Evidence map›Paper›PMID 39875734›Full record

Trial reportPediatric nephrology (Berlin, Germany)2025

Nedosiran in pediatric patients with PH1 and relatively preserved kidney function, a phase 2 study (PHYOX8).

David J Sas, Sevcan A Bakkaloglu, Vladimir Belostotsky, Wesley Hayes, Gema Ariceta, Jing Zhou, Verity Rawson

Registry-linked trialAbstract readClinical Trial, Phase IIMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Pediatric nephrology (Berlin, Germany), 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05001269 (A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function), which is not on this map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

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.

NCT05001269 phase2completednot on this map

A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function

TypeinterventionalSponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk companyRan2022 to 2025Enrolled27ConditionsPrimary Hyperoxaluria, Primary Hyperoxaluria Type 1, Primary Hyperoxaluria Type 2, Primary Hyperoxaluria Type 3Armsnedosiran
3 · Its place in the literature

Who cites it

6 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Hyperoxaluria: Diagnosis and Treatment.The Urologic clinics of North America · 2025
    Review
  6. Small interfering RNA: From designing to therapeutic in cancer.Journal, genetic engineering & biotechnology · 2025
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

David J SasDivision of Pediatric Nephrology and Hypertension, Mayo Clinic, Rochester, MN, USA. Sas.David@mayo.edu.
Sevcan A BakkalogluDepartment of Pediatric Nephrology, Gazi University, Ankara, Turkey.
Vladimir BelostotskyDepartment of Pediatrics, McMaster Children's Hospital, Hamilton, Canada.
Wesley HayesDepartment of Pediatric Nephrology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
Gema AricetaPediatric Nephrology, University Hospital Vall d'Hebron, Barcelona, Spain.
Jing ZhouNovo Nordisk A/S, Lexington, MA, USA.
Verity RawsonNovo Nordisk A/S, Lexington, MA, USA.

Funding

Dicerna Pharmaceuticals, Inc.Novo Nordisk Company (Lexington, MA, USA)
6 · The paper itself

Abstract

backgroundPrimary hyperoxaluria type 1 (PH1) is an autosomal recessive disorder with dysregulated glyoxylate metabolism in the liver. Oxalate over-production leads to renal stones, progressive kidney damage and renal failure, with potentially life-threatening systemic oxalosis. Nedosiran is a synthetic RNA interference therapy, designed to reduce hepatic lactate dehydrogenase (LDH) to decrease oxalate burden in PH.

methodsCurrently, in the PHYOX8 study (NCT05001269), pediatric participants (2-11 years) with PH1 (N = 15) and estimated glomerular filtration rate (eGFR) ≥ 30mL/min/1.73m

resultsUrinary oxalate:creatinine (Uox:Ucr) levels reduced by 64% on average. Mean Uox:Ucr reduction was 52% at day 60 and ˃60% at day 180. At one or more study visits, 93.3% (N = 14) of participants reached Uox:Ucr < 1.5 × upper limit of normal (ULN), and 53.3% (N = 8) reached ≤ 1.0 × ULN. Median percent change in plasma oxalate (12.0 µmol/L at baseline) to day 180 was -39.23% (n = 10). Average number of kidney stones per participant remained stable, whilst a 10.1% average decrease in summed surface area was observed. Median percent change from baseline in eGFR was 2.5%, indicating preservation of renal function.

conclusionsNedosiran was well tolerated, with only 3 participants experiencing at least one serious adverse event, none considered treatment-related. The incidence of injection site reactions was 6.7% (1/15 participants). In conclusion, nedosiran treatment led to a significant and sustained reduction of Uox levels in children with PH1. These findings support nedosiran treatment in pediatric patients to reduce Uox and shows promise for limiting PH1-related complications.

Indexed as

Hyperoxaluria, PrimaryL-Lactate DehydrogenaseChildChild, PreschoolCreatinineFemaleGlomerular Filtration RateHumansKidneyLactate DehydrogenasesMaleOxalatesTreatment OutcomeCreatinineLactate DehydrogenasesL-Lactate DehydrogenasenedosiranOxalatesChronic kidney diseaseGene expressionHyperoxaluriaPediatric nephrologyRNAiUrology

Identifiers

PMID39875734
PMCPMC12031765

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.