Clinical Trial: Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Open-label Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-type Transthyretin (Ttr) Amyloid Cardiomyopathy

Brief 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 conco

Detailed Summary:
Sponsor: Pfizer

Current Primary Outcome: To obtain additional, long-term, open-label safety and efficacy data for tafamidis in patients with transthyretin amyloid cardiomyopathy (TTR-CM). [ Time Frame: 10 years ]

Original Primary Outcome: To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) [ Time Frame: 3 years ]

Current Secondary Outcome: To continue to provide the investigational product tafamidis to patients with TTR-CM who completed Protocol Fx1B-201 [ Time Frame: 10 years ]

Original Secondary Outcome: To continue to provide the investigational product Fx-1006A to patients with ATTR-CM who completed Protocol Fx1B-201 [ Time Frame: 3 years ]

Information By: Pfizer

Dates:
Date Received: July 6, 2009
Date Started: September 22, 2009
Date Completion: December 31, 2021
Last Updated: April 6, 2017
Last Verified: April 2017