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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
119
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.
Novartis Investigative Site
Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
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 participants 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 participants 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.
Time frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
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Sacramento, California, United States
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Decatur, Georgia, United States
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Chicago, Illinois, United States
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Boston, Massachusetts, United States
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Omaha, Nebraska, United States
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Staten Island, New York, United States
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Nashville, Tennessee, United States
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Westmead, New South Wales, Australia
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Parkville, Victoria, Australia
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Brussels, Belgium
...and 18 more locations