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Report for Clinical Trial NCT00925002

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

OPEN-LABEL SAFETY AND EFFICACY EVALUATION OF FX-1006A IN SUBJECTS WITH TRANSTHYRETIN (TTR) AMYLOIDOSIS

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for the treatment of ATTR-PN in their respective country and access to prescription tafamidis, subjects may have been withdrawn from the study.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.

NCT00925002 ATTR-PN
MeSH:Amyloidosis
HPO:Amyloidosis

1 Interventions

Name: Tafamidis

Description: 20 mg oral Fx-1006A daily
Type: Drug
Group Labels: 1

Open-Label


Primary Outcomes

Measure: Percentage of patients with a change from baseline in Neuropathy Impairment Score (NIS)

Time: Baseline up to 10 years

Measure: Percentage of patients with a change from baseline in Total Quality of Life (TQOL) score

Time: Baseline up to 10 years

Measure: Number or Percentage of patients with a change from baseline in Karnofsky Performance Scale Index

Time: Baseline up to 10 years

Measure: Percentage of patients with a change in subject ambulation as measured by modified Polyneuropathy Disability (mPND) score

Time: Baseline up to 10 years

Secondary Outcomes

Measure: Incidence of treatment emergent adverse events from baseline through 10 years

Time: Baseline up to 10 years

Measure: Number or percentage of patients with change from baseline in Clinical Laboratory parameters

Time: Baseline up to 10 years

Measure: Number of patients with change in ECG parameters

Time: Baseline up to 10 years

Measure: Number or percentage of patients with change from baseline in Vital sign measurements

Time: Baseline up to 10 years

Measure: Descriptive summary of physical examination findings for patients through 10 years

Time: Baseline up to 10 years

Measure: Descriptive summary of concomitant medication use for patients through 10 years

Time: Baseline up to 10 years

Purpose: Treatment


There is one SNP

SNPs


1 V30M

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. --- Val30Met ---

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. --- Val30Met --- --- Val30Met ---



HPO Nodes


HPO: