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

Qilu Pharmaceutical

Status and phase

Begins enrollment in 2 months

Phase 4

Conditions

Transthyretin Amyloid Polyneuropathy

Treatments

Drug: Tafamidis Meglumine Soft Capsules

Study type

Interventional

Funder types

Industry

Identifiers

NCT06940336

QLG1046-401

Details and patient eligibility

About

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

Enrollment

15 estimated patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

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.