A Phase 3 Study of ARO-APOC3 / VSA001 / SAR449124 (Plozasiran) in Chinese Adults With Familial Chylomicronemia Syndrome
Summary
This is a randomized, double-blinded, placebo controlled, two periods phase 3 clinical study. The primary objective of the study was to evaluate the efficacy and safety of Plozasiran injection in Chinese adults with familial chylomicronemia syndrome (FCS). A total of 37 participants were enrolled in the study. The duration of the study randomized period was approximately 112 weeks, including a screening period of up to 8 weeks and a treatment period of up to 104 weeks. Participants who completed the randomized period will continue in a 1-year open-label extension period where all participants will receive Plozasiran.
Timeline
- Start
- 2023-07-10
- Primary completion
- 2024-11-26
- Completion
- 2026-01-09
Outcome
Met primary endpoint
registry analysis (superiority test); Plozasiran 25 mg vs Placebo; p = 0.0022; Median Difference (Final Values) -101.7 (95% CI -167.8 to -35.5) NCT05902598 ↗
release “NCT05902598) of investigational plozasiran in Chinese patients with familial chylomicronemia syndrome (FCS), which successfully met its primary efficacy endpoint” arrowheadpharma.com ↗
release “The trial successfully met its primary efficacy endpoint and all key secondary endpoints” visirna.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Plozasiran | Small interfering RNA (siRNA) | 25 mg | Subcutaneous |
| Subject | Plozasiran | Small interfering RNA (siRNA) | 50 mg | Subcutaneous |