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

Launched by QILU PHARMACEUTICAL CO., LTD. · Apr 15, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medication called Tafamidis Meglumine Soft Capsules to see how well it works and how safe it is for adults with a condition known as Transthyretin Amyloid Polyneuropathy (ATTR-PN). This condition occurs when certain proteins build up in the body and damage the nerves, leading to symptoms like pain, numbness, and weakness. The trial is looking for participants aged 18 to 80 who have been diagnosed with ATTR-PN and have certain genetic markers associated with the disease. Participants must also have a specific level of disability that indicates a moderate stage of the disease.

If you join this trial, you'll be taking the study medication and will be closely monitored by the research team to track your health and any side effects. It's important to know that you cannot have used certain other medications for amyloidosis in the months leading up to the trial, and you should not have other conditions that could cause similar symptoms. This study is not yet recruiting participants, but if you're interested, you can keep an eye out for updates on when it will start.

Gender

ALL

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.