Condition (MeSH)
Argininosuccinic Aciduria
ClinicalTrials.gov lists 12 trials for Argininosuccinic Aciduria 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
12 of the 12 trials are tagged with Argininosuccinic Aciduria itself; the rest are tagged with a narrower condition beneath it.
When did Argininosuccinic Aciduria trials start? (10)
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 2
- Not applicable
- Observational
Top of scale: 3
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) | 1 |
| 2022 | 3 |
| 2021 | 1 |
| 2020 | 1 |
| 2018 | 1 |
| 2017 | 1 |
| 2014 | 1 |
| 2011 | 1 |
| 2008 | 1 |
| 2007 | 1 |
No forecast is made for this condition: it averages 1.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 12 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Argininosuccinic Aciduria trials in? (4)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1/2 | 1 |
| Phase 2 | 2 |
| Not applicable | 2 |
| Observational | 7 |
How many Argininosuccinic Aciduria trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Completed | 5 |
| Terminated | 1 |
| Unknown status | 1 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Argininosuccinic Aciduria trials? (2)
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 |
|---|---|
| Korro Bio, Inc. | 1 |
| National Human Genome Research Institute (NHGRI) | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Standard of Care Therapy | 1 |
| 4-Phenylbutyrate | 1 |
| KRRO-121 | 1 |
| Arginine | 1 |
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 publications cover Argininosuccinic Aciduria? (8)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| From genotype to phenotype: Early prediction of disease severity in argininosuccinic aciduria. | Human mutation | 2020 | Yes |
| Biochemical markers and neuropsychological functioning in distal urea cycle disorders. | Journal of inherited metabolic disease | 2018 | Yes |
| Nitric-oxide supplementation for treatment of long-term complications in argininosuccinic aciduria. | American journal of human genetics | 2012 | Yes |
| Optimizing therapy for argininosuccinic aciduria. | Molecular genetics and metabolism | 2012 | Yes |
| Requirement of argininosuccinate lyase for systemic nitric oxide production. | Nature medicine | 2011 | Yes |
Showing 5 of 8 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Argininosuccinic aciduria | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Argininosuccinic aciduria | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Exact |
| Argininosuccinic aciduria | Prevalence at birth | 1-9 / 1 000 000 | United States | US | Unspecified |
| Argininosuccinic aciduria | Prevalence at birth | 1-9 / 1 000 000 | Finland | Not stated | Unspecified |
| Argininosuccinic aciduria | Prevalence at birth | 1-9 / 100 000 | Austria | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Argininosuccinic aciduria | 23 | Exact | Orphadata Science knowledge files, Product 1 |
| Argininosuccinic aciduria | 23 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials and sub-conditions in mesh.