A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1

Summary

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).

Timeline

Start

2019-04-22

Primary completion

2020-06-29

Completion

2024-07-26

Publications

• Frishberg Y, Hayes W, Shasha-Lavsky H, Sas DJ, Michael M, Sellier-Leclerc AL, Hogan J, Willey R, Gansner JM, Magen D. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 30-month analysis of the phase 3 ILLUMINATE-B trial. Front Pediatr. 2024 Sep 16;12:1392644. doi: 10.3389/fped.2024.1392644. eCollection 2024.
• 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.
• Hayes W, Sas DJ, Magen D, Shasha-Lavsky H, Michael M, Sellier-Leclerc AL, Hogan J, Ngo T, Sweetser MT, Gansner JM, McGregor TL, Frishberg Y. Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. Pediatr Nephrol. 2023 Apr;38(4):1075-1086. doi: 10.1007/s00467-022-05684-1. Epub 2022 Aug 1.
• Sas DJ, Magen D, Hayes W, Shasha-Lavsky H, Michael M, Schulte I, Sellier-Leclerc AL, Lu J, Seddighzadeh A, Habtemariam B, McGregor TL, Fujita KP, Frishberg Y; ILLUMINATE-B Workgroup. Phase 3 trial of lumasiran for primary hyperoxaluria type 1: A new RNAi therapeutic in infants and young children. Genet Med. 2022 Mar;24(3):654-662. doi: 10.1016/j.gim.2021.10.024. Epub 2021 Dec 8.

Drugs

Indications