Testosterone

Summary

Testosterone (CHEMBL386630) is an approved small-molecule androgen (ATC G03BA03) targeting AR; indicated across 40 conditions including orchitis and klinefelter syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: G03BA03
• Targets: 1 (AR)
• Indications: 40 conditions
• Clinical trials: 206
• Chemistry: 288.4 Da · C19H28O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL386630
Name	Testosterone
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	5408
ChEBI	CHEBI:17347
ATC	G03BA03
Molecular formula	C19H28O2
Molecular weight	288.4
InChIKey	MUMGGOZAMZWBJJ-UHFFFAOYSA-N

SMILES: CC12CCC3C(C1CCC2O)CCC4=CC(=O)CCC34C

IUPAC name: 17-hydroxy-10,13-dimethyl-1,2,6,7,8,9,11,12,14,15,16,17-dodecahydrocyclopenta[a]phenanthren-3-one

ChEBI definition: An androstanoid having 17β-hydroxy and 3-oxo groups, together with unsaturation at C-4‒C-5..

Pharmacological roles (ChEBI): androgen.

Other ChEBI roles (chemical / environmental): human metabolite, Daphnia magna metabolite, mouse metabolite.

Also known as: Androderm, Androgel, Android-t, Androlan, Andropatch, Axiron, Androgel 1%, Fortesta, Homosterone, Intrinsa, Livensa, Natesto

Patent coverage: 41,853 distinct patent families (129,997 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
AR	Androgen receptor	Agonist	7.8		P10275

Broader ChEMBL bioactivity targets: 21 (assay-derived). Sample: Prelamin-A/C, Solute carrier organic anion transporter family member 1A1, Androgen receptor, Voltage-dependent L-type calcium channel subunit alpha-1C, Thyrotropin receptor, Aromatase, Glucocorticoid receptor, Progesterone receptor, Steroid 5-alpha-reductase, Corticosteroid-binding globulin.

Bioactivity

ChEMBL activities: 49 potent at pChembl ≥ 5 of 55 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
AR	9.4	Kd	0.4	nM	CHEMBL_ACT_24824108
SHBG	9.2	Kd	0.63	nM	CHEMBL_ACT_2155746
AR	8.96	EC50	1.1	nM	CHEMBL_ACT_18184277
P15207	8.85	Ki	1.4	nM	CHEMBL_ACT_1268390
P15207	8.85	Ki	1.41	nM	CHEMBL_ACT_1411325
AR	8.72	EC50	1.9	nM	CHEMBL_ACT_24775579
P19091	8.7	EC50	2	nM	CHEMBL_ACT_2980140
AR	8.57	IC50	2.7	nM	CHEMBL_ACT_1680760
AR	8.57	IC50	2.7	nM	CHEMBL_ACT_2030222
AR	8.57	IC50	2.7	nM	CHEMBL_ACT_2049384
AR	8.5	EC50	3.16	nM	CHEMBL_ACT_2980131
P15207	8.49	IC50	3.2	nM	CHEMBL_ACT_725912
AR	8.41	IC50	3.9	nM	CHEMBL_ACT_1469054
P15207	8.39	Ki	4.11	nM	CHEMBL_ACT_7825340
AR	8.38	IC50	4.17	nM	CHEMBL_ACT_15198857
P15207	8.37	Ki	4.3	nM	CHEMBL_ACT_13340333
AR	8.37	IC50	4.27	nM	CHEMBL_ACT_15198867
AR	8.34	IC50	4.57	nM	CHEMBL_ACT_12079075
P15207	8.31	Ki	4.9	nM	CHEMBL_ACT_1210461
P15207	8.31	Ki	4.9	nM	CHEMBL_ACT_1456591
P15207	8.3	AC50	5	nM	CHEMBL_ACT_25187179
P15207	8.21	IC50	6.17	nM	CHEMBL_ACT_7825339
P15207	8.19	IC50	6.5	nM	CHEMBL_ACT_13340398
P19091	7.96	IC50	11	nM	CHEMBL_ACT_26707229
SHBG	7.85	IC50	14	nM	CHEMBL_ACT_1796081
AR	7.54	Ki	29	nM	CHEMBL_ACT_5221852
AR	7.52	IC50	30	nM	CHEMBL_ACT_3591567
TSHR	7.5	Potency	31.6	nM	CHEMBL_ACT_3926498
TSHR	7.5	Potency	31.6	nM	CHEMBL_ACT_4691397
CACNA1C	7.47	IC50	34	nM	CHEMBL_ACT_15373297

Target pathways

Aggregated over 1 target gene(s): AR.

