Zalcitabine

Summary

Zalcitabine (CHEMBL853) is an approved small-molecule antiviral drug (ATC J05AF03); indicated across 2 conditions including viral infectious disease and hiv infectious disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J05AF03
• Indications: 2 conditions
• Clinical trials: 39
• Chemistry: 211.22 Da · C9H13N3O3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL853
Name	Zalcitabine
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	24066
ChEBI	CHEBI:10101
ATC	J05AF03
Molecular formula	C9H13N3O3
Molecular weight	211.22
InChIKey	WREGKURFCTUGRC-POYBYMJQSA-N

SMILES: C1C[C@@H](O[C@@H]1CO)N2C=CC(=NC2=O)N

IUPAC name: 4-amino-1-[(2R,5S)-5-(hydroxymethyl)oxolan-2-yl]pyrimidin-2-one

ChEBI definition: A pyrimidine 2’,3’-dideoxyribonucleoside compound having cytosine as the nucleobase.

Pharmacological roles (ChEBI): antiviral drug, HIV-1 reverse transcriptase inhibitor.

Other ChEBI roles (chemical / environmental): antimetabolite.

Also known as: Ddc (ddc), Dideoxycytidine, Hivid, NSC-606170, RO-242027000, Zalcitabina, Zalcitabine, Zalcitabine (dideoxycytidine,ddc), dideoxycytidine, 2’,3’-di-deoxycytidine, 2’,3’-dideoxycytidine, SID11532889

Patent coverage: 11,076 distinct patent families (41,219 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: 8 (assay-derived). Sample: Microtubule-associated protein tau, Prelamin-A/C, RecQ-like DNA helicase BLM, Menin/Histone-lysine N-methyltransferase MLL, C-X-C chemokine receptor type 4, Nuclear factor NF-kappa-B p105 subunit, Albumin, Mitogen-activated protein kinase 1.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
CXCR4	7.06	EC50	88	nM	CHEMBL_ACT_989924
LMNA	7	Potency	100	nM	CHEMBL_ACT_3660636
MAPT	6.9	Potency	125.9	nM	CHEMBL_ACT_4525583
NFKB1	6.1	Potency	794.3	nM	CHEMBL_ACT_3671853
NFKB1	6.1	Potency	794.3	nM	CHEMBL_ACT_4589001

Target pathways

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

Indications & clinical

Indications

2 indications (1 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
viral infectious disease	4	MONDO:0005108	EFO:0000763
HIV infectious disease	3	MONDO:0005109	EFO:0000764

Clinical trials

Total trials: 39.

