Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients - Full Text View

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00718341

Purpose

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.

Condition	Intervention	Phase
Fragile X Syndrome	Drug: AF056 Drug: Placebo	Phase II

Genetics Home Reference related topics: familial encephalopathy with neuroserpin inclusion bodies fragile X syndrome L1 syndrome

MedlinePlus related topics: Fragile X Syndrome

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Safety/Efficacy Study
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

Further study details as provided by Novartis:

Primary Outcome Measures:

Aberrant-Behavior Checklist- Community Edition

Secondary Outcome Measures:

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…)

Estimated Enrollment:	30
Study Start Date:	June 2008
Estimated Primary Completion Date:	May 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Active Comparator	Drug: AF056
2: Placebo Comparator	Drug: Placebo

Eligibility

Ages Eligible for Study:	18 Years to 35 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00718341

Locations

France
Novartis Investigator Site
Bron cedex, France, 69677
Italy
Novartis Investigator Site
Rome, Italy, 00168
Switzerland
Novartis Investigator Site
Lausanne, Switzerland, 1011

Sponsors and Collaborators

Novartis

Investigators

Principal Investigator:

Novartis

Novartis investigator site

More Information

Responsible Party:	Novartis ( External Affairs )
Study ID Numbers:	CAFQ056A2204
Study First Received:	July 17, 2008
Last Updated:	July 17, 2008
ClinicalTrials.gov Identifier:	NCT00718341
Health Authority:	France: Afssaps - French Health Products Safety Agency

Keywords provided by Novartis:

Fragile X Syndrome, adults, efficacy

Study placed in the following topic categories:

Chromosomal abnormalities
Mental Retardation
Heredodegenerative Disorders, Nervous System
Fragile X syndrome
Genetic Diseases, Inborn
X-linked mental retardation and macro-orchidism

Genetic Diseases, X-Linked
Chromosome Disorders
Neurologic Manifestations
Fragile X Syndrome
Congenital Abnormalities
Neurobehavioral Manifestations

Additional relevant MeSH terms:

Pathologic Processes
Disease
Syndrome

Nervous System Diseases
Sex Chromosome Disorders
Mental Retardation, X-Linked

ClinicalTrials.gov processed this record on January 13, 2009