Disease · HP_0001987
Hyperammonemia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Hyperammonemia. Diseases, record HP_0001987. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/HP_0001987
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Name
- Hyperammonemia
- Open Targets ID
- HP_0001987
- Other names
- High blood ammonia levels
- Highest stage of its drugs
- Approval, for 3 of its 10 drugs, from DailyMed, EMA Human Drugs and PMDA
Source Open Targets
Drugs10
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| CARGLUMIC ACID | CHEMBL1201780 | Small molecule | Approval | DailyMed, EMA Human Drugs and PMDA | 5 | 2 | 1 | Drugs and targets |
| RIFAXIMIN | CHEMBL1617 | Small molecule | Approval | PMDA | 1 | 0 | 0 | Drugs and targets |
| SODIUM PHENYLBUTYRATE | CHEMBL1746 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| ORNITHINE | CHEMBL446143 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| VITAMIN E | CHEMBL3989727 | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets | |
| RIFAQUIZINONE | CHEMBL4594404 | Small molecule | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| GLUTAMINE | CHEMBL930 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ORNITHINE OXOGLURATE | CHEMBL3138616 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SODIUM CITRATE | CHEMBL1355 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| SODIUM BENZOATE | CHEMBL1356 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets1
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes |
|---|---|---|---|---|---|
| CPS1 | Carbamoyl-phosphate synthase [ammonia], mitochondrial | 1 | Carbamoyl-phosphate synthase [ammonia], mitochondrial positive allosteric modulator | Approval | https://conductscience.org/library/genes/1373 |
Source Drugs and targets
Trials6
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01966419 | Phase 2B Efficacy/Safety of Ornithine Phenylacetate in Hospitalized Cirrhotic Patients With Hepatic Encephalopathy (STOP-HE) | Phase 2 | 231 | 2021-09-16 | Acute Episode of Overt Hepatic Encephalopathy, Hepatic Encephalopathy | ORNITHINE | Its record names the disease | ClinicalTrials.gov |
| NCT06135675 | PK Profile and Preliminary Efficacy of TNP-2092 Capsules in Liver Cirrhosis Patients With Hyperammonemia | Phase 1/2 | 36 | 2024-05-10 | Hyperammonemia | RIFAQUIZINONE | Its record names the disease | ClinicalTrials.gov |
| NCT04022941 | Efficacy and Safety of Sodium Benzoate in the Management of Hyperammonemia in Infants, Children and Adolescent With Chronic Liver Disease. | Not applicable | 108 | Chronic Liver Disease | SODIUM BENZOATE | Its record names the disease | ClinicalTrials.gov | |
| NCT05040178 | An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics | 20 | Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia | CARGLUMIC ACID | Its record names the disease | ClinicalTrials.gov | ||
| NCT00645879 | Anaplerotic Therapy in Propionic Acidemia | Phase 1 | 3 | Propionic Acidemia | GLUTAMINE, ORNITHINE OXOGLURATE | Its record names the disease | ClinicalTrials.gov | |
| NCT04984798 | Vitamin E Efficacy in HI/HA | Phase 2 | 0 | Hyperinsulinism-Hyperammonemia Syndrome | VITAMIN E | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials2
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Ornithine Phenylacetate vs Placebo: deaths from any cause | 11 of 114 | 15 of 112 | 226 | NCT01966419 | Trial comparisons |
| Ornithine Phenylacetate vs Placebo: serious adverse events | 29 of 114 | 34 of 112 | 226 | NCT01966419 | Trial comparisons |
Papers from its trials4
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/HP_0001987
GET https://conductscience.org/api/diseases/v1/HP_0001987/drugs