Phase distribution

Phase	Trials
PHASE2	16
Not specified	9
PHASE3	7
PHASE1	6
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00002386	PHASE4	COMPLETED	Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
NCT00000651	PHASE3	COMPLETED	A Randomized, Double Blind, Comparative Study of Dideoxycytidine (ddC) Alone or ddC/AZT Combination Versus Zidovudine (ZDV) Alone in Patients With HIV Infection Who Have Received Prior ZDV Therapy
NCT00000682	PHASE3	COMPLETED	A Randomized, Unblinded Trial of Zidovudine Versus ddC in the Treatment of Patients Status Post PCP Who Received Long-Term Zidovudine Therapy in Protocol ACTG 002
NCT00001022	PHASE3	COMPLETED	A Comparison of Zidovudine (AZT) Used Alone or in Combination With Didanosine (ddI) or Dideoxycytidine (ddC) in HIV-Infected Patients
NCT00002109	PHASE3	COMPLETED	Double-Blind Study of Timunox (Thymopentin) in Asymptomatic HIV-Infected Patients Receiving Either Mono (AZT or ddI) or Combination (AZT / ddI or AZT / ddC) Anti-Retroviral Therapy
NCT00002117	PHASE3	COMPLETED	A Randomized, Open-Label Study of Alternative Treatment Combinations of Dideoxycytidine (HIVID; ddC) and Zidovudine (AZT) in Patients With HIV Infection
NCT00002334	PHASE3	COMPLETED	A Study of Zidovudine (AZT) Used Alone or in Combination With Other Anti-HIV Drugs in HIV-Infected Patients With Little or No Previous Treatment
NCT00002436	PHASE3	COMPLETED	The Safety and Effectiveness of Three Anti-HIV Drug Combinations in HIV-Infected Patients Who Have Taken AZT
NCT00000625	PHASE2	COMPLETED	A Randomized, Double-Blind Phase II/III Trial of Monotherapy vs. Combination Therapy With Nucleoside Analogs in HIV-Infected Persons With CD4 Cells of 200-500/mm3
NCT00000653	PHASE2	COMPLETED	A Trial of Two Doses of 2’,3’-Dideoxycytidine (ddC) in the Treatment of Children With Symptomatic HIV Infection Who Are Intolerant of AZT and/or Who Show Progressive Disease While on AZT
NCT00000678	PHASE2	COMPLETED	Dideoxycytidine ( Ro 24-2027 ) A Randomized, Open-Label, Comparative Study of Dideoxycytidine ( ddC ) Versus Zidovudine ( AZT ) in Patients With AIDS or Advanced ARC Who Have Received Long-Term AZT Therapy.
NCT00000679	PHASE2	COMPLETED	(Ro 24-2027) A Randomized, Double-Blind, Comparative Study of Dideoxycytidine (ddC) Versus Zidovudine (AZT) in Patients With AIDS or Advanced ARC
NCT00000754	PHASE2	COMPLETED	A Randomized Phase II Study of Two Doses of Interferon Alfa-2a (IFN Alfa-2a) in Combination With Zidovudine (AZT) and Dideoxycytidine (ddC) Versus AZT and ddC Only in Patients With HIV Infection and Less Than 400 CD4 Cells/mm3
NCT00000781	PHASE2	COMPLETED	A Randomized, Double-Blind, Four-Arm Study Comparing Combination Nucleoside, Alternating Nucleoside, and Triple-Drug Therapy for the Treatment of Advanced HIV Disease (CD4 <= 50/mm3)
NCT00001029	PHASE2	COMPLETED	A Comparison of Three Treatments for Advanced HIV Disease in Patients Who Have Received Nucleoside Therapy in the Past
NCT00001032	PHASE2	COMPLETED	A Study of Dideoxycytidine Plus Zidovudine (AZT) in HIV-Infected Children Who Have Taken AZT
NCT00001040	PHASE2	COMPLETED	Comparison of Ro 31-8959 Plus Zidovudine (AZT) Versus AZT Plus Zalcitabine (ddC) Versus Ro 31-8959 Plus AZT Plus ddC
NCT00001048	PHASE2	COMPLETED	Comparison of Anti HIV Drugs Used Alone or in Combination With Cytosine Arabinoside to Treat Progressive Multifocal Leukoencephalopathy (PML) in HIV-Infected Patients
NCT00001087	PHASE2	COMPLETED	The Effectiveness of Nelfinavir and Efavirenz, Used Alone or Together, Combined With Other Anti-HIV Drugs in Patients Who Have Taken Anti-HIV Drugs
