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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

Brief 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 Summary: 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.
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). [ Time Frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial ]

Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which patients entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study. AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 patients are shown under ('Prior to Ext. first dose'). AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated


Original Primary Outcome: Evaluation of the safety and tolerability of AFQ056 in adult patients with FXS as assessed by: Incidence and severity of adverse events and serious adverse events and change in vital signs, laboratory assessments, and ECGs. [ Time Frame: 9 months ]

Current Secondary Outcome:

Original Secondary Outcome:

  • Change from baseline in the Aberrant Behavior Checklist - Community edition (ABC-C) total score and subscale scores [ Time Frame: 9 months ]
  • Rating of global improvement of symptoms in Fragile X patients using the Clinical Global Impression - Improvement (CGI-I) scale [ Time Frame: 9 months ]
  • Change from baseline in the Repetitive Behavior Symptom - Research version (RBS) total score and subscale score [ Time Frame: 9 months ]


Information By: Novartis

Dates:
Date Received: May 3, 2011
Date Started: August 2011
Date Completion:
Last Updated: April 11, 2016
Last Verified: April 2016