Summary
Zidovudine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in HIV/AIDS would be drug repurposing rather than first-in-human development. This does not mean it is approved for HIV/AIDS. ClinicalTrials.gov lists 8 registered trials linking Zidovudine to HIV/AIDS: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT01075152). The largest enrolment is 3000 participants (NCT00002334). Registration activity spans 1999 to 2011. The literature layer holds 15 publications for this pair: 5 Cochrane reviews, 3 meta-analyses, 1 systematic review, 1 randomised controlled trial publication and 5 clinical trial publications. Publication years run from 2021 to 2026. Because at least one synthesis-level source exists (systematic review, meta-analysis or Cochrane review), this pair has been assessed beyond single studies, although the synthesis may concern a different indication.
The RepurpOS disease-intelligence file for HIV/AIDS ranks 249 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those. Spontaneous remission context recorded for the condition: Spontaneous elite control (natural viremic suppression without ART) in <1% of people living with HIV; untreated, median time to AIDS ~10 years; viral rebound within weeks of ART interruption in 99%+ of patients.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 73.5 (trials 23.5, literature 30.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 6 completed, 2 other |
| Linked publications | 15 (5 Cochrane reviews, 5 clinical trial publications, 3 meta-analyses, 1 systematic review, 1 randomised controlled trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT01075152 | Cryptococcal Optimal ART Timing Trial 2010 | completed | Phase 4 | 177 |
| NCT01511237 | Perinatal Antiretroviral Intensification for PMTCT of HIV in Late Comers 2011 | completed | Phase 3 | 379 |
| NCT00002334 | 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 | completed | Phase 3 | 3,000 |
| NCT00004998 | Safety and Effectiveness of a New Anti-HIV Drug (AG1549) in Combination With Other Anti-HIV Drugs in HIV-Infected Patients 1999 | suspended | Phase 2 | 30 |
| NCT00000887 | A Study to Evaluate the Safety and Tolerance of Nelfinavir (NFV) Given With Zidovudine (ZDV) and Lamivudine (3TC) in HIV-Positive Pregnant Women and Their Infants | completed | Phase 1 | 24 |
| NCT00087945 | Blood Levels of Abacavir in HIV Infected Adolescents 2004 | completed | Not applicable | 30 |
| NCT00000734 | Evaluation of the Interaction Between High Dose Sulfamethoxazole/Trimethoprim and Zidovudine | completed | Not applicable | 10 |
| NCT00000875 | Controlled Clinical Trial of Antiviral Cytotoxic T Lymphocyte (CTL) Infusion Following Combination Antiretroviral Drug Therapy for Asymptomatic HIV-1 Infection | terminated | Not applicable | 16 |
Published literature
- Clinical trial publication Acceptability of a Dolutegravir Oral Dispersible Film in Term Neonates Born to Mothers Living With HIVViljoen L, Purdy C, Luke V et al. · Journal of the International AIDS Society · 2026 · PMID 42319215
- Clinical trial publication Comparative evaluation of human T lymphocytes in HIV patients treated with lamivudine + zidovudine combined with nevirapine versus efavirenzZhu X, Zou M · Medicine · 2026 · PMID 41961664
- Clinical trial publication Regional Variations of Rates and Determinants of Drug Resistance Mutations in People Failing First-line Therapy for HIV-1: A Substudy from the D2EFT Phase 3b/4 Clinical TrialObeng BM, Hutchinson J, Shaik A et al. · Clinical infectious diseases : an official publication of the Infectious Diseases Society of America · 2026 · PMID 41482685
- Clinical trial publication Maternal and health worker preferences for paediatric antiretroviral formulations in neonates exposed to HIVPurdy C, Luke V, du Toit S et al. · AIDS care · 2026 · PMID 41348547
- Systematic review Clinical Presentation, Etiology, and Outcomes of HIV-Associated Cardiomyopathy: A Systematic Review of Published Case ReportsHozayen O, Hozayen J, Behers BJ et al. · Viruses · 2026 · PMID 42198713
- Clinical trial publication Effects of antiretrovirals on major adverse cardiovascular events in the REPRIEVE trial: a longitudinal cohort analysisFichtenbaum CJ, Malvestutto CD, Watanabe MG et al. · The lancet. HIV · 2025 · PMID 40482662
- Cochrane review Predictors of anemia among HIV-infected children in Ethiopia: systematic review and meta-analysisGirma D, Abita Z, Agegnehu W et al. · Scientific reports · 2025 · PMID 39966543
- Meta-analysis Epidemiology of Antiretroviral Therapy Related Adverse Drug Reactions and its Predictors Among Patients with Human Immunodeficiency Virus/AIDS in Ethiopia: A Systematic Review and Meta-analysisZewdu WS, Dagnew SB, Zeleke MM et al. · Journal of the International Association of Providers of AIDS Care · 2025 · PMID 40665675
- Meta-analysis Prevalence and risk factors of anemia among people living with HIV/AIDS in Southeast Asia: a systematic review and meta-analysisShrivastav V, M Y, Ramachandran A et al. · BMC infectious diseases · 2025 · PMID 40247203
- Meta-analysis Association of nucleoside reverse transcriptase inhibitors with adverse perinatal outcomes in pregnant women living with HIV: systematic review and meta-analysisCowdell I, Beck K, Hey M et al. · Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases · 2025 · PMID 39848582
- Randomized controlled trial Second-Line Antiretroviral Therapy for Children Living with HIV in AfricaMusiime V, Bwakura-Dangarembizi M, Szubert AJ et al. · The New England journal of medicine · 2025 · PMID 40367375
- Cochrane review A Systematical Review on ART Use in HTLV Infection: Clinical, Virological, and Immunological OutcomesFernandez T, Marconi C, Montaño-Castellón I et al. · Pathogens (Basel, Switzerland) · 2024 · PMID 39338913
- Cochrane review Comparison of safety and effectiveness of antiretroviral therapy regimens among pregnant women living with HIV at preconception or during pregnancy: a systematic review and network meta-analysis of randomized trialsMehrabi F, Karamouzian M, Farhoudi B et al. · BMC infectious diseases · 2024 · PMID 38641597
- Cochrane review Antivirals for prevention of hepatitis B virus mother-to-child transmission in human immunodeficiency virus positive pregnant women co-infected with hepatitis B virusUgwu EO, Eleje GU, Ugwu AO et al. · The Cochrane database of systematic reviews · 2023 · PMID 37306558
- Cochrane review Does antiretroviral therapy cause congenital malformations? A systematic review and meta-analysisAlemu FM, Yalew AW · Epidemiology and health · 2021 · PMID 33541012
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Zidovudine approved for HIV/AIDS?
Zidovudine has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in HIV/AIDS would be drug repurposing rather than first-in-human development. This does not mean it is approved for HIV/AIDS. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Zidovudine in clinical trials for HIV/AIDS?
ClinicalTrials.gov lists 8 registered trials linking Zidovudine to HIV/AIDS: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT01075152). The largest enrolment is 3000 participants (NCT00002334). Registration activity spans 1999 to 2011.
What does the evidence show for Zidovudine in HIV/AIDS?
Zidovudine has both completed registered trials and synthesis-level publications linked to HIV/AIDS. That is the strongest profile in this database, but the summaries here do not extract effect sizes, so read the linked reviews for direction and magnitude of benefit. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in Open Targets and ChEMBL. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.