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RecruitingNCT06940336Updated Jul 30, 2025

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

A Phase 4 interventional study of Tafamidis Meglumine Soft Capsules in Transthyretin Amyloid Polyneuropathy, sponsored by Qilu Pharmaceutical Co., Ltd.. Recruiting at 1 site in China. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2025-07-30.

Sponsored by Qilu Pharmaceutical Co., Ltd. · Phase 4, Interventional, and Treatment

From the registry’s dates

  • Started Jun 2025; still recruiting 1 year 3 months later.
Phase
Phase 4
Study type
Interventional
Enrollment
15
Allocation
Not applicable
Ages
18 Years to 80 Years
Sex
All
01

Study summary

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

02

Conditions studied

  • Transthyretin Amyloid Polyneuropathy
03

In context

Amyloid Neuropathies, Familial

122 studies on the registry are indexed under Amyloid Neuropathies, Familial; 71 are open to participants now.

This study's planned enrollment of 15 is below the median of 60 across 61 interventional studies indexed under Amyloid Neuropathies, Familial.

Browse Amyloid Neuropathies, Familial studies →

Lead sponsor

Qilu Pharmaceutical Co., Ltd. is the lead sponsor of 193 studies on the registry; 119 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 80 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
15 participants (estimated)

Study arms

  • Experimental
    Tafamidis Meglumine Soft Capsules

    Drug: Tafamidis Meglumine Soft Capsules

Interventions

  • DrugTafamidis Meglumine Soft Capsules

    20 mg orally once daily for 72 weeks

06

What researchers measure

Primary outcomes

  1. Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72

    Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72

    Time frame: Baseline to 72 Weeks

Secondary outcomes

  1. Change from Baseline NIS-LL at Week 24 and Week 48

    Change from Baseline NIS-LL at Week 24 and Week 48

    Time frame: Baseline to 24 and 48 Weeks

  2. Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72

    Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72

    Time frame: Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks

07

Study locations

1 of 1 sites recruiting
  • Qilu Hospital of Shandong University
    Jinan, China
    Recruiting
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 30, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06940336
Lead sponsor
Qilu Pharmaceutical Co., Ltd.
Responsible party
Sponsor
First posted
Apr 23, 2025
Start date
Jun 19, 2025
Primary completion
Jan 2028 (estimated)
Completion
Feb 2028 (estimated)
Last update
Jul 30, 2025

Study contacts

chuanzhu yan, Doctor
Contact
zzl2002@medmail.com
18560082288

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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