Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Novartis

Status and phase

Completed

Phase 2

Conditions

Fragile X Syndrome

Treatments

Drug: AF056

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00718341

CAFQ056A2204

Details and patient eligibility

About

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.

Enrollment

30 patients

Sex

Male

Ages

18 to 35 years old

Volunteers

No Healthy Volunteers

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.