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Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00718341
Recruitment Status : Completed
First Posted : July 18, 2008
Last Update Posted : February 11, 2020
Sponsor:
Information provided by:
Novartis

Study Description
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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.

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: AF056 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : June 2008
Actual Primary Completion Date : February 2009

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Arms and Interventions
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Arm Intervention/treatment
Active Comparator: 1 Drug: AF056
Placebo Comparator: 2 Drug: Placebo


Outcome Measures
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Primary Outcome Measures :
  1. Aberrant-Behavior Checklist- Community Edition

Secondary Outcome Measures :
  1. 28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
  2. 28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes 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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00718341


Locations
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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
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Principal Investigator: Novartis Novartis investigator site
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: External Affairs, Novartis
ClinicalTrials.gov Identifier: NCT00718341    
Other Study ID Numbers: CAFQ056A2204
First Posted: July 18, 2008    Key Record Dates
Last Update Posted: February 11, 2020
Last Verified: May 2010
Keywords provided by Novartis:
Fragile X Syndrome, adults, efficacy
Additional relevant MeSH terms:
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Fragile X Syndrome
Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
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