Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

This study is currently recruiting participants.
Verified January 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01348087
First received: May 3, 2011
Last updated: January 14, 2014
Last verified: January 2014
  Purpose

The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adult patients with FXS who have participated in the CAFQ056A2212 core study and patients who have participated in the previous proof-of-concept study CAFQ056A2204.


Condition Intervention Phase
Fragile X Syndrome
Drug: AFQ056
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Evaluation of the safety and tolerability of AFQ056 in adult patients with FXS as assessed by: Incidence and severity of adverse events and serious adverse events and change in vital signs, laboratory assessments, and ECGs. [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change from baseline in the Aberrant Behavior Checklist - Community edition (ABC-C) total score and subscale scores [ Time Frame: 24 months ] [ Designated as safety issue: No ]
  • Rating of global improvement of symptoms in Fragile X patients using the Clinical Global Impression - Improvement (CGI-I) scale [ Time Frame: 24 months ] [ Designated as safety issue: No ]
  • Change from baseline in the Repetitive Behavior Symptom - Research version (RBS) total score and subscale score [ Time Frame: 24 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 200
Study Start Date: August 2011
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AFQ056 100 mg (Bid) Drug: AFQ056

  Eligibility

Ages Eligible for Study:   18 Years to 46 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have been enrolled in Studies CAFQ056A2204 or CAFQ056A2212
  • Has a caregiver or caregivers who spend(s), on average, at least 6 hours per day with the patient, who is/are willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.

Exclusion Criteria:

  • Any advanced, severe or unstable disease
  • History of severe self-injurious behavior
  • History of uncontrolled seizure disorder or resistant to therapy within the past 2 years (Patients who are clinically stable under anti-convulsant therapy for the past 2 years are not excluded)
  • History of clinically significant allergies requiring hospitalization or non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.)
  • Any treatment regimen, including psychotropic and/or anticonvulsant therapy that has not been stable for ≥ 6 weeks prior to randomization
  • Using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4
  • Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of randomization
  • Planning to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01348087

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

  Hide Study Locations
Locations
United States, Arizona
Novartis Investigative Site Active, not recruiting
Phoenix, Arizona, United States, 85006
United States, California
Novartis Investigative Site Recruiting
Sacramento, California, United States, 95817
United States, Georgia
Novartis Investigative Site Active, not recruiting
Decatur, Georgia, United States, 30033
United States, Illinois
Novartis Investigative Site Active, not recruiting
Chicago, Illinois, United States, 60612
United States, Indiana
Novartis Investigative Site Recruiting
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Novartis Investigative Site Recruiting
Boston, Massachusetts, United States, 02115
United States, Nebraska
Novartis Investigative Site Active, not recruiting
Omaha, Nebraska, United States, 68198-5575
United States, New York
Novartis Investigative Site Recruiting
Staten Island, New York, United States, 10314
United States, Pennsylvania
Novartis Investigative Site Active, not recruiting
Media, Pennsylvania, United States, 19063
United States, South Carolina
Novartis Investigative Site Not yet recruiting
Greenwood, South Carolina, United States, 29646
United States, Tennessee
Novartis Investigative Site Recruiting
Nashville, Tennessee, United States, 37212
United States, Texas
Novartis Investigative Site Not yet recruiting
Houston, Texas, United States, 77090
United States, Washington
Novartis Investigative Site Withdrawn
Seattle, Washington, United States, 98105
Australia, New South Wales
Novartis Investigative Site Active, not recruiting
Ryde, New South Wales, Australia, 2112
Novartis Investigative Site Active, not recruiting
Waratah, New South Wales, Australia, 2298
Australia, Victoria
Novartis Investigative Site Active, not recruiting
Caulfield, Victoria, Australia, 3161
Brazil
Novartis Investigative Site Withdrawn
Porto Alegre, RS, Brazil, 90035-903
Novartis Investigative Site Withdrawn
Florianopolis, SC, Brazil, 88015-440
Novartis Investigative Site Withdrawn
Ribeirao Preto, SP, Brazil, 14048-900
Novartis Investigative Site Withdrawn
Sao Paulo, SP, Brazil, 05508-090
Novartis Investigative Site Withdrawn
Sao Paulo, SP, Brazil, 04020-041
Canada, Ontario
Novartis Investigative Site Active, not recruiting
Brampton, Ontario, Canada, L6Y 1M5
Canada, Quebec
Novartis Investigative Site Withdrawn
Montreal, Quebec, Canada, H3T1C5
Novartis Investigative Site Active, not recruiting
Sherbrooke, Quebec, Canada, J1H 5N4
Denmark
Novartis Investigative Site Active, not recruiting
Glostrup, Denmark, 2600
France
Novartis Investigative Site Recruiting
Bron Cedex, France, 69677
Novartis Investigative Site Recruiting
Paris, France, 75013
Germany
Novartis Investigative Site Active, not recruiting
Berlin, Germany, 12200
Novartis Investigative Site Active, not recruiting
Mainz, Germany, 55131
Novartis Investigative Site Active, not recruiting
Tübingen, Germany, 72076
Novartis Investigative Site Active, not recruiting
Würzburg, Germany, 97070
Italy
Novartis Investigative Site Recruiting
Genova, GE, Italy, 16147
Novartis Investigative Site Withdrawn
Roma, RM, Italy, 00168
Novartis Investigative Site Withdrawn
Padova, Italy, 35128
Netherlands
Novartis Investigative Site Withdrawn
Rotterdam, Netherlands, 3015 CE
Spain
Novartis Investigative Site Active, not recruiting
Málaga, Andalucia, Spain, 29010
Novartis Investigative Site Active, not recruiting
Sant Cugat, Cataluña, Spain, 08190
Switzerland
Novartis Investigative Site Recruiting
Lausanne, Switzerland, 1011
Novartis Investigative Site Active, not recruiting
Zurich, Switzerland, 8091
United Kingdom
Novartis Investigative Site Active, not recruiting
Edinburgh, United Kingdom, EH10 5HF
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: NCT01348087     History of Changes
Other Study ID Numbers: CAFQ056B2279, 2011-001952-12
Study First Received: May 3, 2011
Last Updated: January 14, 2014
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: National Health and Medical Research Council
Switzerland: Swissmedic

Keywords provided by Novartis:
Fragile X Syndrome
Martin-Bell Syndrome
Genetic Diseases
X-Linked
Escalante's syndrome

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 April 16, 2014