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Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label) | Clinical Trials | Clareo Health

TERMINATEDPHASE2/PHASE3

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

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

NCT01433354•Novartis Pharmaceuticals

View on ClinicalTrials.gov

Summary

The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adolescent patients with FXS who have participated in the CAFQ056B2214 study, the PK study CAFQ056B2131, or another study of AFQ056 which included FXS patients below 18 years of age provided the patient is at least 12 years of age at the time of entry into the current study.

Eligibility

Age

12 - 18 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Group 1 patients:
•Must have completed study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age within one week of enrollment into the open-label study.
•Has a caregiver or caregivers who spend, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.
•Group 2 patients:
•Must meet one of the following conditions:
•Completed Study CAFQ056B2131
•Completed Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age but enrollment into the current study was delayed for more than a week.
•Discontinued prematurely from Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age due to intolerability of the dosage in the patient's assigned treatment group.
•Has a caregiver or caregivers who spend, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.

Exclusion Criteria

•Discontinuation from Study CAFQ056B2214 or CAFQ056B2131 or another study of AFQ056 which included FXS patients below 18 years of age due to safety reasons
•Female patients who are sexually active at any time during the study
•Any advanced, severe or unstable disease
•History and/or presence of schizophrenia, bipolar disease, psychosis, confusional states and/or repeated hallucinations as per DSM-IV criteria
•History of suicidal behavior or considered a high suicidal risk
•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.)
•History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether or not there is evidence of local recurrence or metastases
•Patients who are using (or used within 6 weeks before baseline) digoxin or warfarin
+2 more criteria

Locations (28)

Novartis Investigative Site

Sacramento, California, United States

Novartis Investigative Site

Decatur, Georgia, United States

Novartis Investigative Site

Chicago, Illinois, United States

Novartis Investigative Site

Boston, Massachusetts, United States

Novartis Investigative Site

Omaha, Nebraska, United States

Novartis Investigative Site

Staten Island, New York, United States

Novartis Investigative Site

Nashville, Tennessee, United States

Novartis Investigative Site

Westmead, New South Wales, Australia

Novartis Investigative Site

Parkville, Victoria, Australia

Novartis Investigative Site

Brussels, Belgium

Key Dates

Start Date

November 1, 2011

Primary Completion Date

September 1, 2014

Completion Date

September 1, 2014

Last Updated

March 24, 2016

Enrollment

119

ACTUAL participants

Conditions

Fragile X Syndrome

Interventions

AFQ056

DRUG

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

NCT05418049

Fragile X Syndrome

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NOT_YET_RECRUITINGPHASE2/PHASE3

A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants

NCT07439510

Fragile X Syndrome (FXS)

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NOT_YET_RECRUITINGPHASE2

Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 24, 2016Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Inclusion Criteria

Exclusion Criteria

Evaluating the Neurophysiologic and Clinical Effects of Single Dose Drug Challenge

A Study to Investigate the Effects and Safety of SPG601 for the Treatment of Fragile X Syndrome in Male Participants

Effect of CANnabidiol on Anxiety and GABAergic Function in Individuals with Fragile-X Syndrome

Study of SPG601 in Adult Men With Fragile X Syndrome

An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome

Pediatric Exploratory Research Study of EGCG Use and Safety (PERSEUS)