Status:
COMPLETED
Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
Lead Sponsor:
Novartis
Conditions:
Fragile X Syndrome
Eligibility:
MALE
18-35 years
Phase:
PHASE2
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 effic...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
June 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00718341
Start Date
June 1 2008
Last Update
February 11 2020
Active Locations (3)
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1
Novartis Investigator Site
Bron, France, 69677
2
Novartis Investigator Site
Rome, Italy, 00168
3
Novartis Investigator Site
Lausanne, Switzerland, 1011