Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Fragile X Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00718341
• Other study ID #: CAFQ056A2204
• Status: Completed
• Phase: Phase 2
• Start date: June 2008

Study information

• Verified date: May 2010
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. primary completion date: February 2009
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 35 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• AF056

• Placebo

Locations

Country	Name	City	State
France	Novartis Investigator Site	Bron cedex
Italy	Novartis Investigator Site	Rome
Switzerland	Novartis Investigator Site	Lausanne

Sponsors (1)

Lead Sponsor	Collaborator
Novartis

Countries where clinical trial is conducted

France,  Italy,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Aberrant-Behavior Checklist- Community Edition
Secondary	28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
Secondary	28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)

External Links

•   Results for CAFQ056A2204 on the Novartis Clinical Trials website