Condition (MeSH)
Urea Cycle Disorders, Inborn
ClinicalTrials.gov lists 114 trials for Urea Cycle Disorders, Inborn or a narrower condition beneath it. Amgen has the most among industry and government lead sponsors, with 10.
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Trials per phase
65 of the 114 trials are tagged with Urea Cycle Disorders, Inborn itself; the rest are tagged with a narrower condition beneath it.
When did Urea Cycle Disorders, Inborn trials start? (23)
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. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 8
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) | 6 |
| 2025 | 4 |
| 2024 | 7 |
| 2023 | 5 |
| 2022 | 8 |
| 2021 | 5 |
| 2020 | 5 |
| 2019 | 8 |
| 2018 | 7 |
| 2017 | 7 |
Showing 10 of 23 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 8 trials starting in 2026 and about 7 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 7 trials still running at the 2026-10-09 snapshot; registered median of 22 months across 73 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Urea Cycle Disorders, Inborn trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 11 |
| Phase 1/2 | 8 |
| Phase 2 | 16 |
| Phase 3 | 10 |
| Phase 4 | 4 |
| Not applicable | 21 |
| Observational | 44 |
How many Urea Cycle Disorders, Inborn trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 4 |
| Recruiting | 17 |
| Active, not recruiting | 7 |
| Completed | 62 |
| Terminated | 14 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Urea Cycle Disorders, Inborn trials? (10)
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 |
|---|---|
| Amgen | 10 |
| Ultragenyx Pharmaceutical Inc | 6 |
| Arcturus Therapeutics, Inc. | 4 |
| Cellaion SA | 4 |
| Cytonet GmbH & Co. KG | 4 |
Showing 5 of 10 sponsors.
Which drugs are being tested for Urea Cycle Disorders, Inborn? (3)
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 |
|---|---|
| HPN-100 | 9 |
| Pegzilarginase | 5 |
| KB195 | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| 4-Phenylbutyrate | 5 |
| ARCT-810 | 4 |
| NaPBA | 2 |
| Arginine | 2 |
| HepaStem | 2 |
Showing 5 of 12 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 Urea Cycle Disorders, Inborn 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 |
|---|---|---|
| Hyperammonaemia | 95 | 8 |
| Chest Pain | 24 | 1 |
| Vomiting | 22 | 6 |
| Hyperammonaemic Crisis | 14 | 2 |
| Other | 14 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Urea Cycle Disorders, Inborn? (28)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Rapid Targeted Sequencing Using Dried Blood Spot Samples for Patients With Suspected Actionable Genetic Diseases. | Annals of laboratory medicine | 2023 | Yes |
| Health-related quality of life in a systematically assessed cohort of children and adults with urea cycle disorders. | Molecular genetics and metabolism | 2023 | Yes |
| The challenge of understanding and predicting phenotypic diversity in urea cycle disorders. | Journal of inherited metabolic disease | 2023 | Yes |
| Fifteen years of urea cycle disorders brain research: Looking back, looking forward. | Analytical biochemistry | 2022 | Yes |
| Biomarkers for liver disease in urea cycle disorders. | Molecular genetics and metabolism | 2021 | Yes |
Showing 5 of 28 publications.
Sub-conditions in MeSH (5)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.