Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1

Lumasiran treatment for up to 60 months in ILLUMINATE-A was associated with sustained reductions in UOx excretion and plasma oxalate concentration, encouraging clinical outcomes including stable eGFR in a population that would be expected to show eGFR decline, reduced kidney stone event rates, improved medullary nephrocalcinosis, and indications of improved health-related quality of life.Clinical Trial registry name and registration number: ClinicalTrials.gov NCT03681184 .

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Journal
Clinical Journal of the American Society of Nephrology
Published
2025-12-04
DOI
https://doi.org/10.2215/cjn.0000000916
Primary Topic
Kidney Stones and Urolithiasis Treatments
Type
article
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article

Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1

Jaap W. Groothoff, W. J. Du, Cristin Kaspar, John M. Gansner et al.
Clinical Journal of the American Society of Nephrology
Kidney Stones and Urolithiasis Treatments
article

Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1

Jaap W. Groothoff, W. J. Du, Cristin Kaspar, John M. Gansner, Julien Hogan, John Charles Lieske, Anne‐Laure Sellier‐Leclerc, Jeffrey M. Saland, Yaacov Frishberg, Martin Coenen, Sally A. Hulton
article en

Abstract

Lumasiran treatment for up to 60 months in ILLUMINATE-A was associated with sustained reductions in UOx excretion and plasma oxalate concentration, encouraging clinical outcomes including stable eGFR in a population that would be expected to show eGFR decline, reduced kidney stone event rates, improved medullary nephrocalcinosis, and indications of improved health-related quality of life.Clinical Trial registry name and registration number: ClinicalTrials.gov NCT03681184 .

Clinical Journal of the American Society of NephrologyVol. 21(3)
Mayo Clinic (US), Alnylam Pharmaceuticals (United States) (US), Inserm (FR), University Hospital Bonn (DE), Shaare Zedek Medical Center (IL), Hôpital Femme Mère Enfant (FR), Emma Kinderziekenhuis (NL), Assistance Publique – Hôpitaux de Paris (FR), Birmingham Women's Hospital (GB), Birmingham Children's Hospital (GB), Hospices Civils de Lyon (FR), Hôpital Robert-Debré (FR), Icahn School of Medicine at Mount Sinai (US)
Openalex Percentile: Top 43%
Kidney Stones and Urolithiasis Treatments
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Final Results of the ILLUMINATE-A Phase 3 Clinical Trial of Lumasiran for Primary Hyperoxaluria 1 — Jaap W. Groothoff, W. J. Du, et al. · Clinical Journal of the American Society of Nephrology (2025) | TGRS Research Map | TGRS