0.7  (0.55) 0.0  (0.56) 0.5  (0.59)
Compulsive behavior -0.9  (0.58) -1.5  (0.62) -0.2  (0.61) -1.0  (0.65)
Ritualistic behavior -0.1  (0.55) -1.5  (0.58) -1.0  (0.58) -1.8  (0.62)
Sameness behavior -2.2  (0.77) -1.6  (0.83) -1.3  (0.82) -2.8  (0.88)
Restricted behavior -0.6  (0.40) -0.7  (0.43) -0.4  (0.42) -0.3  (0.45)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Placebo AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid
Hide Arm/Group Description 2 capsules of placebo per intake 1 capsule of 25 mg and 1 capsule of placebo per intake 2 capsules of 25 mg per intake 1 capsule of 100 mg and 1 capsule of placebo per intake
All-Cause Mortality
Placebo AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/42 (4.76%)   0/31 (0.00%)   0/27 (0.00%)   0/39 (0.00%) 
Infections and infestations         
Appendicitis  1  1/42 (2.38%)  0/31 (0.00%)  0/27 (0.00%)  0/39 (0.00%) 
Nervous system disorders         
Convulsion  1  1/42 (2.38%)  0/31 (0.00%)  0/27 (0.00%)  0/39 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo AFQ056 25 mg Bid AFQ056 50 mg Bid AFQ056 100 mg Bid
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   17/42 (40.48%)   22/31 (70.97%)   9/27 (33.33%)   30/39 (76.92%) 
Gastrointestinal disorders         
Abdominal pain upper  1  0/42 (0.00%)  2/31 (6.45%)  0/27 (0.00%)  2/39 (5.13%) 
Diarrhoea  1  0/42 (0.00%)  3/31 (9.68%)  0/27 (0.00%)  2/39 (5.13%) 
Vomiting  1  0/42 (0.00%)  2/31 (6.45%)  1/27 (3.70%)  6/39 (15.38%) 
General disorders         
Fatigue  1  0/42 (0.00%)  0/31 (0.00%)  1/27 (3.70%)  2/39 (5.13%) 
Pyrexia  1  1/42 (2.38%)  2/31 (6.45%)  1/27 (3.70%)  3/39 (7.69%) 
Infections and infestations         
Influenza  1  2/42 (4.76%)  2/31 (6.45%)  0/27 (0.00%)  2/39 (5.13%) 
Nasopharyngitis  1  6/42 (14.29%)  7/31 (22.58%)  6/27 (22.22%)  5/39 (12.82%) 
Oral herpes  1  0/42 (0.00%)  2/31 (6.45%)  0/27 (0.00%)  2/39 (5.13%) 
Rhinitis  1  0/42 (0.00%)  2/31 (6.45%)  1/27 (3.70%)  1/39 (2.56%) 
Upper respiratory tract infection  1  4/42 (9.52%)  2/31 (6.45%)  1/27 (3.70%)  5/39 (12.82%) 
Metabolism and nutrition disorders         
Decreased appetite  1  1/42 (2.38%)  0/31 (0.00%)  1/27 (3.70%)  4/39 (10.26%) 
Nervous system disorders         
Headache  1  4/42 (9.52%)  4/31 (12.90%)  0/27 (0.00%)  10/39 (25.64%) 
Psychiatric disorders         
Anxiety  1  1/42 (2.38%)  0/31 (0.00%)  0/27 (0.00%)  2/39 (5.13%) 
Initial insomnia  1  0/42 (0.00%)  0/31 (0.00%)  0/27 (0.00%)  2/39 (5.13%) 
Insomnia  1  0/42 (0.00%)  0/31 (0.00%)  0/27 (0.00%)  6/39 (15.38%) 
Self injurious behaviour  1  0/42 (0.00%)  0/31 (0.00%)  0/27 (0.00%)  2/39 (5.13%) 
Respiratory, thoracic and mediastinal disorders         
Cough  1  1/42 (2.38%)  3/31 (9.68%)  1/27 (3.70%)  1/39 (2.56%) 
Nasal congestion  1  0/42 (0.00%)  3/31 (9.68%)  0/27 (0.00%)  1/39 (2.56%) 
Oropharyngeal pain  1  1/42 (2.38%)  2/31 (6.45%)  2/27 (7.41%)  1/39 (2.56%) 
Skin and subcutaneous tissue disorders         
Rash  1  0/42 (0.00%)  2/31 (6.45%)  0/27 (0.00%)  0/39 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS 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.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01357239     History of Changes
Other Study ID Numbers: CAFQ056B2214
2010-022638-96 ( EudraCT Number )
First Submitted: May 18, 2011
First Posted: May 20, 2011
Results First Submitted: December 29, 2014
Results First Posted: May 12, 2015
Last Update Posted: May 12, 2015