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

Novartis

Status and phase

Completed

Phase 1

Conditions

Fragile X Syndrome

Treatments

Drug: AFQ056

Study type

Interventional

Funder types

Industry

Identifiers

2011-004867-65 (EudraCT Number)

CAFQ056B2154

Details and patient eligibility

About

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

Enrollment

21 patients

Sex

All

Ages

3 to 11 years old

Volunteers

No 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