ClinicalTrials.gov record NCT06940336

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Single Arm, Multicenter, Open-label,Phase IV Clinical Study to Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients withTransthyretin Amyloid Polyneuropathy

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 4A study of a treatment that is already approved, to learn more about its long-term effects, safety or use in everyday practice.
Age18 years old to 80 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This single-arm, open-label Phase IV study gives tafamidis meglumine soft capsules 20 mg orally once daily for 72 weeks to adults with transthyretin amyloid polyneuropathy. The main measure is change in Neuropathy Impairment Score–Lower Limb (NIS-LL) at Week 72. Secondary measures include interim NIS-LL assessments and changes in modified body mass index (mBMI) at several time points.

What it is studying. Whether tafamidis meglumine soft capsules (20 mg once daily) change neuropathy impairment scores and are safe over 72 weeks in adults with transthyretin amyloid polyneuropathy.

What the researchers hope to learn. To evaluate the efficacy and safety of tafamidis meglumine in adults with transthyretin amyloid polyneuropathy. The primary scientific goal is to measure change from baseline in Neuropathy Impairment Score–Lower Limb (NIS-LL) at Week 72. Secondary goals include NIS-LL at Weeks 24 and 48 and changes in modified body mass index (mBMI) at multiple time points up to Week 72.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Tafamidis Meglumine Soft Capsules

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants take tafamidis meglumine soft capsules 20 mg by mouth once daily for 72 weeks. They return for scheduled visits to have their neuropathy measured (Neuropathy Impairment Score–Lower Limb at baseline, Week 24, Week 48, and Week 72) and nutrition status measured (modified BMI at baseline and at multiple visits up to Week 72). Participants must not have used disallowed medicines before enrollment as listed and must not plan to use those medicines during the study. Follow-up consists of the listed treatment period and the scheduled assessments above.

How the study is structured. Interventional, open-label study with a single treatment group. All participants receive tafamidis meglumine 20 mg orally once daily for 72 weeks. No placebo or comparison group. Neuropathy scores and body-mass measures are recorded at baseline and at scheduled visits through Week 72.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on ATTRv amyloidosis

Clinical trials106 trials listed for ATTRv amyloidosisEvery trial the registry lists for ATTRv amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in ATTRv amyloidosis researchLatest monthly update: September 2026. Includes: Gene-silencing drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 19, 2025
Primary completion
January 2028 (estimated)
Study completion
February 2028 (estimated)
Target enrollment
15 participants (estimated)
Last updated on registry
Jul 30, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Qilu Pharmaceutical Co., Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

biopsy
Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
enrollment
The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
Karnofsky Performance Status
A scale from 100 (no complaints) down to 0 that describes how well a person can carry out daily activities. Studies often require a minimum score.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

← Back to clinical trials