NCT00002086	PHASE2	COMPLETED	A Randomized Trial to Evaluate the Safety and Efficacy of Combination Therapy With Retrovir ( AZT ) and HIVID ( ddC ) Versus Retrovir, HIVID, and Wellferon ( Interferon Alfa-n1 ) for the Treatment of HIV Infection
NCT00002162	PHASE2	COMPLETED	A Randomized, Parallel Arm, Comparative, Open Label, Multicenter Study of the Activity and Safety of Two Formulations of Saquinavir in Combination With Other Antiretroviral Drugs
NCT00002265	PHASE2	COMPLETED	An Open-Label, Multicenter Study to Evaluate the Safety and Tolerability of Dideoxycytidine (ddC) in Patients With AIDS or Advanced ARC Who Previously Demonstrated Intolerance to Zidovudine (AZT) in Protocol N3300 or N3492
NCT00002333	PHASE2	COMPLETED	A Study of Saquinavir and Zalcitabine, Used Alone and Together, in the Treatment of Advanced HIV Infection in Patients Who Stopped Taking or Who Cannot Take Zidovudine
NCT00002347	PHASE2	COMPLETED	The Safety and Effectiveness of Retrovir Plus HIVID Combined With Either Nevirapine or Invirase in the Treatment of HIV Infection
NCT00000704	PHASE1	COMPLETED	A Multicenter Dose Ranging Clinical Trial of 2’,3’-Dideoxycytidine in the Treatment of Patients With AIDS and Advanced ARC.
NCT00000753	PHASE1	COMPLETED	A Phase I Concentration-Targeted Multidose Study of Atevirdine Mesylate ( U-87201E ), AZT, and ddI or ddC
NCT00000902	PHASE1	COMPLETED	A Study on the Management of Combination Anti-HIV Drug Therapy in HIV-Positive Children With Prior Treatment
NCT00000954	PHASE1	COMPLETED	A Study of Chemotherapy Plus ddI or ddC in the Treatment of AIDS-Related Kaposi’s Sarcoma
NCT00000978	PHASE1	COMPLETED	A Study of Dideoxycytidine Plus Zidovudine in the Treatment of AIDS or Advanced AIDS Related Complex (ARC)
NCT00001035	PHASE1	COMPLETED	The Safety and Effectiveness of a Two-Drug Combination in the Treatment of Patients With Hepatitis C Plus Advanced HIV Infections
NCT00000718	Not specified	COMPLETED	A Clinical Trial of Alternating and Intermittent Regimens of 2’,3’-Dideoxycytidine and 3’-Azido-3’-Deoxythymidine in the Treatment of Patients With AIDS and Advanced ARC
NCT00000719	Not specified	COMPLETED	A Trial of Alternating 2’,3’-Dideoxycytidine and Zidovudine in the Treatment of Patients With Advanced HIV Disease
NCT00000969	Not specified	COMPLETED	A Prospective, Randomized, Open-Label, Comparative Trial of Dideoxyinosine (ddI) Versus Dideoxycytidine (ddC) in HIV-Infected Patients Who Are Intolerant of or Who Have Failed Zidovudine (AZT) Therapy
NCT00000997	Not specified	COMPLETED	A Study of Dideoxycytidine in HIV-Infected Patients
NCT00002001	Not specified	COMPLETED	The Antiviral Efficacy of Concurrent Zidovudine and 2’,3’-Dideoxyinosine or 2’,3’-Dideoxycytidine in Patients With Human Immunodeficiency Virus Disease
NCT00002081	Not specified	COMPLETED	Open-Label Program of Dideoxycytidine ( ddC ) to Be Used in Combination With Zidovudine ( AZT ) for Treatment of Advanced HIV Disease
NCT00002118	Not specified	COMPLETED	An Open-Label Safety Program for the Use of Zalcitabine (Dideoxycytidine; ddC) in Pediatric Patients With Symptomatic HIV Infection Who Have Failed or Are Intolerant to AZT Monotherapy, or Who Have Completed Other ddC Protocols, or Are Ineligible for Other Ongoing Clinical Studies
NCT00002256	Not specified	COMPLETED	Dideoxycytidine ( Ro 24-2027 ). A Treatment Protocol for the Use of Dideoxycytidine ( ddC ) in Patients With AIDS or Advanced ARC Who Cannot Be Maintained on Zidovudine ( AZT ) Therapy.
NCT00002279	Not specified	COMPLETED	A Study of ddC in Patients With AIDS or Advanced AIDS-Related Complex (ARC) Who Have Not Had Success With Zidovudine (AZT)

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

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