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The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Primary Purpose

Transthyretin Amyloid Polyneuropathy (ATTR-PN)

Status
Completed
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
tafamidis meglumine
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Transthyretin Amyloid Polyneuropathy (ATTR-PN) focused on measuring tafamidis meglumine

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Male or female participants between the ages of 18 and 80 years.
  2. Participants have amyloid documented by biopsy
  3. Participants must have a TTR mutation that is associated with ATTR-PN.
  4. Participants have peripheral and/or autonomic neuropathy
  5. Stages of disease according to symptom severity-stage I.

Exclusion Criteria:

  1. Other acute or chronic medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality, in the judgment of the investigator, would make the participant inappropriate for entry into this study.
  2. Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs.

  3. Use of diflunisal, tauroursodeoxycholate, doxycycline, inotersen, patisiran or any other TTR stabilizing agent, or experimental interventions for familial amyloidosis within 30 days prior to the study entry and/or during study participation. Participants who are taking or who have previously taken tafamidis.

    Prior/Concurrent Clinical Study Experience:

  4. Previous administration with an investigational drug within 30 days or 5 half lives preceding the first dose of investigational product used in this study (whichever is longer).
  5. Participant has primary (light chain) or secondary amyloidosis.
  6. If female, participant is pregnant or breast feeding, or plans to be pregnant or breast feeding in the next 18 months.
  7. Participant has received prior liver or any other organ except cornea transplantation.
  8. Participant requires significant assistance with ambulation or is wheel chair bound.
  9. Participants with cardiomyopathy specific TTR mutations.
  10. Participant has other causes of sensorimotor neuropathy.

Sites / Locations

  • Peking University First Hospital
  • Peking University Third Hospital
  • The First Affiliated Hospital of Fujian Medical University
  • NanFang Hospital of Southern Medical University
  • Huashan Hospital Fudan University
  • Peking union hospital of Chinese academy of medical sciences
  • Xuanwu Hospital Capital Medical University
  • Tiantan Hospital Capital Medical University
  • Peking union hospital of Chinese academy of medical sciences

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Tafamidis treatment arm

Arm Description

Chinese patients diagnosed with ATTR-PN treated with tafamidis 20mg once daily oral adminiistration for 72 weeks (18 months).

Outcomes

Primary Outcome Measures

change from baseline in Neuropathy Impairment Score-lower limb (NIS-LL) at week 72
The scale score ranges from 0-80, the higher score indicates the worse outcome.

Secondary Outcome Measures

The incidence and severity of TEAE during the whole study
change from baseline in Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at week 24, week 48, and week 72
change from baseline in Modified Body Mass Index (mBMI) at week 24, week 48 and week 72
We use participant's weight, height , and albumin concentration to calculate mBMI.
Proportion of patients achieving transthyretin (TTR) stabilization at baseline, Week 8, Week 12 and Week 24, Week 48, week 72
TTR concentration at baseline, Week 8, Week 12, Week 24, Week 48 and week 72
The incidence of laboratory abnormalities during the whole study
Incidence of ECG abnormalities during the whole study
change from baseline in 36-item survey form (SF-36) at Week 24, Week 48 and week 72
change from baseline in EQ-5D-5L Index Score at week 24, week 48 and week 72
Incidence of vital sign abnormalities during the whole study
Incidence of clinically significant changes on Echocardiography during the whole study

Full Information

First Posted
March 17, 2021
Last Updated
April 25, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT04828993
Brief Title
The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients
Official Title
A SINGLE ARM, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY, SAFETY, TOLERABILITY, AND PHARMACODYNAMICS OF ORALLY ADMINISTERED TAFAMIDIS MEGLUMINE IN TRANSTHYRETIN AMYLOID POLYNEUROPATHY PARTICIPANTS IN CHINA
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
April 28, 2021 (Actual)
Primary Completion Date
February 12, 2023 (Actual)
Study Completion Date
February 12, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a single-arm, open-label, multicenter study designed to determine the effect of tafamidis meglumine on TTR stabilization as well as tafamidis meglumine safety, tolerability and efficacy in ATTR-PN patients in China. Approximately 10-15 participants are planned to be enrolled. All enrolled participants will receive oral tafamidis meglumine 20 mg soft capsules once daily for 72 weeks (18 months).
Detailed Description
This is a single-arm, open-label, multicenter study designed to evaluate the efficacy, safety, tolerability as well as pharmacodynamics of tafamidis meglumine in ATTR-PN participants in China. All enrolled participants will receive oral tafamidis meglumine 20 mg soft capsules once daily starting on Day 1. Clinical visitswill be scheduled at Baseline (Day 1) and at Week 4, Week 8, Week 12, Week 24, Week 48, Week 60 and Week 72. At Week 36 and Week 60 site visit, assessment of adverse events, safety related lab testings, concomitant medications and investigational product compliance will be scheduled. Every 6 weeks (do not exceed 7 weeks since last confirmation) telephone contacts will be made during visits in which no investigative site visits are scheduled for assessment of adverse events, concomitant medications and investigational product compliance (between Week 12 and 24, between Week 24 and 36, between Week 36 and 48, between Week 48 and 60, and between Week 60 and 72).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Transthyretin Amyloid Polyneuropathy (ATTR-PN)
Keywords
tafamidis meglumine

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Tafamidis treatment arm
Arm Type
Experimental
Arm Description
Chinese patients diagnosed with ATTR-PN treated with tafamidis 20mg once daily oral adminiistration for 72 weeks (18 months).
Intervention Type
Drug
Intervention Name(s)
tafamidis meglumine
Other Intervention Name(s)
Vyndaqel
Intervention Description
Tafamidis meglumine 20 mg, once daily, oral administration, for 72 weeks (18 months).
Primary Outcome Measure Information:
Title
change from baseline in Neuropathy Impairment Score-lower limb (NIS-LL) at week 72
Description
The scale score ranges from 0-80, the higher score indicates the worse outcome.
Time Frame
Week 72
Secondary Outcome Measure Information:
Title
The incidence and severity of TEAE during the whole study
Time Frame
baseline through week 76
Title
change from baseline in Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at week 24, week 48, and week 72
Time Frame
baseline, week 24, week 48, week 72
Title
change from baseline in Modified Body Mass Index (mBMI) at week 24, week 48 and week 72
Description
We use participant's weight, height , and albumin concentration to calculate mBMI.
Time Frame
baseline, week 24, week 48, week 72
Title
Proportion of patients achieving transthyretin (TTR) stabilization at baseline, Week 8, Week 12 and Week 24, Week 48, week 72
Time Frame
Baseline, Week 8, Week 12, Week 24, Week 48, week 72
Title
TTR concentration at baseline, Week 8, Week 12, Week 24, Week 48 and week 72
Time Frame
baseline, Week 8, Week 12, Week 24, Week 48, week 72
Title
The incidence of laboratory abnormalities during the whole study
Time Frame
baseline through week 72
Title
Incidence of ECG abnormalities during the whole study
Time Frame
baseline through week 72
Title
change from baseline in 36-item survey form (SF-36) at Week 24, Week 48 and week 72
Time Frame
baseline, week 24, week 48, week 72
Title
change from baseline in EQ-5D-5L Index Score at week 24, week 48 and week 72
Time Frame
baseline, week 24, week 48, week 72
Title
Incidence of vital sign abnormalities during the whole study
Time Frame
baseline through week 72
Title
Incidence of clinically significant changes on Echocardiography during the whole study
Time Frame
baseline through week 72

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female participants between the ages of 18 and 80 years. Participants have amyloid documented by biopsy Participants must have a TTR mutation that is associated with ATTR-PN. Participants have peripheral and/or autonomic neuropathy Stages of disease according to symptom severity-stage I. Exclusion Criteria: Other acute or chronic medical or psychiatric condition including recent or active suicidal ideation or behavior or laboratory abnormality, in the judgment of the investigator, would make the participant inappropriate for entry into this study. Chronic use of non-protocol approved non-steroidal anti-inflammatory drugs. Use of diflunisal, tauroursodeoxycholate, doxycycline, inotersen, patisiran or any other TTR stabilizing agent, or experimental interventions for familial amyloidosis within 30 days prior to the study entry and/or during study participation. Participants who are taking or who have previously taken tafamidis. Prior/Concurrent Clinical Study Experience: Previous administration with an investigational drug within 30 days or 5 half lives preceding the first dose of investigational product used in this study (whichever is longer). Participant has primary (light chain) or secondary amyloidosis. If female, participant is pregnant or breast feeding, or plans to be pregnant or breast feeding in the next 18 months. Participant has received prior liver or any other organ except cornea transplantation. Participant requires significant assistance with ambulation or is wheel chair bound. Participants with cardiomyopathy specific TTR mutations. Participant has other causes of sensorimotor neuropathy.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Peking University First Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100034
Country
China
Facility Name
Peking University Third Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100191
Country
China
Facility Name
The First Affiliated Hospital of Fujian Medical University
City
Fuzhou
State/Province
Fujian
ZIP/Postal Code
350005
Country
China
Facility Name
NanFang Hospital of Southern Medical University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510515
Country
China
Facility Name
Huashan Hospital Fudan University
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200040
Country
China
Facility Name
Peking union hospital of Chinese academy of medical sciences
City
Beijing
ZIP/Postal Code
100005
Country
China
Facility Name
Xuanwu Hospital Capital Medical University
City
Beijing
ZIP/Postal Code
100053
Country
China
Facility Name
Tiantan Hospital Capital Medical University
City
Beijing
ZIP/Postal Code
100070
Country
China
Facility Name
Peking union hospital of Chinese academy of medical sciences
City
Beijing
ZIP/Postal Code
100730
Country
China

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing 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.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=B3461078
Description
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The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

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