Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Study Overview
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Brief Summary
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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.

Official Title
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A Multi-centre, Randomized, Double-blind, Placebo Controlled, Two-period, Crossover Proof-of-concept Study in Male Patients With Fragile X Syndrome to Assess the Efficacy, Safety and Tolerability of Multiple Oral Doses of AFQ056

Conditions
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Fragile X Syndrome

Intervention / Treatment
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* Drug: AF056
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: 1<br> | Drug: AF056<br> <br> |
| Placebo Comparator: 2<br> | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Aberrant-Behavior Checklist- Community Edition |  |  | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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…) |  |  | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fragile X Syndrome, adults, efficacy