Transthyretin Amyloid Polyneuropathy
Conditions
Brief summary
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
DRUGTafamidis Meglumine Soft Capsules
20 mg orally once daily for 72 weeks
Sponsors
Qilu Pharmaceutical Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72 | Baseline to 72 Weeks | Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline NIS-LL at Week 24 and Week 48 | Baseline to 24 and 48 Weeks | Change from Baseline NIS-LL at Week 24 and Week 48 |
| 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 | Change from Baseline Modified Body Mass Index(mBMI) at Week 4, Week 8, Week 12,Week 24, Week 36,Week 48 and Week 72 |
Countries
China
Contacts
Primary Contactchuanzhu yan, Doctor
Outcome results
None listed