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Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

NCT ID: NCT01357239

Last Updated: 2015-05-12

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

139 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-05-31

Study Completion Date

2014-01-31

Brief Summary

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This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

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Detailed Description

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Conditions

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Fragile X Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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25 mg bid

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

50 mg bid

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

100 mg bid

Group Type EXPERIMENTAL

AFQ056

Intervention Type DRUG

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Interventions

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AFQ056

Intervention Type DRUG

Placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at Visit 1

Exclusion Criteria

* Advanced, severe or unstable disease that may interfere with the study outcome evaluations
* Cancer within the past 5 years, other than localized skin cancer
* Current treatment with more than two psychoactive medications, excluding anti-epileptics
* History of severe self-injurious behavior
* Weigh less than 32 kg
* Females who are sexually active
Minimum Eligible Age

12 Years

Maximum Eligible Age

17 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

Decatur, Georgia, United States

Site Status

Novartis Investigative Site

Chicago, Illinois, United States

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Novartis Investigative Site

Boston, Massachusetts, United States

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Novartis Investigative Site

Omaha, Nebraska, United States

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Novartis Investigative Site

Staten Island, New York, United States

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Novartis Investigative Site

Nashville, Tennessee, United States

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Novartis Investigative Site

Houston, Texas, United States

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Novartis Investigative Site

Westmead, New South Wales, Australia

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Novartis Investigative Site

Parkville, Victoria, Australia

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Novartis Investigative Site

Brussels, , Belgium

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Novartis Investigative Site

Leuven, , Belgium

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Novartis Investigative Site

Edmonton, Alberta, Canada

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Novartis Investigative Site

Vancouver, British Columbia, Canada

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Montreal, Quebec, Canada

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Novartis Investigative Site

Glostrup Municipality, , Denmark

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Novartis Investigative Site

Bron, , France

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Paris, , France

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Mainz, Germany, Germany

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Dresden, , Germany

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München, , Germany

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München, , Germany

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Tübingen, , Germany

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Würzburg, , Germany

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Novartis Investigative Site

Semarang, Central Java, Indonesia

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Novartis Investigative Site

Ramat Gan, , Israel

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Novartis Investigative Site

Genova, GE, Italy

Site Status

Novartis Investigative Site

Roma, RM, Italy

Site Status

Novartis Investigative Site

Padua, , Italy

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Novartis Investigative Site

Rotterdam, , Netherlands

Site Status

Novartis Investigative Site

Málaga, Andalusia, Spain

Site Status

Novartis Investigative Site

Sabadell, Barcelona, Spain

Site Status

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, Spain

Site Status

Novartis Investigative Site

Spånga, , Sweden

Site Status

Novartis Investigative Site

Lausanne, , Switzerland

Site Status

Novartis Investigative Site

Zurich, , Switzerland

Site Status

Novartis Investigative Site

Istanbul, , Turkey (Türkiye)

Site Status

Novartis Investigative Site

Edinburgh, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada Denmark France Germany Indonesia Israel Italy Netherlands Spain Sweden Switzerland Turkey (Türkiye) United Kingdom

References

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Bailey DB Jr, Berry-Kravis E, Wheeler A, Raspa M, Merrien F, Ricart J, Koumaras B, Rosenkranz G, Tomlinson M, von Raison F, Apostol G. Mavoglurant in adolescents with fragile X syndrome: analysis of Clinical Global Impression-Improvement source data from a double-blind therapeutic study followed by an open-label, long-term extension study. J Neurodev Disord. 2016;8:1. doi: 10.1186/s11689-015-9134-5. Epub 2015 Dec 15.

Reference Type DERIVED
PMID: 26855682 (View on PubMed)

Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

Reference Type DERIVED
PMID: 26764156 (View on PubMed)

Other Identifiers

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2010-022638-96

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CAFQ056B2214

Identifier Type: -

Identifier Source: org_study_id

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