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 Adult Patients With Fragile X Syndrome

Fragile X Syndrome

AFQ056

Placebo

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

The ABC-C is a 58-item questionnaire that should have been completed as much as possible
by the same rater. It is comprised of five subscales (irritability, lethargy/social
withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total
score which ranks from 0 to 174 in patients who were fully methylated (FM)

The ABC-C is a 58-item questionnaire that should have been completed as much as possible
by the same rater. It is comprised of five subscales (irritability, lethargy/social
withdrawal, stereotypic behavior, hyperactivity and inappropriate speech). Assessing the
reduction in the (ABC-C) total score after 12 weeks of treatment in FXS patients with
partially methylated (PM) FMR1 gene.

The CGI-I score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no
change" to 7 being "very much worse")

comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic
behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0
to 174

response is defined as 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 at Week 12

The Repetitive Behavior Scale - Revised (RBS-R) includes six domains: ritualistic
behavior, sameness behavior, stereotypic behavior, self-injurious behavior, compulsive
behavior, and restricted interests. A negative change from baseline indicates improvement

1 capsule of 25 mg and 1 capsule of placebo per intake

1 capsule of 100 mg and 1 capsule of placebo per intake

Safety and Efficacy of AFQ056 in Adult 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