Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
Phase 2
Completed
- Conditions
- Fragile X Syndrome
- Interventions
- Drug: AF056Drug: Placebo
- Registration Number
- NCT00718341
- Lead Sponsor
- Novartis
- Brief Summary
This study will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg b.i.d. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 30
Inclusion Criteria
- Male, non-smoking patients between 18 and 35 years of age (both inclusive).
- Patients with fmr1 full mutation (> 200 CGG repeats)
- Patients with a Clinical Global Impression Severity Score (CGI-S) of > 4 (moderately ill)
- Patients with a score of >20 in the ABC-C scale (at screening)
- Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test
Exclusion Criteria
- Patients with DSM-IV diagnosis of schizophrenia, history and/or presence of psychosis, confusional states and/or repeated hallucinations.
- Patients with a history of seizures in the past 5 years without any therapeutic treatment controlling the disorders.
- Patients under stable anti-convulsant therapies that experienced seizures in the 2 years prior to randomization
- Patients with ECG abnormalities, autonomic dysfunctions, bronchospastic diseases, drug or atopic allergy
- Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs
- Patients using (or have used within four weeks before randomization) concomitant medications that are potent inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir, etc.)
Other protocol-defined inclusion/exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description 1 AF056 - 2 Placebo -
- Primary Outcome Measures
Name Time Method Aberrant-Behavior Checklist- Community Edition
- Secondary Outcome Measures
Name Time Method 28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement) 28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)
Trial Locations
- Locations (1)
Novartis Investigator Site
🇨🇭Lausanne, Switzerland