The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
21
Novartis Investigative Site
Sacramento, California, United States
Novartis Investigative Site
Chicago, Illinois, United States
Novartis Investigative Site
Nashville, Tennessee, United States
Novartis Investigative Site
Sant Cugat del Vallès, Catalonia, Spain
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Maximum observed plasma concentration (Cmax)
Time frame: Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Physical examination
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Vital signs and body measurements
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Electrocardiograms
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
hematology
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
blood chemistry
Time frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
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neurological examination
Time frame: Screening: once anytime between Day -30 and Day -1; once on Day 7
Adverse events (AE) monitoring
Time frame: During the study (total of approximately 32 days) and 3 days after study completion
Serious adverse events (SAE) monitoring
Time frame: During the study (total of approximately 32 days) and 30 days after study completion