Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
- Registration Number
- NCT01253629
- Lead Sponsor
- Novartis Pharmaceuticals
- 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 available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 175
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description 25 mg bid AFQ056 AFQ056 1 capsule of 25 mg and 1 capsule of placebo per intake Placebo Placebo 2 capsules of placebo per intake 100 mg bid AFQ056 AFQ056 1 capsule of 100 mg and 1 capsule of placebo per intake 50 mg bid AFQ056 AFQ056 2 capsules of 25 mg per intake
- Primary Outcome Measures
Name Time Method Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I 12 weeks 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)
- Secondary Outcome Measures
Name Time Method The proportion of patients with clinical response in the ABC-C total score 12 weeks 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
Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II 12 weeks 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.
Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale 12 weeks comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174
Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale 12 weeks 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")
improvement of repetitive behavior as measured by changes in the RBS-R Week 12 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
Trial Locations
- Locations (1)
Novartis Investigative Site
🇬🇧Edinburgh, United Kingdom
Novartis Investigative Site🇬🇧Edinburgh, United Kingdom