Zidovudine

Summary

Zidovudine (CHEMBL129) is an approved small-molecule antiviral drug (ATC J05AF01); indicated across 21 conditions including hiv infectious disease and viral infectious disease.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J05AF01
• Indications: 21 conditions
• Clinical trials: 354
• Chemistry: 267.24 Da · C10H13N5O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL129
Name	Zidovudine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	35370
ChEBI	CHEBI:10110
ATC	J05AF01
Molecular formula	C10H13N5O4
Molecular weight	267.24
InChIKey	HBOMLICNUCNMMY-XLPZGREQSA-N

SMILES: CC1=CN(C(=O)NC1=O)[C@H]2C[C@@H]([C@H](O2)CO)N=[N+]=[N-]

IUPAC name: 1-[(2R,4S,5S)-4-azido-5-(hydroxymethyl)oxolan-2-yl]-5-methylpyrimidine-2,4-dione

ChEBI definition: A pyrimidine 2’,3’-dideoxyribonucleoside compound having a 3’-azido substituent and thymine as the nucleobase.

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

Other ChEBI roles (chemical / environmental): antimetabolite.

Also known as: 3’-azt, Azidothymidine, AZT, BW A509U, BW-A-509U, BW-A509U, Bwa509u, COMPOUND S, COMPOUND-S, NSC-602670, Retrovir, Zidovudina

Patent coverage: 2,128 distinct patent families (4,996 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: 6 (assay-derived). Sample: Thymidine kinase, Pyruvate kinase PKM, 4’-phosphopantetheinyl transferase ffp, Thymidylate kinase, Thymidine kinase, cytosolic, Albumin.

Bioactivity

ChEMBL activities: 3 potent at pChembl ≥ 5 of 11 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
TK1	5.54	Ki	2900	nM	CHEMBL_ACT_833080
TK1	5.52	IC50	3000	nM	CHEMBL_ACT_3287341
TK1	5.16	Ki	7000	nM	CHEMBL_ACT_833079

Target pathways

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

Indications & clinical

Indications

3 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
HIV infectious disease	4	MONDO:0005109	EFO:0000764
viral infectious disease	4	MONDO:0005108	EFO:0000763
autoimmune disease	4	MONDO:0007179	MONDO:0021094

13 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
AIDS	3	MONDO:0012268	EFO:0000765
AIDS dementia complex	3	MONDO:0020689	EFO:0002608
AIDS related complex	3	MONDO:0005639	EFO:0007137
diarrheal disease	3	MONDO:0001673	HP:0002014
pneumocystosis	3	MONDO:0019121	EFO:0007448
severe acute respiratory syndrome	3	MONDO:0005091	MONDO:0100096
tuberculosis	3	MONDO:0018076	MONDO:0018076
Kaposi’s sarcoma	2	MONDO:0005055	EFO:0000558
lymphoma	2	MONDO:0005062	EFO:0000574
tropical spastic paraparesis	2	MONDO:0008039	EFO:0007527
primary biliary cholangitis	2	MONDO:0005388	EFO:1001486
Castleman disease	2	MONDO:0015564	MONDO:0015564
severe combined immunodeficiency	1	MONDO:0015974	MONDO:0015974

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

Clinical trials

Total trials: 354.

