NCT01348087

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 148 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341).

Detailed description

A 148 patients were enrolled into this treatment extension trial conducted for at least 3 years. This extension trial was terminated after the decision to terminate the AFQ056 development program. The decision was based on the results of two randomized, double blind, placebo controlled phase IIb trials in adult and adolescent FXS patients (CAFQ056A2212 and CAFQ056B2214respectivly), both of which failed to demonstrate efficacy in the FXS population. As this extension trial was terminated, only the primary objective and safety are represented.

Conditions

• Fragile X Syndrome

Interventions

Type	Name	Description
DRUG	AFQ056	The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths, identical in appearance, will be used.

Timeline

• Start date: 2011-08-01
• Primary completion: 2014-09-01
• Completion: 2014-09-01
• First posted: 2011-05-05
• Last updated: 2016-05-25
• Results posted: 2016-05-25

Locations

28 sites across 10 countries: United States, Australia, Canada, Denmark, France, Germany, Italy, Spain, Switzerland, United Kingdom