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

This study is currently recruiting participants.

Verified January 2013 by Novartis

Sponsor:

Information provided by (Responsible Party):

Novartis ( Novartis Pharmaceuticals )

ClinicalTrials.gov Identifier:

NCT01253629

First received: December 2, 2010

Last updated: January 9, 2013

Last verified: January 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

December 2, 2010

Last Updated Date

January 9, 2013

Start Date ^ICMJE

November 2010

Estimated Primary Completion Date

December 2013 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01253629 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Change from baseline in behavioral symptoms of Fragile X Syndrome using the ABC-C Total score in Stratum II [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-C total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

Brief Summary

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.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition ^ICMJE

Fragile X Syndrome

Intervention ^ICMJE

Drug: AFQ056
Drug: Placebo

Study Arm (s)

Experimental: 25 mg bid
Intervention: Drug: AFQ056
Experimental: 50 mg bid
Intervention: Drug: AFQ056
Experimental: 100 mg bid
Intervention: Drug: AFQ056
Placebo Comparator: Placebo
Intervention: Drug: Placebo

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Recruiting

Estimated Enrollment ^ICMJE

175

Estimated Completion Date

December 2013

Estimated Primary Completion Date

December 2013 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Other protocol-defined inclusion/exclusion criteria may apply

Gender

Both

Ages

18 Years to 45 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Novartis Pharmaceuticals

+1(862)778-8300

Location Countries ^ICMJE

United States, Australia, Canada, Denmark, France, Germany, Italy, Spain, Switzerland, United Kingdom

Administrative Information

NCT Number ^ICMJE

NCT01253629

Other Study ID Numbers ^ICMJE

CAFQ056A2212, 2009-013667-19

Has Data Monitoring Committee

Not Provided

Responsible Party

Novartis ( Novartis Pharmaceuticals )

Study Sponsor ^ICMJE

Novartis Pharmaceuticals

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Novartis Pharmaceuticals

Information Provided By

Novartis

Verification Date

January 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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