Condition (MeSH)
Hyperoxaluria, Primary
ClinicalTrials.gov lists 41 trials for Hyperoxaluria, Primary or a narrower condition beneath it.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
41 of the 41 trials are tagged with Hyperoxaluria, Primary itself; the rest are tagged with a narrower condition beneath it.
When did Hyperoxaluria, Primary trials start? (16)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 1 |
| 2024 | 1 |
| 2022 | 3 |
| 2021 | 2 |
| 2020 | 2 |
| 2019 | 5 |
| 2018 | 4 |
| 2017 | 2 |
| 2016 | 3 |
Showing 10 of 16 earlier years.
No forecast is made for this condition: it averages 1.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 3 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Hyperoxaluria, Primary trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 4 |
| Phase 2 | 9 |
| Phase 2/3 | 2 |
| Phase 3 | 8 |
| Not applicable | 1 |
| Observational | 13 |
| Expanded access | 1 |
How many Hyperoxaluria, Primary trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 8 |
| Active, not recruiting | 1 |
| Completed | 24 |
| Approved for marketing | 1 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Hyperoxaluria, Primary trials? (8)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Alnylam Pharmaceuticals | 6 |
| OxThera | 6 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 5 |
| Biocodex | 2 |
| YolTech Therapeutics Co., Ltd | 2 |
Showing 5 of 8 sponsors.
Which drugs are being tested for Hyperoxaluria, Primary? (5)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Lumasiran | 6 |
| Nedosiran | 5 |
| Oxalobacter formigenes | 3 |
| Stiripentol | 2 |
| Betaine | 1 |
Interventions as named in the registry (10)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| YOLT-203 | 2 |
| Oxabact OC5 capsules | 1 |
| Urine samples collect | 1 |
| Oxabact OC5 - Oxalobacter formigenes HC-1 | 1 |
| Hydroxyproline and Leucine | 1 |
Showing 5 of 10 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Hyperoxaluria, Primary trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Nephrolithiasis | 11 | 6 |
| Abdominal pain | 4 | 3 |
| Renal colic | 4 | 3 |
| Ureterolithiasis | 4 | 2 |
| Device related infection | 3 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Hyperoxaluria, Primary? (28)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Population Pharmacokinetic and Pharmacodynamic Modelling and Simulation for Nedosiran Clinical Development and Dose Guidance in Pediatric Patients with Primary Hyperoxaluria Type 1. | Clinical pharmacokinetics | 2025 | Yes |
| Lumasiran for Advanced Primary Hyperoxaluria Type 1: Phase 3 ILLUMINATE-C Trial. | American journal of kidney diseases : the official journal of the National Kidney Foundation | 2023 | Yes |
| Efficacy and safety of lumasiran for infants and young children with primary hyperoxaluria type 1: 12-month analysis of the phase 3 ILLUMINATE-B trial. | Pediatric nephrology (Berlin, Germany) | 2023 | Yes |
| PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2. | Kidney international | 2023 | Yes |
| Safety, pharmacodynamics, and exposure-response modeling results from a first-in-human phase 1 study of nedosiran (PHYOX1) in primary hyperoxaluria. | Kidney international | 2022 | Yes |
Showing 5 of 28 publications.
Prevalence (9)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Primary hyperoxaluria | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Primary hyperoxaluria | Annual incidence | <1 / 1 000 000 | Worldwide | Not stated | Unspecified |
| Primary hyperoxaluria | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Primary hyperoxaluria | Annual incidence | <1 / 1 000 000 | Worldwide | Not stated | Exact |
| Primary hyperoxaluria | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Unspecified |
Showing 5 of 9 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Primary hyperoxaluria | 416 | Exact | Orphadata Science knowledge files, Product 1 |
| Primary hyperoxaluria | 416 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.