Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial
| Autor: | 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. |
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| Přispěvatelé: | 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] |
| Rok vydání: | 2023 |
| Předmět: |
Adult
Male RNA interference (RNAi) safety Lumasiran urinary oxalate (UOx) Adolescent [SDV]Life Sciences [q-bio] kidney disease efficacy Young Adult nephrocalcinosis glycolate pharmacodynamics Humans Child Hyperoxaluria Oxalates systemic oxalosis hemodialysis cardiac dysfunction phase 3 clinical trial Infant Newborn Infant Middle Aged adverse events primary hyperoxaluria type 1 (PH1) [SDV] Life Sciences [q-bio] plasma oxalate (POx) anti-drug antibodies pediatric Nephrology Child Preschool Hyperoxaluria Primary Female Kidney Diseases pharmacokinetics |
| Zdroj: | 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⟩ |
| ISSN: | 0272-6386 1523-6838 |
| DOI: | 10.1053/j.ajkd.2022.05.012 |
| Popis: | 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 |
| Databáze: | OpenAIRE |
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