The Effect Of Tafamidis Meglumine In Transthyretin Amyloid Polyneuropathy Patients

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-04-28

Study Completion Date

2023-02-12

Brief Summary

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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.

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).

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Detailed Description

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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 visits will be scheduled at Baseline (Day 1) and at Week 4, Week 8, Week 12, Week 24, Week 36, 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).

Conditions

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Transthyretin Amyloid Polyneuropathy (ATTR-PN)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Tafamidis treatment arm

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

Group Type EXPERIMENTAL

tafamidis meglumine

Intervention Type DRUG

Tafamidis meglumine 20 mg, once daily, oral administration, for 72 weeks (18 months).

Interventions

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tafamidis meglumine

Tafamidis meglumine 20 mg, once daily, oral administration, for 72 weeks (18 months).

Intervention Type DRUG

Other Intervention Names

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Vyndaqel

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No