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

Fragile X Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01253629
• Other study ID #: CAFQ056A2212
• Secondary ID: 2009-013667-19
• Status: Completed
• Phase: Phase 2
• Start date: November 2010
• Est. completion date: August 2013

Study information

• Verified date: March 2016
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 175
• Est. completion date: August 2013
• Est. primary completion date: August 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 45 Years

Eligibility

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

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• AFQ056
AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used
• Placebo
Placebo medication identical in appearance to active medication was provided

Locations

Country	Name	City	State
Australia	Novartis Investigative Site	Caulfield	Victoria
Australia	Novartis Investigative Site	Ryde	New South Wales
Australia	Novartis Investigative Site	Waratah	New South Wales
Canada	Novartis Investigative Site	Brampton	Ontario
Canada	Novartis Investigative Site	Sherbrooke	Quebec
Denmark	Novartis Investigative Site	Glostrup
France	Novartis Investigative Site	Bron Cedex
France	Novartis Investigative Site	Paris
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Mainz
Germany	Novartis Investigative Site	Tübingen
Germany	Novartis Investigative Site	Würzburg
Italy	Novartis Investigative Site	Genova	GE
Italy	Novartis Investigative Site	Roma	RM
Spain	Novartis Investigative Site	Málaga	Andalucia
Spain	Novartis Investigative Site	Sant Cugat	Cataluña
Switzerland	Novartis Investigative Site	Lausanne
Switzerland	Novartis Investigative Site	Zurich
United Kingdom	Novartis Investigative Site	Edinburgh
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Decatur	Georgia
United States	Novartis Investigative Site	Greenwood	South Carolina
United States	Novartis Investigative Site	Houston	Texas
United States	Novartis Investigative Site	Indianapolis	Indiana
United States	Novartis Investigative Site	Media	Pennsylvania
United States	Novartis Investigative Site	Nashville	Tennessee
United States	Novartis Investigative Site	Omaha	Nebraska
United States	Novartis Investigative Site	Phoenix	Arizona
United States	Novartis Investigative Site	Sacramento	California
United States	Novartis Investigative Site	Staten Island	New York

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I	The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174 in patients who were fully methylated (FM)	12 weeks
Secondary	Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II	The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech). Assessing the reduction in the (ABC-C) total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene.	12 weeks
Secondary	Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale	The CGI-I score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse")	12 weeks
Secondary	Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale	comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174	12 weeks
Secondary	The proportion of patients with clinical response in the ABC-C total score	response is defined as reduction of at least 25% from baseline in the ABC-CFX total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale at Week 12	12 weeks
Secondary	improvement of repetitive behavior as measured by changes in the RBS-R	The Repetitive Behavior Scale - Revised (RBS-R) includes six domains: ritualistic behavior, sameness behavior, stereotypic behavior, self-injurious behavior, compulsive behavior, and restricted interests. A negative change from baseline indicates improvement	Week 12

External Links

•   Results for CAFQ056A2212 from the Novartis Clinical Trial website