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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT01482143

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

5.3

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Fragile X Syndrome	Drug: AFQ056	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

21 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

None (Open Label)

Primary Purpose:

Other

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) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)

Study Start Date :

Mar 1, 2012

Actual Primary Completion Date :

Oct 1, 2013

Actual Study Completion Date :

Oct 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: All Study subjects	Drug: AFQ056

Arm

Intervention/Treatment

Experimental: All Study subjects

Drug: AFQ056

Outcome Measures

Primary Outcome Measures

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

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 11 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Sacramento	California	United States	95817
2	Novartis Investigative Site	Chicago	Illinois	United States	60612
3	Novartis Investigative Site	Nashville	Tennessee	United States	37232-7548
4	Novartis Investigative Site	Sant Cugat	Catalunya	Spain	08190