Condition (MeSH)
Ornithine Carbamoyltransferase Deficiency Disease
ClinicalTrials.gov lists 59 trials for Ornithine Carbamoyltransferase Deficiency Disease or a narrower condition beneath it. Ultragenyx Pharmaceutical Inc has the most among industry and government lead sponsors, with 6.
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Trials per phase (log scale)
59 of the 59 trials are tagged with Ornithine Carbamoyltransferase Deficiency Disease itself; the rest are tagged with a narrower condition beneath it.
When did Ornithine Carbamoyltransferase Deficiency Disease trials start? (20)
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: 7
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) | 3 |
| 2025 | 2 |
| 2024 | 5 |
| 2023 | 2 |
| 2022 | 7 |
| 2021 | 3 |
| 2020 | 5 |
| 2019 | 5 |
| 2018 | 4 |
| 2017 | 3 |
Showing 10 of 20 earlier years.
No forecast is made for this condition: it averages 4.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 4 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 Ornithine Carbamoyltransferase Deficiency Disease trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 5 |
| Phase 2 | 7 |
| Phase 3 | 4 |
| Phase 4 | 1 |
| Not applicable | 6 |
| Observational | 28 |
How many Ornithine Carbamoyltransferase Deficiency Disease 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 | 3 |
| Recruiting | 9 |
| Active, not recruiting | 7 |
| Completed | 25 |
| Terminated | 10 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Ornithine Carbamoyltransferase Deficiency Disease trials? (7)
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 |
|---|---|
| Ultragenyx Pharmaceutical Inc | 6 |
| Arcturus Therapeutics, Inc. | 4 |
| Abbott Medical Devices | 2 |
| iECURE, Inc. | 2 |
| Amgen | 1 |
Showing 5 of 7 sponsors.
Interventions as named in the registry (15)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| ARCT-810 | 4 |
| Paracetamol formulation 4 | 1 |
| Human Heterologous Liver Cells | 1 |
| Paracetamol formulation 3 | 1 |
| Standard of Care Therapy | 1 |
Showing 5 of 15 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 Ornithine Carbamoyltransferase Deficiency Disease 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 | 47 | 1 |
| Chest Pain | 24 | 1 |
| Vomiting | 15 | 1 |
| Hyperammonaemic Crisis | 14 | 2 |
| Other | 14 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Ornithine Carbamoyltransferase Deficiency Disease? (25)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Predicting the disease severity in male individuals with ornithine transcarbamylase deficiency. | Annals of clinical and translational neurology | 2022 | Yes |
| Executive functioning profiles from the BRIEF across pediatric medical disorders: Age and diagnosis factors. | Child neuropsychology : a journal on normal and abnormal development in childhood and adolescence | 2016 | Yes |
| Reduced Functional Connectivity of Default Mode and Set-Maintenance Networks in Ornithine Transcarbamylase Deficiency. | PloS one | 2015 | Yes |
| Significant hepatic involvement in patients with ornithine transcarbamylase deficiency. | The Journal of pediatrics | 2014 | Yes |
| Investigating neurological deficits in carriers and affected patients with ornithine transcarbamylase deficiency. | Molecular genetics and metabolism | 2014 | Yes |
Showing 5 of 25 publications.
Prevalence (21)
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 |
|---|---|---|---|---|---|
| Ornithine transcarbamylase deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Ornithine transcarbamylase deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Exact |
| Ornithine transcarbamylase deficiency | Prevalence at birth | 1-9 / 100 000 | United States | US | Unspecified |
| Ornithine transcarbamylase deficiency | Prevalence at birth | 1-9 / 100 000 | Worldwide | Not stated | Unspecified |
| Ornithine transcarbamylase deficiency | Prevalence at birth | 1-9 / 100 000 | Australia | Not stated | Exact |
Showing 5 of 21 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Ornithine transcarbamylase deficiency | 664 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Ornithine transcarbamylase deficiency | 664 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.