Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
Condition: ATTR-CM; TTR-CM
MeSH terms: Cardiomyopathies
Intervention: tafamidis
Intervention description: Once daily 20 mg oral tafamidis (soft gelatin capsule)
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 31.0
Sex: All
Minimum age: 18 Years
Maximum age: 75 Years
Healthy volunteers: No
Keywords: TTR, ATTR, cardiomyopathy, amyloidosis, tafamidis

Summary:

      Open-label Safety and Efficacy Evaluation of Fx-1006a in Patients with V122i Or Wild-type
      Transthyretin (ttr) Amyloid Cardiomyopathy.

      Patients who successfully complete Fx1B-201 will report to the clinical unit on Day 0 to sign
      the informed consent form and determine eligibility for Protocol Fx1B-303. In addition, on
      Day 0, patients will have their entrance criteria reviewed, and medical histories and
      demographic characteristics obtained.

      The physical examination (including weight and vital signs) and the relevant end of study
      clinical laboratory tests (alkaline phosphatase, alanine aminotransferase, aspartate
      aminotransferase, blood urea nitrogen, gamma glutamyl transferase, creatinine, total
      bilirubin, international normalized ratio, troponin I, troponin T, and amino-terminal B-type
      natriuretic peptide) from Protocol Fx1B-201 will be used for Protocol Fx1B-303. If more than
      30 days has elapsed between the final study visit of Protocol Fx1B-201 and Day 0 of Protocol
      Fx1B-303, an abbreviated physical examination (including weight and vital signs) and clinical
      laboratory assessments must be performed on Day 0.

      Eligible patients will begin once-daily dosing with 20 mg Fx-1006A at home on Day 1 (i.e.,
      first dose) and will return to the clinical unit for study visits every 6 months.

      Adverse events (AEs) and concomitant medication use will be collected at each 6-month visit
      to the clinical unit. Blood draws for clinical safety laboratory tests and abbreviated
      physical examinations (including weight and vital signs) will also be performed at each
      6-month clinic visit. ECGs will be performed every 12 months on an annual basis. A telephone
      call will be made at 3-month intervals between clinic visits to assess safety and use of
      concomitant medications.

      For the evaluation of efficacy, the Patient Global Assessment, NYHA classification, KCCQ,
      6-minute walk test, and efficacy-related clinical laboratory tests (serum levels of troponin
      T, troponin I, and NT-pro-BNP) will be determined every 6 months. In addition,
      echocardiograms will be performed every 12 months on an annual basis.

      An end of study visit including all safety and efficacy assessments will occur upon patient
      completion of the study, premature withdrawal (for any reason), or in the event of program
      discontinuation by the Sponsor.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patient successfully completed Protocol Fx1B-201.

          -  If female; patient is post menopausal. If male, female partner is post-menopausal. If
             female is of child bearing potential, willing to use acceptable method of birth
             control up to 3 months after last dose (included female partners of male
             participants).

          -  Patient is willing to comply with protocol.

        Exclusion Criteria:

          -  Patient did not successfully complete Fx1B-201.

          -  Chronic use of NSAIDS.

          -  Patient has a clinically significant medication condition that increases risk of study
             participation.

          -  Patient has received heart or liver transplant.