Phase distribution

Phase	Trials
PHASE2	88
Not specified	88
PHASE3	73
PHASE1	70
PHASE4	26
PHASE2/PHASE3	5
PHASE1/PHASE2	2
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00002179	PHASE4	COMPLETED	The Effectiveness of Indinavir Plus Zidovudine Plus Lamivudine in HIV-Infected Patients With No Symptoms of Infection
NCT00002376	PHASE4	COMPLETED	The Effectiveness of HIV RNA Viral Load Testing in Determining Treatment Type in HIV-Infected Patients
NCT00002386	PHASE4	COMPLETED	Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
NCT00004585	PHASE4	COMPLETED	A Study of the Safety and Effectiveness of Combination Anti-HIV Therapy in HIV-Infected Adults
NCT00005000	PHASE4	UNKNOWN	Treatment With Nelfinavir or Efavirenz of HIV-Infected Patients Who Have Never Received Anti-HIV Drugs
NCT00005106	PHASE4	COMPLETED	A Comparison of Three Anti-HIV Drug Combinations in HIV-Infected Patients
NCT00005764	PHASE4	COMPLETED	A Study of Increased Lactic Acid and Abnormal Fat Distribution in HIV-Positive Patients
NCT00038506	PHASE4	COMPLETED	Study Of Investigational Regimen Combining FDA Approved HIV Drugs In HIV Subjects Experiencing Early Virologic Failure
NCT00084136	PHASE4	COMPLETED	Prospective Evaluation of Anti-retroviral Combinations for Treatment Naive, HIV Infected Persons in Resource-limited Settings
NCT00127959	PHASE4	COMPLETED	Virological and Clinical Anti-Hepatitis B Virus (HBV) Efficacy of Tenofovir and Emtricitabine in Patients With HIV/HBV co-Infection
NCT00144157	PHASE4	COMPLETED	Open Label Study of NVP+CBV Treatment in Women Who Have Received sdNVP for the pMTCT of HIV
NCT00162643	PHASE4	UNKNOWN	PI Vs. NNRTI Based Therapy for HIV Advanced Disease
NCT00192660	PHASE4	COMPLETED	HIV Infection And Metabolic Abnormalities Protocol 1 (HAMA001)
NCT00199121	PHASE4	UNKNOWN	A Pilot Study of Triple NtRTI/NsRTI Therapy in Antiretroviral Naive HIV-1 Infected Patients
NCT00324649	PHASE4	COMPLETED	Peripheral Body Fat Distribution After Switching Zidovudine and Lamivudine to Truvada
NCT00342355	PHASE4	COMPLETED	Antiretroviral Therapy for Advanced HIV Disease in South Africa
NCT00385957	PHASE4	COMPLETED	Immunological and Viral Response to Antiretrovirals in HIV Patients With CD4 Cell Count Below 100
NCT00854581	PHASE4	TERMINATED	Zidovudine, Interferon Alfa-2b, PEG-Interferon Alfa-2b in Patients With HTLV-I Associated Adult T-Cell Leukemia/Lymphoma
NCT00865475	PHASE4	COMPLETED	Prevention of Lipoatrophy in Patients Treated With Lopinavir/Ritonavir in Monotherapy Versus ZDV + 3TC + ABC
NCT00986063	PHASE4	COMPLETED	Genotype Based Personalized Prescription of Nevirapine
NCT01618305	PHASE4	COMPLETED	Evaluating the Response to Two Antiretroviral Medication Regimens in HIV-Infected Pregnant Women, Who Begin Antiretroviral Therapy Between 20 and 36 Weeks of Pregnancy, for the Prevention of Mother-to-Child Transmission
NCT01694017	PHASE4	COMPLETED	Economic, Clinical and Quality of Life Assessment in Patients on Antiretroviral Therapy
NCT01772940	PHASE4	COMPLETED	Nevirapine vs Ritonavir-boosted Lopinavir in ART Naive HIV-infected Adults in a Resource Limited Setting
NCT02431975	PHASE4	COMPLETED	Latency and Early Neonatal Provision of Antiretroviral Drugs Clinical Trial
NCT02712801	PHASE4	COMPLETED	Antiretroviral Regime for Viral Eradication in Newborns
NCT03088410	PHASE4	COMPLETED	Study of HIV-Infected and Uninfected Pregnant Woman/Child Dyads in Gaborone, Botswana
NCT02369406	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Early Infant HIV Treatment in Botswana
