Browse recruiting and upcoming clinical trials for primary hyperoxaluria.
Patient-friendly, AI-assisted summaries based on official ClinicalTrials.gov ↗ records. Last updated: Oct 10, 2026
About this list Hide ClinicalTrials.gov is the complete and most up-to-date source. Trials may be missing, and trial information may have changed since our last check. Always verify important information in the official registry.
12 trials + 1 related FiltersSex All Female (includes ALL) Male (includes ALL) All sexes only
Age All ages Pediatric (under 18) Adult (18+)
Clear Who can join AI People with Primary Hyperoxaluria Type 1 (PH1).
What is being studied AI Tests safety, tolerability, pharmacokinetics, and effects of a single IV dose of YOLT-203.
Study type Treatment (drug) · Phase 1
Who can join AI People aged 6 years and older with genetically confirmed Primary Hyperoxaluria Type 1 (PH1).
What is being studied AI Tests a single-dose gene-editing infusion (YOLT-203) versus placebo for PH1.
Study type Treatment (drug) · Phase 2
Who can join AI People aged 6–64 with genetically confirmed primary hyperoxaluria type 1 (AGXT) meeting specified kidney, urine oxalate, and weight limits.
What is being studied AI Tests safety, dosing, and biological effects of a single IV dose of ABO-101.
Study type Treatment (drug) · Phase 1/2
Countries France, Germany, Tunisia +2
Who can join AI People aged ≥2 years with genetically confirmed Type 1 Primary Hyperoxaluria (PH1) who meet kidney and urine-oxalate criteria.
What is being studied AI Tests safety, tolerability, PK/PD and effect of a single IV dose of YOLT-203 on oxalate.
Study type Treatment (drug) · Early Phase 1
Who can join AI People with primary hyperoxaluria type 1 (PH1) who have severe renal impairment or end‑stage renal disease (with or without dialysis).
What is being studied AI Tests monthly DCR-PHXC for safety and effects on plasma oxalate in PH1 with ESRD.
Study type Treatment (drug) · Phase 2
Countries France, Germany, Italy +7
Who can join AI People aged 6 years and older with genetically confirmed primary hyperoxaluria type 1, 2, or 3.
What is being studied AI Tests whether stiripentol changes 24-hour urinary oxalate compared with placebo and assesses safety.
Study type Treatment (drug) · Phase 3
Who can join AI People of any age with primary hyperoxaluria type 1 who have been treated with lumasiran in France.
What is being studied AI Collects urine and clinical data to track oxaluria and PH1 course before and after lumasiran.
Who can join AI People with rare or suspected monogenic kidney stone diseases and their family members.
What is being studied AI Genetic testing and collection of DNA and clinical stone-history information.
Who can join AI People with primary or enteric hyperoxaluria, cystinuria, Dent disease (including carriers), Lowe syndrome, or APRT deficiency.
What is being studied AI Collects clinical data and blood/urine inflammatory markers over time.
Countries Canada, Iceland, Israel +1
Who can join AI People with primary hyperoxaluria, Dent disease, APRT deficiency, or cystinuria — and their family members.
What is being studied AI Collects and stores biologic samples for future research.
Who can join AI People with Primary Hyperoxaluria, Dent disease, Cystinuria, or APRT deficiency.
What is being studied AI Collects medical and clinical data into a registry for these hereditary kidney stone diseases.
Countries Iceland, United States
Who can join AI Children and people with one of the many rare kidney diseases listed in this registry.
What is being studied AI Collects clinical data, patient-reported outcomes, and samples to support research.
Trials that may include primary hyperoxaluria (1) ClinicalTrials.gov returned these when we searched for this condition, but we could not confirm from the registry records that they are intended for people with it. Check each trial’s eligibility criteria, and ask the study team if you are unsure.
Who can join AI Adults (≥18 years) with papulopustular rosacea (PPR).
What is being studied AI Tests remibrutinib versus placebo for reducing inflammatory lesions in papulopustular rosacea.
Study type Treatment (drug) · Phase 2
Countries Canada, China, Singapore +2