AR androgen receptor
ENSG00000169083 · protein coding · Open Targets profile
Steroid hormone receptors are ligand-activated transcription factors that regulate eukaryotic gene expression and affect cellular proliferation and differentiation in target tissues (PubMed:19022849). Transcription factor activity is modulated by bound coactivator and corepressor proteins like ZBTB7A that recruits NCOR1 and NCOR2 to the androgen response elements/ARE on target genes, negatively regulating androgen receptor signaling and androgen-induced cell proliferation (PubMed:20812024). Transcription activation is also down-regulated by NR0B2. Activated, but not phosphorylated, by HIPK3 and ZIPK/DAPK3. {ECO:0000269|PubMed:14664718, ECO:0000269|PubMed:15563469, ECO:0000269|PubMed:17591767, ECO:0000269|PubMed:17911242, ECO:0000269|PubMed:18084323, ECO:0000269|PubMed:19022849, ECO:0000269|PubMed:19345326, ECO:0000269|PubMed:20812024, ECO:0000269|PubMed:20980437, ECO:0000269|PubMed:25091737}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, Structure with Ligand, High-Quality Ligand, High-Quality Pocket, Druggable Family
- Antibody
- GO CC med conf
- PROTAC
- Advanced Clinical, Literature, UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.14846999943256378 (upper bound 0.2678999900817871). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 193 registry trials, updated in the last three years, of the most advanced drugs against AR (bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthate), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Samuel DenmeadeJohns Hopkins University · 4 of 5 trials Phase 2+
- Anthony DelConteLipocine Inc. · 2 of 2 trials Phase 2+
- Antoni DulebaUniversity of California, San Diego · 2 of 2 trials Phase 2+
- Joshua F YarrowRocky Mountain Regional VA Medical Center, Aurora, CO · 2 of 2 trials Phase 2+
- Matthew BabcockVirginia Commonwealth University · 2 of 2 trials Phase 2+
- Michael SchweizerECOG-ACRIN Cancer Research Group · 2 of 2 trials Phase 2+
- Paul TorenCHU de Québec-Université Laval · 2 of 2 trials Phase 2+
- Ranjith RamasamyUniversity of Miami · 2 of 2 trials Phase 2+
- Shalender BhasinBrigham and Women's Hospital · 2 of 2 trials Phase 2+
- Shehzad BasariaBrigham and Women's Hospital · 2 of 2 trials Phase 2+
- Abdenour NabidCentre de Recherche Clinique Étienne LeBel/CHUS Fleurimont · 1 of 1 trials Phase 2+
- Abhishek SolankiEdward Hines Jr. VA Hospital, Hines, IL · 1 of 1 trials Phase 2+
- Ahmed AlyRoswell Park Cancer Institute · 1 of 1 trials Phase 2+
- Alan PollackUniversity of Miami · 1 of 1 trials Phase 2+
- Alistair SmithSyneos Health · 1 of 1 trials Phase 2+
Registry trials of bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthate updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 162 of 183 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 10 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
113 trials of drugs against AR were stopped early; 16 give no reason
Terminated, withdrawn and suspended interventional trials of bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthate, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 113.
- terminated · 2026-10-27 · National Heart, Lung, and Blood Institute (NHLBI) · Danazol“Study was stopped due to poor accrual”
- withdrawn · 2026-10-02 · Tulane University · matched by the registry's drug synonyms“The study didn't receive funding to move forward.”
- terminated · 2026-09-17 · Jefferson Drapkin · matched by the registry's drug synonyms“High rates of loss to follow up”
- terminated · 2026-09-02 · University of Colorado, Denver · matched by the registry's drug synonyms“Study completed”
- terminated · 2026-05-07 · University of Southern California · Oxandrolone“Recruitment difficulties”
- suspended · 2026-03-27 · George F. Hatch · Oxandrolone“FDA withdrew approval of the study product”
- withdrawn · 2025-09-23 · OHSU Knight Cancer Institute · Bicalutamide“Loss of funding”
- terminated · 2025-08-05 · The University of Queensland · Danazol“Data Safety Monitoring Board recommended cessation due to futility”
- terminated · 2025-07-22 · University of California, San Diego · Testosterone Cypionate 50 MG/ML Injectable Solution“The study sponsor terminated the grant funding.”
- withdrawn · 2025-07-22 · University of Nebraska · Bicalutamide“PI leaving institution.”
- withdrawn · 2025-01-13 · University of California, San Diego · matched by the registry's drug synonyms“study not funded in previous iteration. new clinicaltrials.gov protocol submitted for funded version of study”
- terminated · 2024-08-09 · University Medical Center Groningen · matched by the registry's drug synonyms“Five patients were included; it did not succeed in recruiting the target number of 6 patients. This is as a result of the rarity of this disease in men.”
- terminated · 2024-07-23 · Icahn School of Medicine at Mount Sinai · Danazol“low enrollment”
- terminated · 2024-05-23 · Carelon Research · Oxandrolone“FDA withdrawal of Oxandrin NDA and generic ANDAs”
- terminated · 2024-05-22 · Fundacion Dexeus · matched by the registry's drug synonyms“Having reached the sample size described for the interim analysis and having seen the post-interim results, the sponsor has decided to stop the trial due to futility.”
- withdrawn · 2024-04-24 · Baylor College of Medicine · matched by the registry's drug synonyms“No longer wanting to enroll or start study.”
- terminated · 2024-03-04 · OHSU Knight Cancer Institute · Bicalutamide“Low accrual”
- withdrawn · 2024-02-15 · CHU de Quebec-Universite Laval · Bicalutamide 150 mg“Results of EV302 study do not make it feasible to complete a study in second line avelumab immunotherapy. The new standard of care make this study not pertinent.”
- terminated · 2024-02-13 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · testosterone cypionate“lack of accrual”
- terminated · 2024-02-06 · University at Buffalo · matched by the registry's drug synonyms“Company no longer making same drug doses.”
- terminated · 2024-02-02 · Yale University · GnRH antagonist + MethylTESTOSTERone 5 MG“Unble to complete all arms of the planned study due to Covid stoppage and difficulty with recruiting and getting enrolled participants to return post Covid.”
- terminated · 2023-10-26 · Acerus Biopharma Inc. · matched by the registry's drug synonyms“Lack of patients and commercial reasons”
- terminated · 2023-10-12 · Massachusetts General Hospital · matched by the registry's drug synonymsNo reason given
- terminated · 2023-10-06 · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Bicalutamide 150 mg“PI Decision to close/stop the study due to no new accrual.”
- terminated · 2023-09-29 · VA Office of Research and Development · Testosterone Enanthate“Enrollment difficulties”
Paediatric incentives and obligations for AR's drugs
For bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthate: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · AR is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: no · No EMA paediatric investigation plan decision names bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthateRule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: no · No EU orphan designation names bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthateRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: no · EMA lists no centrally authorised medicine of bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthateRule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthateRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 67 interventional studies of bicalutamide, stanozolol, oxymetholone, nilutamide, methyltestosterone, danazol, fluoxymesterone, ethylestrenol, oxandrolone, testosterone undecanoate, testosterone cypionate, testosterone enanthate include patients under 18; 7 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 40 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.