To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
Phase 4
Not yet recruiting
- Conditions
- Transthyretin Amyloid Polyneuropathy
- Interventions
- Registration Number
- NCT06940336
- Lead Sponsor
- Qilu Pharmaceutical Co., Ltd.
- 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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 15
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Tafamidis Meglumine Soft Capsules Tafamidis Meglumine Soft Capsules -
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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