| Online-Ressource |
Verfasst von: | Groothoff, Jaap [VerfasserIn]  |
| Sellier-Leclerc, Anne-Laure [VerfasserIn]  |
| Deesker, Lisa [VerfasserIn]  |
| Bacchetta, Justine [VerfasserIn]  |
| Schalk, Gesa [VerfasserIn]  |
| Tönshoff, Burkhard [VerfasserIn]  |
| Lipkin, Graham [VerfasserIn]  |
| Lemoine, Sandrine [VerfasserIn]  |
| Bowman, Thomas [VerfasserIn]  |
| Zhou, Jing [VerfasserIn]  |
| Hoppe, Bernd [VerfasserIn]  |
Titel: | Nedosiran safety and efficacy in PH1 |
Titelzusatz: | interim analysis of PHYOX3 |
Verf.angabe: | Jaap Groothoff, Anne-Laure Sellier-Leclerc, Lisa Deesker, Justine Bacchetta, Gesa Schalk, Burkhard Tönshoff, Graham Lipkin, Sandrine Lemoine, Thomas Bowman, Jing Zhou and Bernd Hoppe |
E-Jahr: | 2024 |
Jahr: | May 2024 |
Umfang: | 10 S. |
Illustrationen: | Diagramme |
Fussnoten: | Gesehen am 22.01.2025 |
Titel Quelle: | Enthalten in: Kidney international. Reports |
Ort Quelle: | Amsterdam : Elsevier, 2016 |
Jahr Quelle: | 2024 |
Band/Heft Quelle: | 9(2024), 5 vom: Mai, Seite 1387-1396 |
ISSN Quelle: | 2468-0249 |
Abstract: | Introduction - Primary hyperoxaluria (PH) is a rare genetic disorder of hepatic glyoxylate metabolism. Nedosiran is an RNA interference (RNAi) therapeutic that the US Food and Drug Administration has approved for treatment of PH1. PHYOX3 is a trial evaluating monthly nedosiran in patients with PH. - Methods - In this PHYOX3 interim analysis, participants with PH1 who continued from a single-dose nedosiran trial (PHYOX1), with no previous kidney or liver transplantation, dialysis, or evidence of systemic oxalosis were eligible. The safety and efficacy of once-monthly nedosiran was assessed over 30 months. - Results - Thirteen participants completed PHYOX1 and continued into PHYOX3. At baseline, the mean (SD) and median (range) age was 24.2 (6.6) years and 23.0 (14-39) years, respectively; 53.8% were female and 61.5% were White. Mean estimated glomerular filtration rate (eGFR) remained stable (62-84.2 mL/min per 1.73 m2) to month 30. Mean 24-hour urinary oxalate (Uox) excretion showed a sustained reduction from baseline of ≥60% at every visit (months 2-30). From month 2, at least 10 of 13 (76.9%) participants achieved normal (<0.46 mmol/24h; upper limit of assay-normal [ULN]) or near-normal (≥0.46 to <0.60 mmol/24h; ≥ULN to <1.3 × ULN) 24-hour Uox excretion. All participants experienced ≥1 adverse event (AE), mostly mild or moderate in severity (primarily, injection site events). Three serious, not treatment-related AEs were reported; there were no deaths or study discontinuations due to AEs. - Conclusion - Nedosiran was well-tolerated in patients with PH1, and treatment resulted in a sustained, substantial reduction in Uox excretion for at least 30 months in this long-term study. No safety signals have been identified to date. The PHYOX3 study is ongoing. |
DOI: | doi:10.1016/j.ekir.2024.02.1439 |
URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
kostenfrei: Volltext: https://doi.org/10.1016/j.ekir.2024.02.1439 |
| kostenfrei: Volltext: https://www.sciencedirect.com/science/article/pii/S2468024924015298 |
| DOI: https://doi.org/10.1016/j.ekir.2024.02.1439 |
Datenträger: | Online-Ressource |
Sprache: | eng |
Sach-SW: | glyoxylate metabolism |
| nedosiran |
| primary hyperoxaluria type 1 |
| RNAi therapy |
| safety/efficacy |
| urinary oxalate excretion |
K10plus-PPN: | 1915385407 |
Verknüpfungen: | → Zeitschrift |
Nedosiran safety and efficacy in PH1 / Groothoff, Jaap [VerfasserIn]; May 2024 (Online-Ressource)