Randomized Clinical Trial on the Long-Term Efficacy and Safety of Lumasiran in Patients With Primary Hyperoxaluria Type 1.
RNA interference
lumasiran
nephrocalcinosis
phase 3 clinical trial
primary hyperoxaluria type 1
urinary oxalate
Journal
Kidney international reports
ISSN: 2468-0249
Titre abrégé: Kidney Int Rep
Pays: United States
ID NLM: 101684752
Informations de publication
Date de publication:
Mar 2022
Mar 2022
Historique:
received:
12
11
2021
accepted:
03
12
2021
entrez:
8
3
2022
pubmed:
9
3
2022
medline:
9
3
2022
Statut:
epublish
Résumé
Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. In the 6-month double-blind period (DBP) of ILLUMINATE-A, a phase 3, randomized, placebo-controlled trial in patients with PH1 ≥6 years old, treatment with lumasiran, an RNA interference therapeutic, led to substantial reductions in urinary oxalate (UOx) levels. We report data to month 12 in the extension period (EP) of ILLUMINATE-A, including patients who continued lumasiran (lumasiran/lumasiran) or crossed over from placebo to lumasiran (placebo/lumasiran). In the lumasiran/lumasiran group ( Long-term lumasiran treatment enabled sustained lowering of UOx levels with acceptable safety and encouraging results on clinical outcomes.
Identifiants
pubmed: 35257062
doi: 10.1016/j.ekir.2021.12.001
pii: S2468-0249(21)01591-6
pmc: PMC8897294
doi:
Types de publication
Journal Article
Langues
eng
Pagination
494-506Informations de copyright
© 2022 International Society of Nephrology. Published by Elsevier Inc.
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