A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
Summary
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
Timeline
- Start
- 2018-11-27
- Primary completion
- 2019-11-05
- Completion
- 2024-01-12
Outcome
Met primary endpoint
registry analysis (superiority test); Placebo/Lumasiran vs Lumasiran/Lumasiran; p <0.0001; Difference in Least Squares (LS) Mean -53.546 (95% CI -62.314 to -44.778); MMRM; note: P=1.685E-14 NCT03681184 ↗
release “At six months, lumasiran met the primary endpoint in patients with PH1 (p less than 0.0001)” alnylam.com ↗
release “Topline data from the pivotal ILLUMINATE-A Phase 3 study show that lumasiran met its primary efficacy endpoint and all tested secondary endpoints.” alnylam.com ↗
release “In the ILLUMINATE-A study (N=39), lumasiran met the primary efficacy endpoint of 24-hour urinary oxalate reduction from Month 3 to Month 6” alnylam.com ↗
release “ILLUMINATE-A study conducted in adults and children six years or older, lumasiran achieved the primary endpoint” alnylam.com ↗
release “clinically significant (53.5 percent) reduction in 24-hour urinary oxalate excretion from baseline to month 6 – the primary endpoint” alnylam.com ↗
Publications
- Frishberg Y, Saland JM, Lieske JC, Du W, Coenen M, Hogan J, Sellier-Leclerc AL, Groothoff JW, Kaspar C, Gansner JM, Hulton SA; ILLUMINATE-A study investigators. Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1. Clin J Am Soc Nephrol. 2026 Mar 1;21(3):377-388. doi: 10.2215/CJN.0000000916. Epub 2025 Dec 4.
- Garrelfs SF, Frishberg Y, Hulton SA, Koren MJ, O'Riordan WD, Cochat P, Deschenes G, Shasha-Lavsky H, Saland JM, Van't Hoff WG, Fuster DG, Magen D, Moochhala SH, Schalk G, Simkova E, Groothoff JW, Sas DJ, Meliambro KA, Lu J, Sweetser MT, Garg PP, Vaishnaw AK, Gansner JM, McGregor TL, Lieske JC; ILLUMINATE-A Collaborators. Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1. N Engl J Med. 2021 Apr 1;384(13):1216-1226. doi: 10.1056/NEJMoa2021712.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Lumasiran | Small interfering RNA (siRNA) | 3 mg/kg | Subcutaneous |