Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

This study has been completed.

Sponsor:

Information provided by:

Novartis

ClinicalTrials.gov Identifier:

NCT00718341

First received: July 17, 2008

Last updated: May 5, 2010

Last verified: May 2010

History of Changes

Tracking Information

First Received Date ^ICMJE

July 17, 2008

Last Updated Date

May 5, 2010

Start Date ^ICMJE

June 2008

Primary Completion Date

February 2009 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Aberrant-Behavior Checklist- Community Edition

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00718341 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

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…)

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

Official Title ^ICMJE

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

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 Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment

Condition ^ICMJE

Fragile X Syndrome

Intervention ^ICMJE

Drug: AF056
Drug: Placebo

Study Arm (s)

Active Comparator: 1
Intervention: Drug: AF056
Placebo Comparator: 2
Intervention: Drug: Placebo

Publications *

Jacquemont S, Curie A, des Portes V, Torrioli MG, Berry-Kravis E, Hagerman RJ, Ramos FJ, Cornish K, He Y, Paulding C, Neri G, Chen F, Hadjikhani N, Martinet D, Meyer J, Beckmann JS, Delange K, Brun A, Bussy G, Gasparini F, Hilse T, Floesser A, Branson J, Bilbe G, Johns D, Gomez-Mancilla B. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci Transl Med. 2011 Jan 5;3(64):64ra1.

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

Not Provided

Primary Completion Date

February 2009 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Gender

Male

Ages

18 Years to 35 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

France, Italy, Switzerland

Administrative Information

NCT Number ^ICMJE

NCT00718341

Other Study ID Numbers ^ICMJE

CAFQ056A2204

Has Data Monitoring Committee

Responsible Party

External Affairs, Novartis

Study Sponsor ^ICMJE

Novartis

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Novartis

Novartis investigator site

Information Provided By

Novartis

Verification Date

May 2010

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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