NCT00000637	PHASE3	COMPLETED	A Randomized Comparative Trial of Zidovudine (AZT) Versus 2’,3’-Dideoxyinosine (ddI) Versus AZT Plus ddI in Symptomatic HIV-Infected Children
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
NCT00000700	PHASE3	COMPLETED	A Multi-Center Clinical Trial To Evaluate Azidothymidine (AZT) in the Treatment of Human Immunodeficiency Virus (HIV) Infection in Patients With AIDS Post First Episode PCP
NCT00000702	PHASE3	COMPLETED	A Multicenter Placebo-Controlled Double-Blind Trial to Evaluate Azidothymidine (AZT) Treatment of the AIDS Dementia Complex and Central Nervous System (CNS) Human Immunodeficiency Virus (HIV) Infection
NCT00000709	PHASE3	COMPLETED	An Open Trial of Zidovudine (AZT) Treatment of the AIDS Dementia Complex in Patients With AIDS or Low CD4+ Lymphocyte Counts
NCT00000720	PHASE3	COMPLETED	A Double-Blind, Placebo-Controlled Trial To Evaluate Intravenous Gamma Globulin in Children With Symptomatic HIV Infection Receiving Zidovudine
NCT00000727	PHASE3	COMPLETED	A Controlled Comparative Trial of Sulfamethoxazole-Trimethoprim Versus Aerosolized Pentamidine for Secondary Prophylaxis of Pneumocystis Carinii Pneumonia in AIDS Patients Receiving Azidothymidine (AZT)
NCT00000736	PHASE3	COMPLETED	Safety and Efficacy of Zidovudine for Asymptomatic HIV-Infected Individuals
NCT00000750	PHASE3	COMPLETED	A Phase III Study to Evaluate the Safety, Tolerance, and Efficacy of Early Treatment With Zidovudine (AZT) in Asymptomatic Infants With HIV Infection
NCT00000751	PHASE3	COMPLETED	A Phase III Randomized, Double-Blind, Controlled Study of the Use of Anti-HIV Immune Serum Globulin (HIVIG) for the Prevention of Maternal-Fetal HIV Transmission in Pregnant Women and Newborns Receiving Zidovudine (AZT)
NCT00000841	PHASE3	COMPLETED	A Study of Indinavir Sulfate Plus Zidovudine (AZT) Plus Lamivudine in HIV-Infected Patients Who Have Taken AZT for Six or More Months
NCT00000903	PHASE3	COMPLETED	Addition of Efavirenz or Nelfinavir to a Lamivudine/Zidovudine/Indinavir HIV Treatment Regimen
NCT00000960	PHASE3	COMPLETED	The Safety and Effectiveness of Zidovudine in HIV-Infected Pregnant Women and Their Infants
NCT00000991	PHASE3	COMPLETED	A Study of Three Drugs Plus Zidovudine in the Prevention of Infections in HIV-Infected Patients
NCT00000994	PHASE3	COMPLETED	A Study of AZT in HIV-Infected Patients With AIDS-Related Kaposi’s Sarcoma
NCT00001011	PHASE3	COMPLETED	The Safety and Effectiveness of Zidovudine in the Treatment of Patients With Early AIDS Related Complex
NCT00001022	PHASE3	COMPLETED	A Comparison of Zidovudine (AZT) Used Alone or in Combination With Didanosine (ddI) or Dideoxycytidine (ddC) in HIV-Infected Patients
NCT00001104	PHASE3	COMPLETED	A Study of Zidovudine in HIV-Infected Patients Who Have Hemophilia
NCT00001267	PHASE3	COMPLETED	A Randomized Pilot Study for the Treatment of AIDS or AIDS Related Complex With an Alternating or Simultaneous Combination Regimen of AZT and 2’,3’-Dideoxyinosine
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
NCT00002124	PHASE3	COMPLETED	A Double-Blind, Randomized, Dose Response Study of Three Doses of Delavirdine Mesylate (U-90152S) in Combination With Zidovudine (ZDV) Versus ZDV Alone in HIV-1 Infected Individuals With CD4 Counts of 200-500mm3
NCT00002195	PHASE3	COMPLETED	A Study of Retrovir and Epivir Alone or in Combination With 141W94 in HIV-Infected Patients
