Disease · MONDO_0008728
classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency. Diseases, record MONDO_0008728. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0008728
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.
Drugs7
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 |
|---|---|---|---|---|---|---|---|---|
| CORTISONE ACETATE | CHEMBL1650 | Small molecule | Approval | DailyMed | 2 | 0 | 0 | Drugs and targets |
| DEXAMETHASONE | CHEMBL384467 | Small molecule | Approval | DailyMed | 2 | 0 | 0 | Drugs and targets |
| DEXAMETHASONE SODIUM PHOSPHATE | CHEMBL2021430 | Small molecule | Approval | DailyMed | 2 | 0 | 0 | Drugs and targets |
| PREDNISOLONE | CHEMBL131 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| PREDNISONE | CHEMBL635 | Small molecule | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| ABIRATERONE ACETATE | CHEMBL271227 | Small molecule | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| TILDACERFONT | CHEMBL5095198 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets2
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 | Source |
|---|---|---|---|---|---|---|
| NR3C1 | Glucocorticoid receptor | 5 | Glucocorticoid receptor agonist | Approval | https://conductscience.org/library/genes/2908 | Drugs and targets |
| CYP17A1 | Steroid 17-alpha-hydroxylase/17,20 lyase | 1 | Cytochrome P450 17A1 inhibitor | Phase 2 | https://conductscience.org/library/genes/1586 | Drugs and targets |
Trials4
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.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03687242 | Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia | Phase 2 | 11 | 2025-04-01 | Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency | TILDACERFONT | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT02574910 | Androgen Reduction in Congenital Adrenal Hyperplasia, Phase 1 | Phase 1 | 4 | 2025-11-04 | Congenital Adrenal Hyperplasia | ABIRATERONE ACETATE | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT01495910 | A Study Examining Doses of Abiraterone Acetate in Adult Women With 21-Hydroxylase Deficiency | Phase 1 | 6 | 21-hydroxylase Deficiency | ABIRATERONE ACETATE | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT03548246 | Androgen Reduction in Congenital Adrenal Hyperplasia | Phase 2 | 0 | Congenital Adrenal Hyperplasia | ABIRATERONE ACETATE | Open Targets' reading of its record | ClinicalTrials.gov |
Comparisons in its trials0
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.
None in this release.
Papers from its trials1
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it |
|---|---|---|
| 34146101 | RESULT | 1 |
Source PubMed
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/MONDO_0008728
GET https://conductscience.org/api/diseases/v1/MONDO_0008728/drugs