This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and
effective in treating the behavioral symptoms of Fragile X Syndrome.

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

AFQ056

Placebo

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome

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

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

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

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.

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

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

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

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.

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

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

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

About this trial

Study ID

Status

Type

Phase

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status