NCT00002199	PHASE3	COMPLETED	A Phase III Randomized Double-Blind, Multicenter Study to Evaluate the Safety and Efficacy of 3TC/ZDV/1592U89 and 3TC/ZDV/IDV in HIV-1 Infected Antiretroviral Therapy-Naive Subjects.
NCT00002208	PHASE3	COMPLETED	A Multicenter, Open-Label, Randomized, 24-Week Study to Compare the Safety and Activity of Indinavir Sulfate, 800 Mg q 8 h Versus 1,200 Mg q 12 h in Combination With Zidovudine and 3TC
NCT00002238	PHASE3	COMPLETED	Phase III Randomized Double-Blind Placebo Controlled Study To Evaluate the Safety and Efficacy of Betaseron in AIDS and Advanced ARC Patients Receiving a Reduced-Dose AZT Regimen
NCT00002320	PHASE3	COMPLETED	A Comparison of Lamivudine and Zidovudine, Used Alone and Together, in HIV-Infected Patients Who Have Not Used Zidovudine in the Past
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
NCT00002367	PHASE3	COMPLETED	A Study of Saquinavir Soft Gelatin Capsules Plus Zidovudine Plus Lamivudine in the Treatment of HIV-1 Infected Patients Who Have Never Taken Anti-HIV Drugs
NCT00002371	PHASE3	COMPLETED	The Safety and Effectiveness of Lamivudine Plus Stavudine or Zidovudine in HIV-Infected Patients Who Have Taken Zidovudine
NCT00002378	PHASE3	COMPLETED	A Comparison of Three Anti-HIV Drug Combinations Containing Saquinavir Soft Gelatin Capsules Used in HIV-1 Infected Patients
NCT00002389	PHASE3	COMPLETED	The Safety and Effectiveness of Lamivudine Plus Zidovudine, Used With and Without 1592U89, in HIV-1 Infected Patients Who Have Never Taken Anti-HIV Drugs
NCT00002391	PHASE3	COMPLETED	The Safety and Effectiveness of Zidovudine Plus Lamivudine, Used With and Without 1592U89, in HIV-1 Infected Children Who Have Taken Anti-HIV-1 Drugs
NCT00002409	PHASE3	COMPLETED	The Effect of Teaching HIV-Infected Patients About HIV and Treatment
NCT00002410	PHASE3	COMPLETED	A Study of Three Different Anti-HIV Drug Combinations in HIV-Infected Patients
NCT00002429	PHASE3	COMPLETED	Evaluation of an Anti-HIV Drug Combination That Includes a Coated Form of Didanosine (ddI EC) Compared to a Typical Anti-HIV Drug Regimen
NCT00002436	PHASE3	COMPLETED	The Safety and Effectiveness of Three Anti-HIV Drug Combinations in HIV-Infected Patients Who Have Taken AZT
NCT00004981	PHASE3	UNKNOWN	Safety and Effectiveness of Three Anti-HIV Drugs Combined in One Pill (Trizivir)
NCT00005002	PHASE3	UNKNOWN	Effectiveness of Treating HIV-Positive Patients With an HIV Vaccine (Remune)
NCT00006396	PHASE3	COMPLETED	Effectiveness of AZT and Nevirapine in Preventing HIV Transmission From Ugandan Mothers to Their Newborns
NCT00013520	PHASE3	COMPLETED	Comparison of Three Different Initial Treatments Without Protease Inhibitors for HIV Infection
NCT00013897	PHASE3	COMPLETED	A Comparison of BMS-232632 With Efavirenz, Each in Combination With Zidovudine-Lamivudine
NCT00017719	PHASE3	COMPLETED	Combination Treatment With and Without Protease Inhibitors for Women Who Begin Therapy for HIV Infection During Pregnancy
NCT00028327	PHASE3	COMPLETED	Potent Antiviral Therapy for Critically Ill HIV Infected Patients Admitted to Intensive Care
NCT00050895	PHASE3	COMPLETED	Comparing the Safety, Effectiveness, and Tolerability of Three Anti-HIV Drug Regimens for Treatment-Naive Patients
NCT00074581	PHASE3	COMPLETED	Preventing Sexual Transmission of HIV With Anti-HIV Drugs
NCT00078247	PHASE3	COMPLETED	Anti-HIV Drugs for Ugandan Patients With HIV and Tuberculosis
NCT00086359	PHASE3	COMPLETED	Comparison of Anti-HIV Drug Combinations to Prevent Mother-to-Child Transmission of HIV
