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The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

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ClinicalTrials.gov Identifier: NCT04828993
Recruitment Status : Recruiting
First Posted : April 2, 2021
Last Update Posted : September 29, 2021
Sponsor:
Information provided by (Responsible Party):
Pfizer

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 48 weeks.


Condition or disease Intervention/treatment Phase
Transthyretin Amyloid Polyneuropathy (ATTR-PN) Drug: tafamidis meglumine Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 28, 2021
Estimated Primary Completion Date : March 9, 2023
Estimated Study Completion Date : March 9, 2023


Arm Intervention/treatment
Experimental: Chinese participants treated with Tafamidis Meglumine Drug: tafamidis meglumine
Tafamidis meglumine 20 mg, once daily, oral administration, for 48 weeks.
Other Name: Vyndaqel




Primary Outcome Measures :
  1. change from baseline in Neuropathy Impairment Score-lower limb (NIS-LL) at week 48 [ Time Frame: Week 48 ]
    The scale score ranges from 0-80, the higher score indicates the worse outcome.


Secondary Outcome Measures :
  1. The incidence and severity of TEAE during the whole study [ Time Frame: baseline through week 52 ]
  2. change from baseline in Norfolk QOL - Diabetic Neuropathy (Norfolk QOL-DN) at week 24 and week 48 [ Time Frame: baseline, week 24, week 48 ]
  3. change from baseline in Modified Body Mass Index (mBMI) at week 24 and week 48 [ Time Frame: baseline, week 24, week 48 ]
    We use participant's weight, height , and albumin concentration to calculate mBMI.

  4. Proportion of patients achieving transthyretin (TTR) stabilization at baseline, Week 8, Week 12 and Week 24, Week 48 [ Time Frame: Baseline, Week 8, Week 12, Week 24, Week 48 ]
  5. TTR concentration at baseline, Week 8, Week 12, Week 24, and Week 48 [ Time Frame: baseline, Week 8, Week 12, Week 24, Week 48 ]
  6. The incidence of laboratory abnormalities during the whole study [ Time Frame: baseline through week 48 ]
  7. Incidence of ECG abnormalities during the whole study [ Time Frame: baseline through week 48 ]
  8. change from baseline in 36-item survey form (SF-36) at Week 24 and Week 48 [ Time Frame: baseline, week 24, week 48 ]
  9. change from baseline in EQ-5D-5L Index Score at week 24 and week 48 [ Time Frame: baseline, week 24, week 48 ]
  10. Incidence of vital sign abnormalities during the whole study [ Time Frame: baseline through week 48 ]
  11. Incidence of clinically significant changes on Echocardiography during the whole study [ Time Frame: baseline through week 48 ]


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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT04828993


Contacts
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Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
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China, Beijing
Peking University First Hospital Recruiting
Beijing, Beijing, China, 100034
Peking University Third Hospital Recruiting
Beijing, Beijing, China, 100191
China, Fujian
The First Affiliated Hospital of Fujian Medical University Recruiting
Fuzhou, Fujian, China, 350005
China, Guangdong
NanFang Hospital of Southern Medical University Recruiting
Guangzhou, Guangdong, China, 510515
China, Shanghai
Huashan Hospital Fudan University Recruiting
Shanghai, Shanghai, China, 200040
China
Xuanwu Hospital Capital Medical University Recruiting
Beijing, China, 100053
Tiantan Hospital Capital Medical University Recruiting
Beijing, China, 100070
Peking union hospital of Chinese academy of medical sciences Recruiting
Beijing, China, 100730
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT04828993    
Other Study ID Numbers: B3461078
First Posted: April 2, 2021    Key Record Dates
Last Update Posted: September 29, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
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

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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
Keywords provided by Pfizer:
tafamidis meglumine
Additional relevant MeSH terms:
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Polyneuropathies
Amyloid Neuropathies
Amyloidosis
Proteostasis Deficiencies
Metabolic Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Nervous System Diseases