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: June 27, 2014
Last verified: June 2014

July 6, 2009
June 27, 2014
September 2009
December 2020   (final data collection date for primary outcome measure)
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 ]
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 ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00935012 on ClinicalTrials.gov Archive Site
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 ]
To continue to provide the investigational product Fx-1006A to patients with ATTR-CM who completed Protocol Fx1B-201 [ Time Frame: 3 Years ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy
Open-Label Safety And Efficacy Evaluation Of Fx-1006a In Patients With V122i Or Wild-Type Transthyretin (TTR) Amyloid Cardiomyopathy

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.

Not Provided
Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
TTR-CM
Drug: tafamidis
Once daily 20 mg oral tafamidis (soft gelatin capsule)
Experimental: Open-Label
Intervention: Drug: tafamidis
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
31
December 2020
December 2020   (final data collection date for primary outcome measure)

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.
Both
18 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00935012
FX1B-303, B3461026
Yes
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP