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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00935012
First received: July 6, 2009
Last updated: April 11, 2014
Last verified: April 2014
  Purpose

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.


Condition Intervention Phase
TTR-CM
Drug: tafamidis
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open-Label Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy

Resource links provided by NLM:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • To continue to provide the investigational product tafamidis to patients with TTR-CM who completed Protocol Fx1B-201 [ Time Frame: 10 years ] [ Designated as safety issue: Yes ]

Enrollment: 31
Study Start Date: September 2009
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open-Label Drug: tafamidis
Once daily 20 mg oral tafamidis (soft gelatin capsule)

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00935012

Locations
United States, Georgia
Pfizer Investigational Site
Atlanta, Georgia, United States, 30322-4510
United States, Illinois
Pfizer Investigational Site
Chicago, Illinois, United States, 60637
United States, Maryland
Pfizer Investigational Site
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Pfizer Investigational Site
Boston, Massachusetts, United States, 021153904
United States, New York
Pfizer Investigational Site
New York, New York, United States, 10032
Pfizer Investigational Site
New York, New York, United States, 10034-1159
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00935012     History of Changes
Other Study ID Numbers: FX1B-303, B3461026
Study First Received: July 6, 2009
Last Updated: April 11, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Amyloidosis
Cardiomyopathies
Proteostasis Deficiencies
Metabolic Diseases
Heart Diseases
Cardiovascular Diseases

ClinicalTrials.gov processed this record on April 15, 2014