Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome
Condition: Fragile X Syndrome
MeSH terms: Fragile X Syndrome; Syndrome
Intervention: AFQ056
Intervention description: The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths, identical in appearance, will be used.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 148.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Fragile X Syndrome; Martin-Bell Syndrome; Genetic Diseases; X-Linked; Escalante's syndrome

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).
    

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