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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)

NCT ID: NCT01482143

Last Updated: 2020-12-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-03-31

Study Completion Date

2013-10-31

Brief Summary

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

Detailed Description

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Conditions

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Fragile X Syndrome

Keywords

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Fragile X Syndrome pharmacokinetics safety and tolerability

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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All Study subjects

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

Interventions

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AFQ056

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Sacramento, California, United States

Site Status

Novartis Investigative Site

Chicago, Illinois, United States

Site Status

Novartis Investigative Site

Nashville, Tennessee, United States

Site Status

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, Spain

Site Status

Countries

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Switzerland United States Spain

Related Links

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https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=11925

Results for CAFQ056B2154 can be found on the Novartis Clinical Trial Results Website

Other Identifiers

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2011-004867-65

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CAFQ056B2154

Identifier Type: -

Identifier Source: org_study_id