To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients 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

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

Study Overview

• Status: Recruiting
• Conditions: Transthyretin Amyloid Polyneuropathy
• Intervention / Treatment: Drug: Tafamidis Meglumine Soft Capsules

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: chuanzhu yan, Doctor; Phone Number: 18560082288; Email: zzl2002@medmail.com

Study Locations

• China
  • Jinan, China
    • Recruiting
    • Qilu Hospital of Shandong University
    • Contact: chuanzhu yan, Doctor; Phone Number: 18560082288; Email: zzl2002@medmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tafamidis Meglumine Soft Capsules	Drug: Tafamidis Meglumine Soft Capsules; 20 mg orally once daily for 72 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Baseline to 72 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline NIS-LL at Week 24 and Week 48	Change from Baseline NIS-LL at Week 24 and Week 48	Baseline to 24 and 48 Weeks
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	Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks

Collaborators and Investigators

• Sponsor: Qilu Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): June 19, 2025
• Primary Completion (Estimated): January 1, 2028
• Study Completion (Estimated): February 1, 2028

Study Registration Dates

• First Submitted: April 8, 2025
• First Submitted That Met QC Criteria: April 15, 2025
• First Posted (Actual): April 23, 2025

Study Record Updates

• Last Update Posted (Actual): July 30, 2025
• Last Update Submitted That Met QC Criteria: July 29, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Peripheral Nervous System Diseases; Proteostasis Deficiencies; Polyneuropathies; Amyloidosis; Amyloid Neuropathies
• Other Study ID Numbers: QLG1046-401

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No