ArticleKidney international reports2025
PHYOX3: Nedosiran Long-Term Safety and Efficacy in Patients With Primary Hyperoxaluria Type 1.
Article in Kidney international reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03392896 (A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection), which is not on this map. Cited by 5 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use)
Who cites it
5 citing papers in PubMed.
- Advancing the Landscape of RNAi Nanotherapeutics for Ischemic Heart Disease.Advanced materials (Deerfield Beach, Fla.) · 2026Review
- Clinical Approaches and Emerging Therapeutic Horizons in Primary Hyperoxaluria.Journal of clinical medicine · 2026Review
- The Dawn of Precision Medicine in Pediatric Nephrology: Lumasiran and the Era of siRNA Therapies for Primary Hyperoxaluria Type 1.Journal of personalized medicine · 2026Review
- Quality assessment of large language model-generated prior authorization letters in nephrology.Frontiers in digital health · 2026Article
- Zilebesiran as an Innovative siRNA-Based Therapeutic Approach for Hypertension: Emerging Perspectives in Cardiovascular Medicine.International journal of molecular sciences · 2025Review
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Authors and funding
11 authors.
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Abstract
Introduction: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease characterized by oxalate overproduction in the liver, leading to hyperoxaluria, calcium oxalate stones, nephrocalcinosis, progressive chronic kidney damage, kidney failure, and systemic oxalate deposition. Nedosiran, an RNA interference therapy against lactate dehydrogenase subunit A mRNA, has been approved in the USA for treating patients with PH1 who are aged ≥ 9 years and have an estimated glomerular filtration rate (eGFR) ≥ 30 ml/min per 1.73 m Methods: This PHYOX3 interim analysis includes 40 participants with PH1 from PHYOX1 (NCT03392896; Results: At baseline, mean (SD) age was 24.9 (9.7) years (55% females; 42.5% White), mean (SD) eGFR was 80.0 (28.6) ml/min per 1.73 m Conclusion: Nedosiran was well-tolerated, reduced average Uox levels, reduced kidney stone occurrence, and maintained stable renal function for over 3 years.
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