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Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis

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ClinicalTrials.gov Identifier: NCT00925002
Recruitment Status : Completed
First Posted : June 19, 2009
Last Update Posted : January 12, 2021
Sponsor:
Information provided by (Responsible Party):
Pfizer

Tracking Information
First Submitted Date  ICMJE June 17, 2009
First Posted Date  ICMJE June 19, 2009
Last Update Posted Date January 12, 2021
Actual Study Start Date  ICMJE August 5, 2009
Actual Primary Completion Date July 8, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 24, 2020)
  • Percentage of patients with a change from baseline in Neuropathy Impairment Score (NIS) [ Time Frame: Baseline up to 10 years ]
  • Percentage of patients with a change from baseline in Total Quality of Life (TQOL) score [ Time Frame: Baseline up to 10 years ]
  • Number or Percentage of patients with a change from baseline in Karnofsky Performance Scale Index [ Time Frame: Baseline up to 10 years ]
  • Percentage of patients with a change in subject ambulation as measured by modified Polyneuropathy Disability (mPND) score [ Time Frame: Baseline up to 10 years ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 17, 2009)
To obtain additional, long-term, open-label safety and efficacy data for Fx-1006A in patients with transthyretin (TTR) amyloidosis (ATTR) [ Time Frame: 3 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2020)
  • Incidence of treatment emergent adverse events from baseline through 10 years [ Time Frame: Baseline up to 10 years ]
  • Number or percentage of patients with change from baseline in Clinical Laboratory parameters [ Time Frame: Baseline up to 10 years ]
  • Number of patients with change in ECG parameters [ Time Frame: Baseline up to 10 years ]
  • Number or percentage of patients with change from baseline in Vital sign measurements [ Time Frame: Baseline up to 10 years ]
  • Descriptive summary of physical examination findings for patients through 10 years [ Time Frame: Baseline up to 10 years ]
  • Descriptive summary of concomitant medication use for patients through 10 years [ Time Frame: Baseline up to 10 years ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 17, 2009)
To continue to provide the investigational product Fx-1006A until its market availability to patients with ATTR who have completed Protocol Fx-006 or Protocol Fx1A-201 [ Time Frame: 3 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety And Efficacy Evaluation Of Fx-1006A In Subjects With Transthyretin Amyloidosis
Official Title  ICMJE OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS
Brief Summary This is a Phase 3, open-label study designed to obtain additional long-term safety and efficacy data for oral tafamidis (20 mg soft gelatin capsule) administered once daily (QD). In addition, this study continued to provide tafamidis to Val30Met subjects who had completed Protocol Fx-006 (a 1-year, open-label extension study to Protocol Fx-005 which was a randomized, double-blind, placebo-controlled, 18-month study to evaluate the safety and efficacy of tafamidis) or non-Val30Met subjects who had completed Protocol Fx1A-201 (a Phase 2, open-label study to evaluate TTR stabilization, safety, and tolerability of tafamidis) for up to 10 years or until subjects had access to tafamidis for ATTR-PN via prescription. Upon regulatory approval for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study. Such subjects were considered study completers.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE ATTR-PN
Intervention  ICMJE Drug: Tafamidis
20 mg oral Fx-1006A daily
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 28, 2017)
93
Original Estimated Enrollment  ICMJE
 (submitted: June 17, 2009)
100
Actual Study Completion Date  ICMJE July 8, 2020
Actual Primary Completion Date July 8, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion criteria:

  • Subject had successfully completed either Protocol Fx-006 or Fx-1A-201.
  • Male or female subjects with ATTR-PN who had not undergone liver or heart transplantation at time of enrollment.
  • If female, subject was post-menopausal, surgically sterilized, or willing to use an acceptable method of birth control

Key Exclusion criteria:

  • Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs (NSAIDs)
  • Pregnant or breast feeding female subjects.
  • Clinically significant medical condition that, in the opinion of the investigator, would place the subject at an increased risk to participate in the study.
  • An alanine aminotransferase (ALT) and aspartate aminotransferase (AST) value >3 × upper limit of normal (ULN) that, in the medical judgment of the investigator, was due to reduced liver function or active liver disease.
  • Sexually active males with partners of childbearing potential not using highly effective contraception or not agreeing to continue highly effective contraception for at least 3 months after last dose of study drug.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Brazil,   France,   Germany,   Italy,   Portugal,   Sweden,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00925002
Other Study ID Numbers  ICMJE FX1A-303
B3461023 ( Other Identifier: Alias Study Number )
2009-011535-12 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date January 2021

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