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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01348087
• Date of registration: 03/05/2011
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
• Scientific title: An Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
• Date of first enrolment: August 2011
• Target sample size: 148
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01348087
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

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

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Drug: AFQ056

Primary Outcome(s)

Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs). [Time Frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial]

Secondary ID(s)

2011-001952-12

CAFQ056B2279

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/05/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01348087