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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:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01482143
First received: November 21, 2011
Last updated: September 29, 2014
Last verified: September 2014

November 21, 2011
September 29, 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: Yes ]
  • 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 ] [ Designated as safety issue: Yes ]
  • Electrocardiograms [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ] [ Designated as safety issue: Yes ]
  • hematology [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ] [ Designated as safety issue: Yes ]
  • blood chemistry [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ] [ Designated as safety issue: Yes ]
  • neurological examination [ Time Frame: Screening: once anytime between Day -30 and Day -1; once on Day 7 ] [ Designated as safety issue: Yes ]
  • Adverse events (AE) monitoring [ Time Frame: During the study (total of approximately 32 days) and 3 days after study completion ] [ Designated as safety issue: Yes ]
  • Serious adverse events (SAE) monitoring [ Time Frame: During the study (total of approximately 32 days) and 30 days after study completion ] [ Designated as safety issue: Yes ]
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: 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
Both
3 Years to 11 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Spain
 
NCT01482143
CAFQ056B2154, 2011-004867-65
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