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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01357239
• Date of registration: 18/05/2011
• Prospective Registration: No
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
• Scientific title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome
• Date of first enrolment: May 2011
• Target sample size: 139
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01357239
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Brazil, Canada, Denmark, France, Germany, Indonesia, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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

- Weigh less than 32 kg

- Females who are sexually active

Other protocol-defined inclusion/exclusion criteria may apply



Age minimum: 12 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Drug: AFQ056

Drug: Placebo

Primary Outcome(s)

Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid [Time Frame: Baseline to week 12]

Secondary Outcome(s)

Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum I [Time Frame: Baseline to week 12]

Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum II [Time Frame: Baseline to week 12]

Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I [Time Frame: Baseline to week 12]

Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients [Time Frame: 12 weeks]

Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I [Time Frame: 12 weeks]

Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056 [Time Frame: Baseline to week 12]

Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients [Time Frame: 12 weeks]

Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid) [Time Frame: Baseline to week 12]

Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II [Time Frame: Baseline to week 12]

Proportion of Patients With Clinical Response, Where Response is Defined as a 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, Stratum II [Time Frame: 12 weeks]

Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II [Time Frame: 12 weeks]

Proportion of Patients With Clinical Response, Where Response is Defined as a 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, Stratum I [Time Frame: 12 weeks]

Secondary ID(s)

2010-022638-96

CAFQ056B2214

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 12/05/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01357239