Megestrol Acetate

Summary

Megestrol Acetate (CHEMBL1201139) is an approved small-molecule antineoplastic agent (ATC L02AB01); indicated across 21 conditions including neoplasm and aids.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L02AB01 (+2 more)
• Indications: 21 conditions
• Clinical trials: 78
• Chemistry: 384.5 Da · C24H32O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201139
Name	Megestrol Acetate
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	11683
ChEBI	CHEBI:6723
ATC	L02AB01, G03DB02, G03AC05
Molecular formula	C24H32O4
Molecular weight	384.5
InChIKey	RQZAXGRLVPAYTJ-GQFGMJRRSA-N

SMILES: CC1=C[C@@H]2[C@H](CC[C@]3([C@H]2CC[C@@]3(C(=O)C)OC(=O)C)C)[C@@]4(C1=CC(=O)CC4)C

IUPAC name: [(8R,9S,10R,13S,14S,17R)-17-acetyl-6,10,13-trimethyl-3-oxo-2,8,9,11,12,14,15,16-octahydro-1H-cyclopenta[a]phenanthren-17-yl] acetate

ChEBI definition: A steroid ester resulting from the formal condensation of the hydroxy group of megestrol with the carboxy group of acetic acid. It is an appetite stimulant used for the treatment of anorexia and cachexia. Also used for birth control and for the treatment of breast cancer.

Pharmacological roles (ChEBI): antineoplastic agent, appetite enhancer, contraceptive drug, progestin, synthetic oral contraceptive.

Also known as: BDH 1298, BDH-1298, Chronopil, Megace, Megace es, Megesterol acetate, Megestrol, Megestrol 17.alpha.-acetate, Megestrol acetate, NSC-71423, SC 10363, SC-10363

Patent coverage: 30,434 distinct patent families (98,000 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

Broader ChEMBL bioactivity targets: 13 (assay-derived). Sample: Survival motor neuron protein, Prelamin-A/C, Androgen receptor, Glucocorticoid receptor, Progesterone receptor, DNA polymerase beta, Androgen receptor, Cytochrome P450 2C9, Gamma-aminobutyric acid receptor subunit alpha-1, Cruzipain.

Bioactivity

ChEMBL activities: 14 potent at pChembl ≥ 5 of 17 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PGR	8.52	AC50	3	nM	CHEMBL_ACT_25204422
NR3C1	8.06	Ki	8.77	nM	CHEMBL_ACT_7753492
AR	7.74	AC50	18.1	nM	CHEMBL_ACT_25203489
NR3C1	7.72	IC50	19	nM	CHEMBL_ACT_7753491
P15207	7.7	Ki	20	nM	CHEMBL_ACT_7755671
P15207	7.52	IC50	30	nM	CHEMBL_ACT_7755670
SMN1	5.9	Potency	1259	nM	CHEMBL_ACT_3904720
NPSR1	5.5	Potency	3162	nM	CHEMBL_ACT_4921294
NPSR1	5.4	Potency	3981	nM	CHEMBL_ACT_4893448
P62813	5.17	AC50	6687	nM	CHEMBL_ACT_25207965
HIF1A	5.1	Potency	7943	nM	CHEMBL_ACT_4129004
HIF1A	5.1	Potency	7943	nM	CHEMBL_ACT_4520826
LMNA	5.05	Potency	8912	nM	CHEMBL_ACT_3655746
CYP2C9	5	IC50	10000	nM	CHEMBL_ACT_7751448

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

21 indications (6 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
neoplasm	4	MONDO:0005070	EFO:0000616
AIDS	4	MONDO:0012268	EFO:0000765
endometrium neoplasm	3	MONDO:0021251	EFO:0004230
breast neoplasm	3	MONDO:0021100	EFO:0003869
endometrial carcinoma	3	MONDO:0002447	EFO:1001512
uterine corpus cancer	3	MONDO:0006003	EFO:0007532
chronic obstructive pulmonary disease	2	MONDO:0005002	EFO:0000341
head and neck cancer	2	MONDO:0005627	EFO:0006859
HIV wasting syndrome	2	MONDO:0005797	EFO:0007312
dementia	2	MONDO:0001627	HP:0000726
in situ carcinoma	2	MONDO:0004647	MONDO:0024474
uterine cancer	2	MONDO:0002715	MONDO:0002715
adrenal cortex carcinoma	2	MONDO:0006639	EFO:1000796

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

Clinical trials

Total trials: 78.

