Clinical Trials Logo

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


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
See also
  Status Clinical Trial Phase
Recruiting NCT05418049 - Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge Phase 2
Enrolling by invitation NCT01364818 - Brain Connectivity in Neurodevelopmental Disorders in Response to Treatment N/A
Completed NCT00965432 - A Single-Dose Study in Normal Volunteers to Assess the Safety, Tolerability and Pharmacokinetics of STX107 Phase 1
Completed NCT01120626 - Randomized Controlled Study of Donepezil in Fragile X Syndrome Phase 2
Completed NCT01204151 - Teaching Math Skills to Individuals With Fragile X Syndrome N/A
Active, not recruiting NCT00334971 - Aromatase Activity and Ovarian Growth Factors in African-American Versus Caucasian Women N/A
Enrolling by invitation NCT06139172 - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities N/A
Recruiting NCT04977986 - Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome Phase 3
Active, not recruiting NCT04698551 - NIPD on cffDNA for Triplet Repeat Diseases
Completed NCT03722290 - Metformin in Children and Adults With Fragile X Syndrome Phase 2
Completed NCT05030129 - Single Blind Study of Ergoloid Mesylates, 5-HTP and the Combination in Adult Males With Fragile X Syndrome Phase 2
Recruiting NCT05957549 - Tracking Early Emergence of Sound Perception Impairments in FXS With Multimodal fNIRS/EEG N/A
Recruiting NCT04141163 - Metformin in Patients With Fragile X Phase 1/Phase 2
Not yet recruiting NCT06081348 - Sertraline vs. Placebo in the Treatment of Anxiety in Children and AdoLescents With NeurodevelopMental Disorders Phase 2
Completed NCT00858689 - Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Enrolling by invitation NCT03802799 - Open Label Extension to Assess the Long-Term Safety and Tolerability of ZYN002 in Children and Adolescents With FXS Phase 2/Phase 3
Recruiting NCT05295277 - Validation of Optical Genome Mapping for the Identification of Constitutional Genomic Variants in a Postnatal Cohort
Enrolling by invitation NCT03836300 - Parent and Infant Inter(X)Action Intervention (PIXI) N/A
Completed NCT01725152 - Ganaxolone Treatment in Children With Fragile X Syndrome Phase 2