Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome Martin-Bell Syndrome Genetic Diseases X-Linked Escalante's syndrome
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 119
Healthy volunteers: no

Brief summary:
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.

Detailed description:

Arms:
arm 1: All patients will initiate treatment with AFQ056 at a starting dose of 25 mg b.i.d. The dose will be titrated from 25 mg b.i.d to 50 mg b.i.d., 75 mg b.i.d. and 100 mg b.i.d. at weekly intervals. Dose adjustments (up- and down-titrations) will be permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose, not to exceed 100 mg b.i.d.

Interventions: intervention 1: AFQ056
Intervention descriptions: intervention 1: The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths,25mg and 100 mg, identical in appearance, will be used.

Protocol document: