Clinical Trial: Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome

Brief Summary: The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome:

  • 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 ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]
  • 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 ]


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: November 21, 2011
Date Started: March 2012
Date Completion:
Last Updated: September 29, 2014
Last Verified: September 2014