Status:

COMPLETED

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

Lead Sponsor:

Novartis Pharmaceuticals

Conditions:

Fragile X Syndrome

Eligibility:

All Genders

3-11 years

Phase:

PHASE1

Brief Summary

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

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

March 1 2012

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

October 1 2013

Estimated Enrollment :

21 Patients enrolled

Trial Details

Trial ID

NCT01482143

Start Date

March 1 2012

End Date

October 1 2013

Last Update

December 8 2020

Active Locations (4)

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Page 1 of 1 (4 locations)

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 del Vallès, Catalonia, Spain, 08190