Tafamidis meglumine soft capsules for adults with transthyretin amyloid polyneuropathy

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

This trial tests whether tafamidis meglumine soft capsules can help adults with transthyretin amyloid polyneuropathy.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 15 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Qilu Pharmaceutical Co., Ltd. (Industry-sponsored)
• Locations: 1 site (Jinan)
• Trial ID: NCT06940336 on ClinicalTrials.gov

What this trial studies

This is a phase 4 interventional study giving tafamidis meglumine soft capsules to adults with biopsy-confirmed transthyretin amyloid deposits and a disease-associated TTR mutation who have stage 1 peripheral neuropathy. Eligible participants are 18–80 years old with a Karnofsky Performance Status ≥50 and no other causes of amyloidosis. The protocol excludes recent use of tafamidis or other ATTR-specific therapies and limits NSAID exposure. Participants will be followed for measures of efficacy and safety per the study protocol at the enrolling site.

Who should consider this trial

Good fit: Adults 18–80 with biopsy-confirmed transthyretin amyloid deposits, a disease-associated TTR mutation, stage 1 peripheral neuropathy, and Karnofsky score ≥50.

Not a fit: Patients with other forms of amyloidosis, recent tafamidis or other ATTR-specific therapy use, or more advanced disease stages are unlikely to benefit from enrollment.

Why it matters

Potential benefit: If successful, this could slow neuropathy progression and improve quality of life for adults with ATTR-PN.

How similar studies have performed: Tafamidis has been studied and used for transthyretin amyloidosis previously, with prior trials showing benefit in slowing disease progression, so this study builds on established therapy.

Eligibility criteria

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.

Where this trial is running

Jinan

• Qilu Hospital of Shandong University — Jinan, China (Recruiting)

Study contacts

• Study coordinator: chuanzhu yan, Doctor

Conditions Transthyretin Amyloid Polyneuropathy