Primary hyperoxaluria type 1 clinical trials
The registry lists 22 studies for Primary hyperoxaluria type 1 across more than 10 countries. 8 are recruiting or about to start. 20 of them are mainly about Primary hyperoxaluria type 1 (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-19.
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Status
Who runs them
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (5) · Alnylam Pharmaceuticals (5) · Hospices Civils de Lyon (2) · YolTech Therapeutics Co., Ltd (2) · RTI International (1) · Ann & Robert H Lurie Children's Hospital of Chicago (1) · Biocodex (1) · Mayo Clinic (1)
What is being tested here
Drugs under test in Primary hyperoxaluria type 1 studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 4
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1) · RenJi Hospital
- Aug 2027Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1) · YolTech Therapeutics Co., Ltd
- Oct 2027Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1) · YolTech Therapeutics Co., Ltd
Who these studies take
Of the 7 Primary hyperoxaluria type 1 studies recruiting now, 5 (71%) set no upper age limit and 1 (14%) stop at 75 or younger. 7 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 7 (100%) | 7 (100%) |
| 18 | 7 (100%) | 7 (100%) |
| 30 | 7 (100%) | 7 (100%) |
| 40 | 7 (100%) | 7 (100%) |
| 50 | 7 (100%) | 7 (100%) |
| 65 | 6 (86%) | 6 (86%) |
| 75 | 6 (86%) | 6 (86%) |
| 85 | 6 (86%) | 6 (86%) |
Across all 22 studies, recruiting or not: 27% stop at 75 or younger and 27% at 65; 100% take children; 0% are for women only and 0% for men only; 18% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 22 of 22. search them.
- Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)RecruitingNCT06839235Phase 1/25 countries
- Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)RecruitingNCT07587021Phase 21 country
- Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)RecruitingNCT07776626Phase 11 country
- Rare Kidney Stone Consortium BiobankRecruitingNCT020263881 country
- Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRDRecruitingNCT04580420Phase 210 countries
- Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)RecruitingNCT06511349Early Phase 11 country
- Evaluation of the Efficacy and Safety of Stiripentol in Patients 6 Years and Older With Primary Hyperoxaluria Type 1, 2 or 3Not yet recruitingNCT06465472Phase 3
- Retrospective and Prospective Follow-up of Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran in France.RecruitingNCT062258821 country
- Long Term Extension Study in Patients With Primary HyperoxaluriaActive, not recruitingNCT04042402Phase 313 countries
- BONAPH1DE, A Prospective Observational Study of Patients With Primary Hyperoxaluria Type 1 (PH1)Active, not recruitingNCT0498239311 countries
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal FunctionCompletedNCT05001269Phase 211 countries
- Treatment of Primary Hyperoxaluria Type 1 With NedosiranNo longer availableNCT05993416
- A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1CompletedNCT04152200Phase 311 countries
- Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)Active, not recruitingNCT06892301Early Phase 11 country
- A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1CompletedNCT03905694Phase 35 countries
- A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1CompletedNCT03681184Phase 38 countries
- A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)TerminatedNCT02795325Phase 12 countries
- A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2CompletedNCT03847909Phase 215 countries
- Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1CompletedNCT02706886Phase 1/25 countries
- Proteomics of Primary Hyperoxaluria Type 1CompletedNCT03067142
- IDENTIFICATION OF A MULTI-ANALYTE PROFILE FOR PRIMARY HYPEROXALURIA AND COMPARISON WITH HEALTHY SIBLINGS AND IDIOPATHIC HYPERCALCIURIACompletedNCT02830009Not applicable1 country