To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Tafamidis Meglumine Soft Capsules.
- Who it may be relevant to
- Registry conditions: Transthyretin Amyloid Polyneuropathy. Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
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
Overview
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
- Drug Tafamidis Meglumine Soft Capsules
20 mg orally once daily for 72 weeks
Primary outcome measures
- Change from Baseline Neuropathy Impairment Score-Lower Limb (NIS-LL) at Week 72 [Time frame: Baseline to 72 Weeks]
Secondary outcome measures (2)
- Change from Baseline NIS-LL at Week 24 and Week 48 [Time frame: 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 [Time frame: Baseline to 4, 8, 12, 24, 36, 48 and 72 Weeks]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Qilu Hospital of Shandong University — Jinan
Identifiers
NCT: NCT06940336 · QLG1046-401