To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-19

Study Completion Date

2028-02-29

Brief Summary

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The purpose of this study is to evaluate the efficacy and safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients with Transthyretin Amyloid Polyneuropathy

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Detailed Description

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Conditions

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Transthyretin Amyloid Polyneuropathy

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 Meglumine Soft Capsules

Group Type EXPERIMENTAL

Tafamidis Meglumine Soft Capsules

Intervention Type DRUG

20 mg orally once daily for 72 weeks

Interventions

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Tafamidis Meglumine Soft Capsules

20 mg orally once daily for 72 weeks

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male or female participants between the ages of 18 and 80 years.
* Participant has amyloid deposits confirmed by biopsy (Biopsy must have been performed within 5 years prior to enrollment).
* Participant must have a TTR mutation that is associated with ATTR-PN (TTR mutation test must have been performed within 5 years prior to enrollment).
* Participant has peripheral neuropathy at screening.
* Participant has a Karnofsky Performance Status Score ≥50.
* Stages of disease according to symptom severity-stage 1.

Exclusion Criteria

* Participant has other causes of amyloidosis, such as light chain amyloidosis, AA amyloidosis.
* Participant has used tafamidis within 2 months prior to enrollment.
* Participant has used diflunisal, patisiran, inotersen, or other agents for familial amyloidosis within 30 days prior to enrollment or plan to use them during the study period.
* Participant has used non-protocol NSAIDs more than 4 times within 30 days prior to enrollment or plan to use them more than 4 times per month during the study period.
* Participant has used doxycycline,tauroursodeoxycholate within 14 days prior to enrollment or plan to use them during the study period.
* Participant has sensory motor neuropathy caused by other causes, such as chronic inflammatory demyelinating polyradiculopathy, chronic idiopathic axonal neuropathy, diabetic neuropathy, chronic alcoholic neuropathy, paraneoplastic neuropathy,Guillain-Barre syndrome, vitamin B12 deficiency.
* Participant has received liver or any other organ except cornea transplantation.

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No