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Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

This study is currently recruiting participants.
Verified January 2013 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01253629
First received: December 2, 2010
Last updated: January 9, 2013
Last verified: January 2013
History of Changes
  Purpose

This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.


Condition Intervention Phase
Fragile X Syndrome
 Drug: AFQ056
Drug: Placebo
 Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:
  • Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline in behavioral symptoms of Fragile X Syndrome using the ABC-C Total score in Stratum II [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
  • Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-C total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 175
Study Start Date: November 2010
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 25 mg bid Drug: AFQ056
Experimental: 50 mg bid Drug: AFQ056
Experimental: 100 mg bid Drug: AFQ056
Placebo Comparator: Placebo Drug: Placebo

  Eligibility

Ages Eligible for Study:   18 Years to 45 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at Visit 1

Exclusion Criteria:

  • Advanced, severe or unstable disease that may interfere with the study outcome evaluations
  • Cancer within the past 5 years, other than localized skin cancer
  • Current treatment with more than two psychoactive medications, excluding anti-epileptics
  • History of severe self-injurious behavior

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01253629

Contacts
Contact: Novartis Pharmaceuticals +1(862)778-8300

  Show 36 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01253629     History of Changes
Other Study ID Numbers: CAFQ056A2212, 2009-013667-19
Study First Received: December 2, 2010
Last Updated: January 9, 2013
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Denmark: Danish Medicines Agency
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: BfArM
Italy: Ethics Committee
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration
Switzerland: Swissmedic
Spain: Agencia Española de Medicamentos y Productos Sanitarios

Keywords provided by Novartis:
Fragile X Syndrome
Martin-Bell Syndrome
Genetic Diseases
X-Linked

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

ClinicalTrials.gov processed this record on May 21, 2013