NCT00099359	PHASE3	COMPLETED	Trial of Three Neonatal Antiretroviral Regimens for Prevention of Intrapartum HIV Transmission
NCT00102960	PHASE3	COMPLETED	Anti-HIV Drugs for Treating Infants Who Acquired HIV Infection at Birth
NCT00112047	PHASE3	COMPLETED	Tenofovir Disoproxil Fumarate/Emtricitabine/Efavirenz Versus Combivir/Efavirenz in Antiretroviral-Naive HIV-1 Infected Subjects
NCT00115648	PHASE3	COMPLETED	Extended Infant Post-exposure Prophylaxis With Antiretrovirals to Reduce Postnatal HIV Transmission
NCT00144183	PHASE3	COMPLETED	A Study of Single Dose Nevirapine (NVP) Combined With Combivir® for the Prevention of Mother to Child Transmission (pMTCT) - Treatment Options Preservation Study (TOPS)
NCT00158405	PHASE3	COMPLETED	Randomised Trial of Structured Treatment Interruption of HAART in HIV-Infected Adults in Abidjan (ANRS 1269 TRIVACAN)
NCT00234091	PHASE3	COMPLETED	When to Start Anti-HIV Drugs in Children Infected With HIV (The PREDICT Study)
NCT00234923	PHASE3	COMPLETED	Efficacy and Safety of Kaletra Monotheraphy Compared to Kaletra Based Triple Therapy to Treat HIV in Antiretroviral Naїve Patients
NCT00272480	PHASE2/PHASE3	COMPLETED	Zidovudine Plus Lamivudine in HTLV-I-associated Myelopathy: a Randomised Trial
NCT00323544	PHASE3	COMPLETED	SWEET: Once Daily Truvada Versus Twice Daily Combivir for the Treatment of HIV Infection
NCT00405925	PHASE3	COMPLETED	FREE Study: Efficacy and Toxicity of Trizivir
NCT00409591	PHASE3	TERMINATED	Maternal/Infant Peripartum NVP, Versus Infant Only Peripartum NVP, or Maternal LPV/r in Addition to Standard ZDV Prophylaxis for the Prevention of Perinatal (PMTCT) HIV in Thailand
NCT00427297	PHASE3	TERMINATED	Optimizing Pediatric HIV-1 Treatment in Infants With Prophylactic Exposure to Nevirapine, Nairobi, Kenya
NCT00528957	PHASE3	COMPLETED	Safety and Efficacy of Switching From Stavudine or Zidovudine to Tenofovir DF in HIV-1 Infected Children
NCT00650637	PHASE3	TERMINATED	A Study to Determine Effective Prophylaxis and Treatment of Nelfinavir-Associated Diarrhea
NCT00669487	PHASE3	COMPLETED	A 72-week Randomized Clinical Trial Comparing the Safety and Efficacy of Three Initial Antiretroviral Regimens -GPO-VIR S (d4T/3TC/NVP) for 24 Weeks Followed by GPO-VIR Z (AZT/3TC/NVP) vs GPO-VIR Z vs TDF/FTC/NVP
NCT00834457	PHASE2/PHASE3	COMPLETED	A Pilot Study Of the Effects of Highly Active Antiretroviral Therapy on Kaposi’s Sarcoma in Zimbabwe
NCT00966160	PHASE3	COMPLETED	CD4 Cell Recovery in HIV-1 Patients Comparing 2 Treatment Regimes
NCT00993031	PHASE3	COMPLETED	Protease Inhibitors to Reduce Malaria Morbidity in HIV-Infected Pregnant Women
NCT01061151	PHASE3	COMPLETED	Evaluating Strategies to Reduce Mother-to-Child Transmission of HIV Infection in Resource-Limited Countries
NCT01352715	PHASE3	COMPLETED	Study of Options for Second-Line Effective Combination Therapy (SELECT)
NCT01511237	PHASE3	COMPLETED	Perinatal Antiretroviral Intensification for PMTCT of HIV in Late Comers
NCT01516970	PHASE3	COMPLETED	Human Immunodeficiency Virus (HIV) Postexposure Prophylaxis (PEP) With Darunavir/Ritonavir (DRV/r)
NCT01540240	PHASE2/PHASE3	UNKNOWN	Safety of Reduced Dose Zidovudine (AZT) Compared With Standard Dose AZT in Antiretroviral-naïve HIV-infected Patients

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 3 clinical and 18 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

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