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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00935012
Recruitment Status : Active, not recruiting
First Posted : July 8, 2009
Last Update Posted : December 4, 2017
Information provided by (Responsible Party):

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 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 or disease Intervention/treatment Phase
TTR-CM Drug: tafamidis Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Masking: None (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
Actual Study Start Date : September 2009
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arm Intervention/treatment
Experimental: Open-Label Drug: tafamidis
Once daily 20 mg oral tafamidis (soft gelatin capsule)

Primary Outcome Measures :
  1. 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 ]

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

Information from the National Library of Medicine

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Ages Eligible for Study:   40 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00935012

United States, Illinois
University of Chicago Medical Center
Chicago, Illinois, United States, 60637
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
United States, New York
Clinical Cardiovascular Research Lab for the Elderly (CCRLE) Columbia Universtiy
New York, New York, United States, 10034
Sponsors and Collaborators
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00935012     History of Changes
Other Study ID Numbers: FX1B-303
B3461026 ( Other Identifier: Alias Study Number )
First Posted: July 8, 2009    Key Record Dates
Last Update Posted: December 4, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Heart Diseases
Cardiovascular Diseases