Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
- Registration Number
- NCT01482143
- Lead Sponsor
- Novartis Pharmaceuticals
- 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 Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 21
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description All Study subjects AFQ056 -
- Primary Outcome Measures
Name Time Method The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf) 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: 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: 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
- Secondary Outcome Measures
Name Time Method Physical examination Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 Vital signs and body measurements Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 Electrocardiograms Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 hematology Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 blood chemistry Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 neurological examination Screening: once anytime between Day -30 and Day -1; once on Day 7 Adverse events (AE) monitoring During the study (total of approximately 32 days) and 3 days after study completion Serious adverse events (SAE) monitoring During the study (total of approximately 32 days) and 30 days after study completion
Trial Locations
- Locations (1)
Novartis Investigative Site
🇪🇸Sant Cugat, Catalunya, Spain