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

May 3, 2011
January 14, 2014
August 2011
December 2015   (final data collection date for primary outcome measure)
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 ]
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: 9 months ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT01348087 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Change from baseline in the Aberrant Behavior Checklist - Community edition (ABC-C) total score and subscale scores [ Time Frame: 9 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: 9 months ] [ Designated as safety issue: No ]
  • Change from baseline in the Repetitive Behavior Symptom - Research version (RBS) total score and subscale score [ Time Frame: 9 months ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

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.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fragile X Syndrome
Drug: AFQ056
Experimental: AFQ056 100 mg (Bid)
Intervention: Drug: AFQ056
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
200
December 2015
December 2015   (final data collection date for primary outcome measure)

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

Both
18 Years to 46 Years
No
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals
United States,   Australia,   Brazil,   Canada,   Denmark,   France,   Germany,   Italy,   Netherlands,   Spain,   Switzerland,   United Kingdom
 
NCT01348087
CAFQ056B2279, 2011-001952-12
Not Provided
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP