Clinical trial NCT01253629: Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Official title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome
Martin-Bell Syndrome
Genetic Diseases
X-Linked
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-12-03
Enrollment: 175
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
timeFrame: 12 weeks
Secondary outcomes:
measure: Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II
timeFrame: 12 weeks
measure: Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale
timeFrame: 12 weeks
measure: Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale
timeFrame: 12 weeks
measure: The proportion of patients with clinical response in the ABC-C total score
timeFrame: 12 weeks
measure: improvement of repetitive behavior as measured by changes in the RBS-R
timeFrame: Week 12
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Sacramento
country: United States
facility: Novartis Investigative Site
state: California
city: Decatur
country: United States
facility: Novartis Investigative Site
state: Georgia
city: Chicago
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Indianapolis
country: United States
facility: Novartis Investigative Site
state: Indiana
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Omaha
country: United States
facility: Novartis Investigative Site
state: Nebraska
city: Staten Island
country: United States
facility: Novartis Investigative Site
state: New York
city: Media
country: United States
facility: Novartis Investigative Site
state: Pennsylvania
city: Greenwood
country: United States
facility: Novartis Investigative Site
state: South Carolina
city: Nashville
country: United States
facility: Novartis Investigative Site
state: Tennessee
city: Houston
country: United States
facility: Novartis Investigative Site
state: Texas
city: Ryde
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Waratah
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Caulfield
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Brampton
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Sherbrooke
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Glostrup
country: Denmark
facility: Novartis Investigative Site
city: Bron Cedex
country: France
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Mainz
country: Germany
facility: Novartis Investigative Site
city: Tübingen
country: Germany
facility: Novartis Investigative Site
city: Würzburg
country: Germany
facility: Novartis Investigative Site
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Málaga
country: Spain
facility: Novartis Investigative Site
state: Andalucia
city: Sant Cugat
country: Spain
facility: Novartis Investigative Site
state: Cataluña
city: Lausanne
country: Switzerland
facility: Novartis Investigative Site
city: Zurich
country: Switzerland
facility: Novartis Investigative Site
city: Edinburgh
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: 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.