Top Reactome pathways

23 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	1	AR
Signaling by Rho GTPases	1	AR
RHO GTPase Effectors	1	AR
Generic Transcription Pathway	1	AR
Cellular responses to stress	1	AR
SUMOylation	1	AR
SUMO E3 ligases SUMOylate target proteins	1	AR
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	1	AR
Nuclear Receptor transcription pathway	1	AR
Metabolism of proteins	1	AR
SUMOylation of intracellular receptors	1	AR
RHO GTPases activate PKNs	1	AR
Activated PKN1 stimulates transcription of AR (androgen receptor) regulated genes KLK2 and KLK3	1	AR
Deubiquitination	1	AR
Ub-specific processing proteases	1	AR
Post-translational protein modification	1	AR
RNA Polymerase II Transcription	1	AR
Gene expression (Transcription)	1	AR
Transcriptional regulation by RUNX2	1	AR
RUNX2 regulates osteoblast differentiation	1	AR
RUNX2 regulates bone development	1	AR
Cellular responses to stimuli	1	AR
Signaling by Rho GTPases, Miro GTPases and RHOBTB3	1	AR

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	1
MAPK cascade	1
in utero embryonic development	1
regulation of systemic arterial blood pressure	1
epithelial cell morphogenesis	1
transcription by RNA polymerase II	1
signal transduction	1
cell-cell signaling	1
spermatogenesis	1
single fertilization	1
positive regulation of cell population proliferation	1
negative regulation of cell population proliferation	1
male gonad development	1
positive regulation of gene expression	1
male somatic sex determination	1

Indications & clinical

Indications

40 indications (9 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
orchitis	4	MONDO:0006882	EFO:1001078
Klinefelter syndrome	4	MONDO:0006823	EFO:1001006
hypogonadism	4	MONDO:0002146	HP:0000044
neoplasm	4	MONDO:0005070	EFO:0000616
metabolic syndrome X	3	MONDO:0011565	EFO:0000195
infertility disorder	3	MONDO:0005047	EFO:0000545
erectile dysfunction	3	MONDO:0005362	EFO:0004234
coronary artery disorder	3	MONDO:0005010	EFO:0001645
hip fracture	3	MONDO:0005327	EFO:0003964
disorder of sexual differentiation	3	MONDO:0002145	Orphanet:325690
chronic kidney disease	3	MONDO:0005300	MONDO:0024327
heart failure	2	MONDO:0005252	EFO:0003144
prostate carcinoma	2	MONDO:0005159	EFO:0001663
obesity disorder	2	MONDO:0011122	EFO:0001073
AIDS	2	MONDO:0012268	EFO:0000765
testicular cancer	2	MONDO:0005447	EFO:0005088
hereditary breast ovarian cancer syndrome	2	MONDO:0003582	Orphanet:145
metastatic prostate carcinoma	2	MONDO:0004956	EFO:0000196
multiple sclerosis	2	MONDO:0005301	MONDO:0005301
anorexia nervosa	2	MONDO:0005351	MONDO:0005351
ductal breast carcinoma in situ	2	MONDO:0005023	EFO:0000432
spinal cord injury	2	MONDO:0043797	MONDO:0043797
Kallmann syndrome	1	MONDO:0018800	MONDO:0018800
polycystic ovary syndrome	0	MONDO:0008487	EFO:0000660
cerebral arterial disease	0	MONDO:0006693	EFO:1000859
psychologic dyspareunia	0	MONDO:0001196	HP:0030016

14 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 206.

Phase distribution

Phase	Trials
PHASE2	49
Not specified	41
PHASE4	34
PHASE3	32
PHASE1	27
EARLY_PHASE1	11
PHASE2/PHASE3	8
PHASE1/PHASE2	4

