Clinical trial NCT01348087: Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Official title: An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Fragile X Syndrome
Keywords: Fragile X Syndrome
Martin-Bell Syndrome
Genetic Diseases
X-Linked
Escalante's syndrome
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2011-05-05
Enrollment: 148
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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 (NCT01253629).study and patients who have participated in the previous proof-of-concept study CAFQ056A2204 (NCT00718341).

Detailed description: A 148 patients were enrolled into this treatment extension trial conducted for at least 3 years. This extension trial was terminated after the decision to terminate the AFQ056 development program. The decision was based on the results of two randomized, double blind, placebo controlled phase IIb trials in adult and adolescent FXS patients (CAFQ056A2212 and CAFQ056B2214respectivly), both of which failed to demonstrate efficacy in the FXS population. As this extension trial was terminated, only the primary objective and safety are represented.

Eligibility criteria: Inclusion criteria Group 1 patients

* Had to have completed the CAFQ056A2212 study or another study of AFQ056 which included adult FXS patients within one week of enrollment into the open-label study.
* Females of child-bearing potential had to follow protocol requirements with respect to contraception.
* Have a caregiver or caregivers who spent, on average, at least six hours per day with the patient, who were willing and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.

Group 2:

* Had to have:
* Completed Study CAFQ056A2204.
* Completed Study CAFQ056A2212 or another study of AFQ056 which included adult patients with FXS but enrollment into the current study was delayed for more than a week.
* Discontinued prematurely from Study CAFQ056A2212 or another study of AFQ056 which included adult patients with FXS due to intolerability of the dosage in the patient's assigned treatment group.
* Females of child-bearing potential had to follow protocol requirements with respect to contraception.
* Have a caregiver or caregivers who spent, on average, at least six hours per day with the patient, who were 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.)
* Using (or used within 6 weeks before baseline) concomitant medications that are potent inhibitors or inducers of CYP3A4
* Using glutamatergic agents (riluzole, memantine, etc.) or lithium, digoxin, or warfarin within 6 weeks of baseline Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs).
timeFrame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Sacramento
country: United States
facility: Novartis Investigative Site
state: California
city: Decatur
country: United States
facility: Novartis Investigative Site
state: Georgia
city: Chicago
country: United States
facility: Novartis Investigative Site
state: Illinois
city: Indianapolis
country: United States
facility: Novartis Investigative Site
state: Indiana
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Omaha
country: United States
facility: Novartis Investigative Site
state: Nebraska
city: Staten Island
country: United States
facility: Novartis Investigative Site
state: New York
city: Media
country: United States
facility: Novartis Investigative Site
state: Pennsylvania
city: Nashville
country: United States
facility: Novartis Investigative Site
state: Tennessee
city: Ryde
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Waratah
country: Australia
facility: Novartis Investigative Site
state: New South Wales
city: Caulfield
country: Australia
facility: Novartis Investigative Site
state: Victoria
city: Brampton
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Sherbrooke
country: Canada
facility: Novartis Investigative Site
state: Quebec
city: Glostrup
country: Denmark
facility: Novartis Investigative Site
city: Bron Cedex
country: France
facility: Novartis Investigative Site
city: Paris
country: France
facility: Novartis Investigative Site
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Mainz
country: Germany
facility: Novartis Investigative Site
city: Tübingen
country: Germany
facility: Novartis Investigative Site
city: Würzburg
country: Germany
facility: Novartis Investigative Site
city: Genova
country: Italy
facility: Novartis Investigative Site
state: GE
city: Malaga
country: Spain
facility: Novartis Investigative Site
state: Andalucia
city: Sant Cugat
country: Spain
facility: Novartis Investigative Site
state: Catalunya
city: Lausanne
country: Switzerland
facility: Novartis Investigative Site
city: Zurich
country: Switzerland
facility: Novartis Investigative Site
city: Edinburgh
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: