RecruitingPhase 1NCT07776626
Renal
Primary Hyperoxaluria
Also known as PH, PH1/PH2/PH3, AGXT deficiency hyperoxaluria
Primary hyperoxaluria is a group of rare autosomal recessive disorders of glyoxylate metabolism causing overproduction of oxalate, leading to recurrent calcium oxalate nephrolithiasis, nephrocalcinosis, and progressive renal failure. Type 1
ORPHA:416 ↗Gene AGXTGene GRHPRGene HOGA1Prevalence Approximately 1–3 per millionOnset Variable; infancy to adulthood depending on subtype
12
studies recruiting now
as of 7 Sept 2026
54
studies registered in total
as of 7 Sept 2026
6
countries with a recruiting site
as of 7 Sept 2026
20 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
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Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
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Prospective Research Rare Kidney Stones (ProRKS)
RecruitingNCT02026388
Rare Kidney Stone Consortium Biobank
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 12 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Primary Hyperoxaluria
Primary hyperoxaluria is a group of rare autosomal recessive disorders of glyoxylate metabolism causing overproduction of oxalate, leading to recurrent calcium oxalate nephrolithiasis, nephrocalcinosis, and progressive renal failure. Type 1 (AGXT deficiency) is the most severe and common form, frequently resulting in systemic oxalosis when the kidney fails and oxalate deposits in bones, eyes, and heart. Lumasiran (siRNA targeting LDHA) has been approved for PH1, representing a significant therapeutic advance.
Common clinical features
Recurrent calcium oxalate kidney stones from early ageNephrocalcinosisProgressive chronic kidney diseaseSystemic oxalosis (bones, eyes, heart, skin in advanced disease)HaematuriaUrinary tract infections secondary to stone burdenAnaemia and bone pain (systemic oxalosis)
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
2 approved treatments and 6 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Approved: LumasiranApproved: Nedosiran Sodium (Rivfloza)
Phase 3Reloxaliase
Phase 3Losartan
Phase 3Lisinopril Anhydrous
Phase 2Stiripentol (Diacomit)
Phase 2Betaine (Amversio)
Phase 1/2Leucine
Before you apply
Things trial teams commonly ask about for Primary Hyperoxaluria. Not eligibility rules; those are set by each study.
- Subtype classification (PH1, PH2, or PH3) by genetic testing is critical as approved therapies and trial eligibility differ by subtype; confirm the causative gene before applying.
- 24-hour urinary oxalate excretion is the primary pharmacodynamic endpoint in most trials; establish a reliable baseline with repeated measurements under controlled dietary conditions.
- Trials for PH1 gene therapy or RNA interference may require a minimum eGFR threshold to ensure adequate drug clearance; check renal function eligibility carefully.
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).