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06733350	PHASE4	RECRUITING	Testosterone Replacement Therapy for the Treatment of Low Testosterone in Hypogonadal Men With Localized Prostate Cancer on Active Surveillance
NCT00009555	PHASE4	COMPLETED	Testosterone for HIV-Positive Men With Reduced Serum Testosterone Levels and Abdominal Fat
NCT00018356	PHASE4	COMPLETED	Physiologic Effects of PRMS & Testosterone in the Debilitated Elderly
NCT00182871	PHASE4	COMPLETED	Testosterone Effects on Bone and Frailty
NCT00304213	PHASE4	WITHDRAWN	Does Testosterone Improve Function in Hypogonadal Older Men
NCT00349362	PHASE4	COMPLETED	Testosterone for Men With Insulin Treated Type 2 Diabetes
NCT00350701	PHASE4	COMPLETED	Effect of Androgel on Type 2 Diabetic Males With Hypogonadism
NCT00355537	PHASE4	COMPLETED	Testosterone Replacement in Diabetes With Vascular Disease (Version 2)
NCT00421460	PHASE4	COMPLETED	The Therapy of Nebido as Mono or in Combination With PDE-5 Inhibitors in Hypogonadal Patients With Erectile Dysfunction
NCT00504712	PHASE4	COMPLETED	Testosterone for Peripheral Vascular Disease
NCT00700024	PHASE4	UNKNOWN	Odense Androgen Study - The Effect of Testim and Training in Hypogonadal Men
NCT00957801	PHASE4	COMPLETED	Anabolic and Inflammatory Responses to Short-Term Testosterone Administration in Older Men
NCT01084369	PHASE4	TERMINATED	Effect of Testosterone on Endothelial Function and Microcirculation in Type 2 Diabetic Patients With Hypogonadism
NCT01123278	PHASE4	COMPLETED	Testosterone Replacement in Metabolic Syndrome and Inflammation
NCT01127659	PHASE4	COMPLETED	Testosterone Replacement in Men With Diabetes and Obesity
NCT01560546	PHASE4	COMPLETED	Testosterone Therapy of Men With Type 2 Diabetes Mellitus (T2DM)
NCT01852994	PHASE4	COMPLETED	Exercise Training and Testosterone Replacement in Heart Failure Patients
NCT01893281	PHASE4	COMPLETED	The Effect of Testosterone Topical Solution (LY900011) in Hypogonadal Men With Suboptimal Response to a Topical Testosterone Gel
NCT02111434	PHASE4	COMPLETED	Visceral Adiposity Index and Triglyceride/High-density Lipoprotein Cholesterol Ratio in the Congenital Hypogonadotropic Hypogonadism and Effect of Testosteron Treatment
NCT02111473	PHASE4	COMPLETED	The Effect of Testosteron Replacement Treatment on the Fibroblast Growth Factor-23, Asymmetric Dimethylarginine and Vitamin D Levels in the Congenital Hypogonadotropic Hypogonadism
NCT02433730	PHASE4	COMPLETED	Testosterone Therapy in Hypogonadal Men Treated With Opioids
NCT02513940	PHASE4	COMPLETED	Influence of Testosterone Administration on Drug-Induced QT Interval Prolongation and Torsades de Pointes
NCT02611154	PHASE4	COMPLETED	Intranasal Delivery of Testosterone and Its Effect on Doping Markers
NCT02715232	PHASE4	UNKNOWN	Effects of Sex Steroids on the Serotonin System
NCT02799277	PHASE4	COMPLETED	A Multinomial Process Model of Moral Judgment
NCT03091348	PHASE4	COMPLETED	Subcutaneous vs. Intramuscular Testosterone
NCT03203681	PHASE4	COMPLETED	Natesto Effects on Testosterone, Luteinizing Hormone, Follicle Stimulating Hormone and Semen Parameters
NCT03619330	PHASE4	COMPLETED	Testosterone Replacement Therapy and Liraglutide Effects on Weight Loss in Hypogonadism.
NCT04274894	PHASE4	COMPLETED	A Study of the Effect of Topical Testosterone Replacement Therapy on Blood Pressure in Adult Male Participants With Hypogonadism
NCT04439799	PHASE4	COMPLETED	A Comparison of Side Effects in Hypogonadal Men Treated With Natesto Versus Testosterone Injections
NCT04456296	PHASE4	COMPLETED	A Study of the Effect of Testosterone Replacement Therapy on Blood Pressure in Adult Male Participants With Hypogonadism
NCT04558567	PHASE4	COMPLETED	Vogelxo(R) ABPM Study in Hypogandal Men
NCT04675788	PHASE4	COMPLETED	Novel Approaches for Minimizing Drug-Induced QT Interval Lengthening
NCT04976595	PHASE4	UNKNOWN	A Study to Monitor Ambulatory Blood Pressure Monitoring in Hypogonadal Men Treated With Nasal Testosterone Gel
NCT07260825	PHASE2/PHASE3	NOT_YET_RECRUITING	Use of Topical Testosterone and Estrogen vs Estrogen Alone in Vulvodynia: a Randomized Controlled Trial
NCT00075855	PHASE3	COMPLETED	Low-Dose Testosterone in Improving Libido in Postmenopausal Female Cancer Survivors
NCT00089843	PHASE2/PHASE3	COMPLETED	Bone Loss in Women With Anorexia Nervosa
NCT00244023	PHASE3	COMPLETED	Co-Administering Testosterone With PDE5 Inhibitors in ED Patients Non Responders to PDE5 Inhibitors Alone
NCT00331123	PHASE3	COMPLETED	Study of Transdermal Testosterone Patches in Surgically Menopausal Women With Low Libido
NCT00331214	PHASE3	COMPLETED	Study of Transdermal Testosterone Patches in Surgically Menopausal Women With Low Libido

