drugset / Trial / NCT04152200
A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).
Timeline
- Start
- 2020-01-21
- Primary completion
- 2021-05-18
- Completion
- 2025-06-23
Outcome
Met primary endpoint
release “new positive results from the six-month primary analysis period of the ILLUMINATE-C Phase 3 open-label study” alnylam.com ↗
release “in Cohorts A and B, respectively, treatment with lumasiran led to 33 percent and 42 percent mean reductions in POx from baseline to Month 6” alnylam.com ↗
release “The label expansion for OXLUMO is based on positive six-month results from ILLUMINATE-C, where treatment with lumasiran resulted in a substantial reduction in POx” alnylam.com ↗
Publications
- Sankar A, Y S RK, Singh A, Roy R, Shukla R, Verma B. Next-generation therapeutics for rare genetic disorders. Mutagenesis. 2024 Apr 24;39(3):157-171. doi: 10.1093/mutage/geae002.
- Michael M, Groothoff JW, Shasha-Lavsky H, Lieske JC, Frishberg Y, Simkova E, Sellier-Leclerc AL, Devresse A, Guebre-Egziabher F, Bakkaloglu SA, Mourani C, Saqan R, Singer R, Willey R, Habtemariam B, Gansner JM, Bhan I, McGregor T, Magen D. Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. Am J Kidney Dis. 2023 Feb;81(2):145-155.e1. doi: 10.1053/j.ajkd.2022.05.012. Epub 2022 Jul 14.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Lumasiran | Small interfering RNA (siRNA) | — | Subcutaneous |