Disease · MONDO_0004739
Trials12
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 to 12 of 12
Download this page as JSON| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01257737 | To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs) | Phase 4 | 88 | 2018-05-04 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT00947297 | Study of the Safety of HPN (Hyperion)-100 for the Long-Term Treatment of Urea Cycle Disorders (Treat UCD) | Phase 3 | 60 | 2013-08-15 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT00992459 | Efficacy and Safety of HPN-100 for the Treatment of Adults With Urea Cycle Disorders | Phase 3 | 46 | 2013-08-12 | Urea Cycle Disorders | SODIUM PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT02246218 | A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders | Phase 4 | 27 | 2019-03-05 | Urea Cycle Disorder | GLYCEROL PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT01347073 | Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders (UCDs) | Phase 3 | 23 | 2015-05-28 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT00947544 | Study of the Safety and Tolerability of HPN-100 Compared to Sodium Phenylbutyrate in Children With Urea Cycle Disorders | Phase 2 | 17 | 2013-12-25 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE, SODIUM PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT03335488 | Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) | Phase 4 | 16 | 2023-07-10 | Urea Cycle Disorder | GLYCEROL PHENYLBUTYRATE, SODIUM PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT00551200 | Dose-Escalation Safety Study of HPN-100 to Treat Urea Cycle Disorders | Phase 2 | 14 | 2015-05-12 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE, SODIUM PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
| NCT06904027 | A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders | 40 | Urea Cycle Disorders | GLYCEROL PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov | ||
| NCT00004767 | Phase II Study of Sodium Phenylbutyrate, Sodium Benzoate, Sodium Phenylacetate, and Dietary Intervention for Urea Cycle Disorders | Phase 2 | 20 | Amino Acid Metabolism, Inborn Errors | SODIUM BENZOATE, SODIUM PHENYLACETATE, SODIUM PHENYLBUTYRATE | Its record names the disease | ClinicalTrials.gov | |
| NCT01765283 | Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN) | Phase 1/2 | 20 | Urea Cycle Disorders, Crigler Najjar Syndrome | BIVALIRUDIN, HEPARIN | Its record names the disease | ClinicalTrials.gov | |
| NCT03933410 | UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder | Phase 2 | 13 | Urea Cycle Disorder | ONTERODRIMER | Its record names the disease | ClinicalTrials.gov |
diseases-2026-09-18-0.1.0
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 list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0004739/trials