Disease × agent evidence record

Efavirenz for HIV/AIDS: evidence, trials and status

Efavirenz 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.

8 registered trials 0 recruiting 13 publications Evidence tier A · Strong Score 77.0
Research Tracker › Pairs › HIV/AIDS › Efavirenz
Research map, not treatment advice. This page aggregates registry and literature records. It does not evaluate efficacy, dosing or safety for any individual. Discuss any treatment decision with a qualified clinician.

Summary

Efavirenz 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 ClinicalTrials.gov lists 8 registered trials linking Efavirenz to HIV/AIDS: 6 have completed; 2 are terminated, withdrawn, suspended or of unknown status.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 728 participants (NCT01065792). Registration activity spans 2007 to 2022. The literature layer holds 13 publications for this pair: 5 Cochrane reviews, 3 meta-analyses and 5 clinical trial publications. Publication years run from 2024 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.

Open in Repurposing ExplorerHIV/AIDS hubHIV/AIDS biomarkers & interventionsChEMBLOpen Targets

Evidence table

Evidence tierA · Strong
Evidence score77.0 (trials 27.0, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 0 recruiting, 0 active / not yet recruiting, 6 completed, 2 other
Linked publications13 (5 Cochrane reviews, 5 clinical trial publications, 3 meta-analyses)
Agent typeDrug (Small molecule)
Development stage (any indication)Approved
Mechanism / classNot recorded
Data sourcesOpen 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 IDTitleStatusPhaseEnrolment
NCT03502005Efficacy, Safety & Tolerability of Switching EFV/TDF/FTC to BIC/FTC/TAF in Virologically Suppressed Adults With HIV-1
2018
completedPhase 4100
NCT00978237Clinical Trial to Assess the Effect of the Change of Efavirenz (EFV) for Lopinavir/Ritonavir (LPV/r) in Lipoatrophy in HIV-infected Patients
2009
completedPhase 420
NCT01075152Cryptococcal Optimal ART Timing Trial
2010
completedPhase 4177
NCT01778413Virological and Immunological Safety of a Dose Reduction Strategy Antiretroviral Regimen With Efavirenz / Tenofovir / Emtricitabine
2013
completedPhase 461
NCT00427297Optimizing Pediatric HIV-1 Treatment in Infants With Prophylactic Exposure to Nevirapine, Nairobi, Kenya
2007
terminatedPhase 334
NCT00001086A Study of Abacavir Plus Indinavir Sulfate Plus Efavirenz in HIV-Infected PatientscompletedPhase 2300
NCT01065792Stocrin Re-examination Study (0831-028)
2009
completedNot applicable728
NCT03158857Hepatitis C Treatment Study in Myanmar
2022
withdrawnNot applicable—

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Published literature

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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 Efavirenz approved for HIV/AIDS?

Efavirenz 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 Efavirenz in clinical trials for HIV/AIDS?

ClinicalTrials.gov lists 8 registered trials linking Efavirenz 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 728 participants (NCT01065792). Registration activity spans 2007 to 2022.

What does the evidence show for Efavirenz in HIV/AIDS?

Efavirenz 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

Open Source Medicine Foundation. Efavirenz for HIV/AIDS: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/hivaids/efavirenz.html

Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.

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This is a research map, not treatment advice. Evidence tiers and scores summarise what has been studied, not whether a treatment works or is safe for you.