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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
  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 2

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

Resource links provided by NLM:

U.S. FDA Resources

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…)

Enrollment: 30
Study Start Date: June 2008
Primary Completion Date: February 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 Drug: AF056
Placebo Comparator: 2 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

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: External Affairs, Novartis
ClinicalTrials.gov Identifier: NCT00718341     History of Changes
Other Study ID Numbers: CAFQ056A2204
Study First Received: July 17, 2008
Last Updated: May 5, 2010
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Novartis:
Fragile X Syndrome, adults, efficacy

Additional relevant MeSH terms:
Fragile X Syndrome
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
 Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on May 16, 2013