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Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial

Authors :
Michael, M.
Groothoff, J. W.
Shasha-Lavsky, H.
Lieske, J. C.
Frishberg, Y.
Simkova, E.
Sellier-Leclerc, A. L.
Devresse, A.
Guebre-Egziabher, F.
Bakkaloglu, S. A.
Mourani, C.
Saqan, R.
Singer, R.
Willey, R.
Habtemariam, B.
Gansner, J. M.
Bhan, I.
Mcgregor, T.
Magen, D.
Paediatric Nephrology
AGEM - Amsterdam Gastroenterology Endocrinology Metabolism
ARD - Amsterdam Reproduction and Development
UCL - SSS/IREC/NEFR - Pôle de Néphrologie
UCL - (SLuc) Service de néphrologie
CarMeN, laboratoire
Texas Children's Hospital [Houston, USA]
Baylor College of Medicine (BCM)
Baylor University
Emma Children’s Hospital
Amsterdam UMC - Amsterdam University Medical Center
Galilee Medical Center [Nahariya, Israel]
Bar-Ilan University [Israël]
Mayo Clinic [Rochester]
Shaare Zedek Medical Center [Jerusalem, Israel]
Al Jalila Children's Specialty Hospital
Filières Maladies Rares ORKID et ERK-Net
Centre d'Investigation Clinique [Bron] (CIC1407)
Hospices Civils de Lyon (HCL)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Groupement Hospitalier Est [Bron]
Cliniques Universitaires Saint-Luc [Bruxelles]
Cardiovasculaire, métabolisme, diabétologie et nutrition (CarMeN)
Université Claude Bernard Lyon 1 (UCBL)
Université de Lyon-Université de Lyon-Hospices Civils de Lyon (HCL)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE)
Gazi University
Hôpital Hôtel Dieu de France [Beirut, Lebanon] (2HDF)
Jordan University of Science and Technology [Irbid, Jordan] (JUST)
Canberra Health Services [Garran, ACT, Australia] (CHS)
Alnylam Pharmaceuticals [Cambridge, MA, USA]
Rambam Health Care Campus [Haifa, Israel]
Source :
American journal of kidney diseases, 81(2), 145-155.e1. W.B. Saunders Ltd, American journal of kidney diseases : the official journal of the National Kidney Foundation, Vol. 81, no.2, p. 145-155.e1 (2023), American Journal of Kidney Diseases, (2022), American Journal of Kidney Diseases, American Journal of Kidney Diseases, 2022, S0272-6386 (22), pp.00771-5. ⟨10.1053/j.ajkd.2022.05.012⟩
Publication Year :
2023
Publisher :
Elsevier BV, 2023.

Abstract

Rationale & Objective: Lumasiran reduces urinary and plasma oxalate (POx) in patients with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function. ILLUMINATE-C evaluates the efficacy, safety, pharmacokinetics, and pharmacodynamics of lumasiran in patients with PH1 and advanced kidney disease. Study Design: Phase 3, open-label, single-arm trial. Setting & Participants: Multinational study; enrolled patients with PH1 of all ages, estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (if age ≥12 months) or increased serum creatinine level (if age

Details

ISSN :
02726386 and 15236838
Volume :
81
Database :
OpenAIRE
Journal :
American Journal of Kidney Diseases
Accession number :
edsair.doi.dedup.....e766f52870784a99182763b902908469