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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01482143
First Posted: November 30, 2011
Last Update Posted: September 30, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
November 21, 2011
November 30, 2011
September 30, 2014
March 2012
October 2013   (Final data collection date for primary outcome measure)
  • 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 ]
Same as current
Complete list of historical versions of study NCT01482143 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
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) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
Not Provided
Interventional
Phase 1
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Fragile X Syndrome
Drug: AFQ056
Experimental: All Study subjects
Intervention: Drug: AFQ056
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
21
October 2013
October 2013   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Genetically confirmed diagnosis of FXS
  • At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.

Exclusion Criteria:

  • Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
  • History of hypersensitivity to AFQ056 or any mGluR antagonist.
  • Female patients who are confirmed or suspected to be sexually active.
  • History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
  • Smokers.
  • Loss of ≥10% of total blood volume within 8 weeks (or less if required for this age group and/or by local regulation) prior to dosing or longer if required for this age group and/or by local regulation.
  • Significant illness that did not completely resolve at least four weeks prior to the first baseline visit.
  • Any abnormal laboratory values at screening or first baseline that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
  • Use of (or use within at least 5 half lives before dosing) concomitant medications that are strong/moderate inhibitors or inducers of CYP1A1/2, CYP2C9/19 or CYP3A4
  • History or presence of Hepatitis B/C or HIV at screening
Sexes Eligible for Study: All
3 Years to 11 Years   (Child)
No
Contact information is only displayed when the study is recruiting subjects
Spain,   United States
Switzerland
 
NCT01482143
CAFQ056B2154
2011-004867-65 ( EudraCT Number )
Not Provided
Not Provided
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
September 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP