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

Fragile X Syndrome Clinical Trial

Official title:

An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01348087
• Other study ID #: CAFQ056B2279
• Secondary ID: 2011-001952-12
• Status: Completed
• Phase: Phase 2
• First received: May 3, 2011
• Last updated: February 12, 2015
• Start date: August 2011
• Est. completion date: September 2014

Study information

• Verified date: February 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health CanadaDenmark: Danish Medicines AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: BfArMItaly: Ethics CommitteeUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyAustralia: National Health and Medical Research CouncilSwitzerland: Swissmedic
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 148
• Est. completion date: September 2014
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• AFQ056

Locations

Country	Name	City	State
Australia	Novartis Investigative Site	Caulfield	Victoria
Australia	Novartis Investigative Site	Ryde	New South Wales
Australia	Novartis Investigative Site	Waratah	New South Wales
Canada	Novartis Investigative Site	Brampton	Ontario
Canada	Novartis Investigative Site	Sherbrooke	Quebec
Denmark	Novartis Investigative Site	Glostrup
France	Novartis Investigative Site	Bron Cedex
France	Novartis Investigative Site	Paris
Germany	Novartis Investigative Site	Berlin
Germany	Novartis Investigative Site	Mainz
Germany	Novartis Investigative Site	Tübingen
Germany	Novartis Investigative Site	Würzburg
Italy	Novartis Investigative Site	Genova	GE
Spain	Novartis Investigative Site	Málaga	Andalucia
Spain	Novartis Investigative Site	Sant Cugat	Catalunya
Switzerland	Novartis Investigative Site	Lausanne
Switzerland	Novartis Investigative Site	Zurich
United Kingdom	Novartis Investigative Site	Edinburgh
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Decatur	Georgia
United States	Novartis Investigative Site	Indianapolis	Indiana
United States	Novartis Investigative Site	Media	Pennsylvania
United States	Novartis Investigative Site	Nashville	Tennessee
United States	Novartis Investigative Site	Omaha	Nebraska
United States	Novartis Investigative Site	Phoenix	Arizona
United States	Novartis Investigative Site	Sacramento	California
United States	Novartis Investigative Site	Staten Island	New York

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Denmark,  France,  Germany,  Italy,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		24 months	Yes
Secondary	Change from baseline in the Aberrant Behavior Checklist - Community edition (ABC-C) total score and subscale scores		24 months	No
Secondary	Rating of global improvement of symptoms in Fragile X patients using the Clinical Global Impression - Improvement (CGI-I) scale		24 months	No
Secondary	Change from baseline in the Repetitive Behavior Symptom - Research version (RBS) total score and subscale score		24 months	No