An open-label study to evaluate the long-term safety and tolerability of AFQ056 in adolescent patients with Fragile X Syndrome

The purpose of this study is to generate long-term safety, tolerability and
efficacy data for AFQ056 in eligible adolescent patients with FXS who
have participated in the CAFQ056B2214 study, the PK study CAFQ056B2131, or
another study of AFQ056 which included FXS patients below 18 years of age
provided the patient is at least 12 years of age at the time of entry into the
current study. Data from this study will be used to support long-term safety
data in labeling should the drug receive market authorization approval by
health authorities.

Primary
To evaluate the long-term safety and tolerability of AFQ056 in adolescent
patients with FXS as assessed by:
* Incidence and severity of adverse events and serious adverse events
* Changes in vital signs, laboratory assessments, and ECGs
* Monitoring the hypothalamic-pituitary-adrenal/thyroid axis function and
childhood developmental milestones

Secondary
To evaluate the long-term efficacy of AFQ056 treatment in both FM (Fully
Methylated) and PM (Partially Methylated) patients with FXS as assessed by:
* Change from baseline in the Aberrant Behavior Checklist * Community edition
(ABC-C) total score and subscale scores (using the FXS specific algorithm -
ABC-CFX)

This is a multi-center, open-label, flexible-dose study to evaluate the
long-term safety and tolerability of AFQ056 in adolescent patients with Fragile
X Syndrome.
Patients who meet all inclusion and none of the exclusion criteria at the
respective baseline visit will enter the open-label study and will be titrated
from a starting dose of 25 mg b.i.d. at one week intervals up to a maximum of
100 mg b.i.d. or the highest tolerated dose if the maximum dose of 100 mg
b.i.d. cannot be reached. Dose adjustments (up- and down-titrations) will be
allowed as needed to handle any tolerability issues during the study and to
ensure that patients reach their best welltolerated dose.
Initial visits (during titration) occur every two weeks with telephone contacts
between scheduled visits. Thereafter, the visit frequency will decrease to 1
month and then 3 month intervals until 24 months of treatment have been reached
(or until the study drug becomes available on the market (whichever occurs
later)).
One week after the conclusion of the open-label treatment period, patients will
undergo a safety follow-up visit.

100mg AFQ056 bid (or 25mg, 50mg or 75mg bid if 100mg bid cannot be tolerated)

Risks are possible side effects of study medicine or another medicine, and
those of taking the blood pressure or taking
blood.
The tests done during the study are standard medical tests. The most unpleasant
is often having blood samples taken.
The risks of taking blood may include fainting, pain and/or bruising.
Rarely, these may be a small blood clot or infection at the site of the needle
puncture.
Information from this study may help the patient and/or other people with
Fragile X Syndrome.