drugset / Trial / NCT05023889
Spectrum of Peripheral and Autonomic Neuropathies in Patients With aTTRwt Amyloidosis and Response to Patisiran Therapy
NaSingle-groupOpen-labelTreatment
Summary
To evaluate the efficacy and safety of patisiran in patients with wtATTR amyloidosis and symptomatic polyneuropathy by evaluating the effect on neurologic impairment and quality of life.
Timeline
- Start
- 2022-08-03
- Primary completion
- 2026-04-01
- Completion
- 2026-04-13
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Patisiran | Small interfering RNA (siRNA) | 0.3 mg/kg | Intravenous |