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

COMPLETEDPHASE1

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

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)

NCT01482143•Novartis Pharmaceuticals

View on ClinicalTrials.gov

Summary

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

Eligibility

Age

3 - 11 years

Sex

ALL

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

Locations (4)

Novartis Investigative Site

Sacramento, California, United States

Novartis Investigative Site

Chicago, Illinois, United States

Novartis Investigative Site

Nashville, Tennessee, United States

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, Spain

Key Dates

Start Date

March 1, 2012

Primary Completion Date

October 1, 2013

Completion Date

October 1, 2013

Last Updated

December 8, 2020

Enrollment

ACTUAL participants

Conditions

Fragile X Syndrome

Interventions

AFQ056

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 8, 2020Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants

Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome

Study of SPG601 in Adult Men With Fragile X Syndrome

An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome

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