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 8 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

129 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
MEGESTROL	ChEMBL + PubChem	Phase 4 (approved)	AR
ABIRATERONE	ChEMBL	Phase 4 (approved)	AR
APALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	AR
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	AR
BETAMETHASONE	ChEMBL	Phase 4 (approved)	AR
BICALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
BITHIONOL	ChEMBL	Phase 4 (approved)	AR
BROMHEXINE	ChEMBL	Phase 4 (approved)	AR
BUDESONIDE	ChEMBL	Phase 4 (approved)	AR
CHLORMADINONE	ChEMBL	Phase 4 (approved)	AR
CLARITHROMYCIN	ChEMBL	Phase 4 (approved)	AR
CLASCOTERONE	ChEMBL	Phase 4 (approved)	AR
CLOCORTOLONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
CLOMIPHENE	ChEMBL	Phase 4 (approved)	AR
CORTISONE	ChEMBL	Phase 4 (approved)	AR
CYCLOFENIL	ChEMBL	Phase 4 (approved)	AR
DAROLUTAMIDE	ChEMBL	Phase 4 (approved)	AR
DESOGESTREL	ChEMBL	Phase 4 (approved)	AR
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	AR
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	AR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	AR
DIFLORASONE DIACETATE	ChEMBL	Phase 4 (approved)	AR
DORZOLAMIDE	ChEMBL	Phase 4 (approved)	AR
DROSPIRENONE	ChEMBL	Phase 4 (approved)	AR
DYDROGESTERONE	ChEMBL	Phase 4 (approved)	AR
ENZALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
EPLERENONE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL CYPIONATE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL VALERATE	ChEMBL	Phase 4 (approved)	AR
ESTRIOL	ChEMBL	Phase 4 (approved)	AR
ESTRONE	ChEMBL	Phase 4 (approved)	AR
ETHINYL ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ETHYNODIOL DIACETATE	ChEMBL	Phase 4 (approved)	AR
ETONOGESTREL	ChEMBL	Phase 4 (approved)	AR
FLUMETHASONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
FLUOCINOLONE ACETONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOCINONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOXYMESTERONE	ChEMBL	Phase 4 (approved)	AR
FLURANDRENOLIDE	ChEMBL	Phase 4 (approved)	AR
FLUTAMIDE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE FUROATE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HALCINONIDE	ChEMBL	Phase 4 (approved)	AR
HALOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HEXACHLOROPHENE	ChEMBL	Phase 4 (approved)	AR
HEXESTROL	ChEMBL	Phase 4 (approved)	AR
HYDROCORTISONE	ChEMBL	Phase 4 (approved)	AR
INDOMETHACIN	ChEMBL	Phase 4 (approved)	AR
LEVONORGESTREL	ChEMBL	Phase 4 (approved)	AR
MEDROXYPROGESTERONE	ChEMBL	Phase 4 (approved)	AR
METHYLPREDNISOLONE	ChEMBL	Phase 4 (approved)	AR
MIFEPRISTONE	ChEMBL	Phase 4 (approved)	AR
MOMETASONE FUROATE	ChEMBL	Phase 4 (approved)	AR
NILUTAMIDE	ChEMBL	Phase 4 (approved)	AR
NOMEGESTROL	ChEMBL	Phase 4 (approved)	AR
NORETHINDRONE	ChEMBL	Phase 4 (approved)	AR
NORETHYNODREL	ChEMBL	Phase 4 (approved)	AR
OXANDROLONE	ChEMBL	Phase 4 (approved)	AR