Fragile X Syndrome
Conditions
Keywords
Fragile X Syndrome, pharmacokinetics, safety and tolerability
Brief summary
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
Interventions
DRUGAFQ056
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 11 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 |
| Physical examination | Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after 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 |
| neurological examination | Screening: once anytime between Day -30 and Day -1; once on 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 |
Countries
Spain, United States
Outcome results
None listed