Phase distribution

Phase	Trials
PHASE2	31
PHASE3	16
PHASE2/PHASE3	9
PHASE1	9
PHASE4	6
Not specified	5
PHASE1/PHASE2	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07254416	PHASE4	NOT_YET_RECRUITING	A Study on Nausea and Vomiting Caused by T-DXd in Breast Cancer Patients
NCT00002345	PHASE4	COMPLETED	The Safety and Effectiveness of Megace in HIV-Infected Women
NCT00163072	PHASE4	WITHDRAWN	Pharmacokinetics and Safety of Transdermal Megestrol Acetate
NCT03777930	PHASE4	UNKNOWN	The Evaluation of Curative Effect on Treatment of Tumor Above Thalidomide Combined With Megestrol
NCT03815019	PHASE4	COMPLETED	iKanEat: A Randomized-controlled, Multi-center Trial of Megestrol for Chronic Oral Food Refusal in Children
NCT03963752	PHASE4	COMPLETED	Clinical Trial of Rapid Progressive Central Precocious Puberty With Integrative Chinese and Western Medicine
NCT04939090	PHASE3	ACTIVE_NOT_RECRUITING	Olanzapine Versus Megestrol Acetate for the Treatment of Loss of Appetite Among Advanced Cancer Patients
NCT05255653	PHASE2/PHASE3	RECRUITING	Refining Adjuvant Treatment IN Endometrial Cancer Based On Molecular Features
NCT06500234	PHASE3	RECRUITING	Nutrition Impact on Immunotherapy of Cancer
NCT06998758	PHASE3	RECRUITING	Megestrol Acetate in Improving Neoadjuvant Chemotherapy-Related Weight Loss in Locally Advanced CRC
NCT07319429	PHASE2/PHASE3	RECRUITING	Refining Fertility-sparing Treatment in Endometrial Carcinoma Based on Molecular Classification
NCT07543536	PHASE3	NOT_YET_RECRUITING	Megestrol Acetate for Fatigue Management in T-DXd Treated Breast Cancer
NCT00002067	PHASE3	COMPLETED	A Study Comparing Megestrol Acetate at 800 mg/Day, and Placebo in AIDS Patients With Anorexia and Cachexia
NCT00005975	PHASE3	COMPLETED	S9626: Megestrol in Treating Hot Flashes Following Treatment for Breast Cancer
NCT00006799	PHASE3	COMPLETED	Megestrol to Limit Weight Loss and Improve Quality of Life in Treating Patients With Head and Neck Cancer
NCT00016341	PHASE3	TERMINATED	Combination Chemotherapy Compared With Hormone Therapy in Treating Patients With Recurrent, Stage III, or Stage IV Endometrial Cancer
NCT00031707	PHASE3	COMPLETED	Comparison of Megestrol and/or Omega-3 Fatty Acid-Enriched Nutritional Supplement in Treating Patients With Cancer-Related Weight Loss and Lack of Appetite
NCT00031785	PHASE3	COMPLETED	Megestrol in Treating Patients Who Are Undergoing Radiation Therapy for Lung Cancer
NCT00041275	PHASE3	COMPLETED	Megestrol in Treating Patients With Liver Cancer That Cannot Be Removed By Surgery
NCT00070148	PHASE3	COMPLETED	Oxandrolone Compared With Megestrol in Preventing Weight Loss in Patients Receiving Chemotherapy for Cancer
NCT00503516	PHASE2/PHASE3	TERMINATED	Study to Evaluate the Effect of Megestrol Acetate in Weight Loss in Dementia Patients
NCT00637728	PHASE3	TERMINATED	Treatment of Cancer-anorexia Using Megestrol Acetate Concentrated Suspension in Lung or Pancreatic Cancer Patients
NCT00637806	PHASE3	TERMINATED	Treatment of Cancer-associated Anorexia Using Megestrol Acetate Concentrated Suspension
NCT01237327	PHASE3	COMPLETED	Multicenter Follow Up Study Of Subjects Who Participated In An Original Protocol Of Exemestane Vs. Megestrol Acetate In Postmenopausal Women With Metastatic Breast Cancer
NCT02619266	PHASE2/PHASE3	UNKNOWN	The Safety and Effect Study of Acupuncture for Anorexia in Patients With Gastrointestinal Tract and Lung Cancers
NCT03241888	PHASE2/PHASE3	COMPLETED	Megestrol Acetate Plus LNG-IUS in Young Women With Endometrial Atypical Hyperplasia
NCT03241914	PHASE2/PHASE3	COMPLETED	Megestrol Acetate Plus LNG-IUS in Young Women With Early Endometrial Cancer
NCT04491682	PHASE2/PHASE3	COMPLETED	Megestrol Acetate Plus Rosuvastatin in Young Women With Atypical Endometrial Hyperplasia
NCT04607252	PHASE2/PHASE3	TERMINATED	Metformin Plus Megestrol Acetate As a Fertility-sparing Treatment in Patients with Atypical Endometrial Hyperplasia
NCT04683237	PHASE2/PHASE3	WITHDRAWN	Liraglutide Plus Megestrol Acetate in Endometrial Atypical Hyperplasia
NCT04897217	PHASE3	WITHDRAWN	Levonorgestrel-Releasing Intrauterine System (LNG-IUS) in the Management of Atypical Endometrial Hyperplasia
NCT03671811	PHASE2	ACTIVE_NOT_RECRUITING	Megestrol Acetate With or Without Pterostilbene in Treating Patients With Endometrial Cancer Undergoing Hysterectomy
NCT04576104	PHASE2	ACTIVE_NOT_RECRUITING	Megestrol Acetate Compared With Megestrol Acetate and Metformin to Prevent Endometrial Cancer
NCT05538897	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Testing the Addition of the AKT Inhibitor, Ipatasertib, to Treatment With the Hormonal Agent Megestrol Acetate for Recurrent or Metastatic Endometrial Cancers
NCT06979596	PHASE2	RECRUITING	A Study of MK-5684 in People With Certain Solid Tumors (MK-5684-015/OMAHA-015)
NCT07391826	PHASE2	NOT_YET_RECRUITING	Megestrol Acetate Oral Suspension for Cachexia in Patients With cStage III Gastric/Gastroesophageal Junction Adenocarcinoma Receiving Neoadjuvant Therapy With Serplulimab Combined With SOX
NCT07436793	PHASE2	NOT_YET_RECRUITING	Progestins for the Treatment of Endometrial Cancer or Precancers of the Uterus Before Surgery, The Pro-Window Trial
NCT00001079	PHASE2	COMPLETED	A Study of Megestrol Acetate Alone or in Combination With Testosterone Enanthate Drug in the Treatment of HIV-Associated Weight Loss
NCT00002300	PHASE2	COMPLETED	A Study of Different Doses of Megestrol Acetate in Patients With AIDS Who Have Anorexia and Malnutrition
NCT00002465	PHASE1/PHASE2	UNKNOWN	High-Dose Megestrol in Treating Patients With Metastatic Breast Cancer, Endometrial Cancer, or Mesothelioma

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).