Summary
Naltrexone has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Alcohol Use Disorder would be drug repurposing rather than first-in-human development. This does not mean it is approved for Alcohol Use Disorder. ClinicalTrials.gov lists 8 registered trials linking Naltrexone to Alcohol Use Disorder: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00145847). The largest enrolment is 7500 participants (NCT00439049). Registration activity spans 2003 to 2023. The literature layer holds 10 publications for this pair: 2 Cochrane reviews, 2 systematic reviews, 1 randomised controlled trial publication and 5 clinical trial publications. Publication years run from 2010 to 2021. 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 Alcohol Abuse ranks 375 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: 30-40% of people with AUD achieve sustained abstinence without formal treatment at 1 year (natural recovery); but functional impairment, liver damage, and mortality risk accumulate; relapse in 50-60% within 1 year even after treatment.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 71.0 (trials 22.0, literature 29.0, tier 15, approved bonus 5.0) |
| Registered trials | 8 total: 0 recruiting, 0 active / not yet recruiting, 5 completed, 3 other |
| Linked publications | 10 (5 clinical trial publications, 2 Cochrane reviews, 2 systematic reviews, 1 randomised controlled trial publication) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | ADH1C |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00145847 | Naltrexone Treatment of Alcohol Abuse in Schizophrenia 2003 | completed | Phase 4 | 90 |
| NCT01115894 | Medication and Counseling for Controlled Drinking 2007 | status unknown | Phase 3 | 200 |
| NCT00667875 | An Exploratory Study of Naltrexone Plus Aripiprazole for Alcohol Dependence 2008 | completed | Phase 2 | 65 |
| NCT06434818 | Enhanced Digital-Chemosensory-Based Olfactory Training for Remote Management of Substance Use Disorders 2023 | status unknown | Phase 2 | 300 |
| NCT01625611 | Kappa-PET Imaging and Naltrexone in Alcohol Drinking Behaviors 2011 | completed | Phase 1 | 59 |
| NCT02885311 | Collaborative Care for Alcohol Use Disorders in the Patient-centered Medical Home 2017 | completed | PHASE1, PHASE2 | 27 |
| NCT01227044 | Reducing Heavy Drinking to Optimize HIV/AIDS Treatment and Prevention 2011 | completed | PHASE2, PHASE3 | 51 |
| NCT00439049 | Substance Abuse Pre-Treatment Screening Study 2005 | status unknown | Not applicable | 7,500 |
Published literature
- Clinical trial publication Combining behavioral harm-reduction treatment and extended-release naltrexone for people experiencing homelessness and alcohol use disorder in the USA: a randomised clinical trialCollins SE, Duncan MH, Saxon AJ et al. · The lancet. Psychiatry · 2021 · PMID 33713622
- Clinical trial publication Treating Alcohol Use Disorder in U.S. Veterans: The Role of Traumatic Brain InjuryJorge RE, Li R, Liu X et al. · The Journal of neuropsychiatry and clinical neurosciences · 2019 · PMID 31117905
- Cochrane review Sleep and the Pharmacotherapy of Alcohol Use Disorder: Unfortunate Bedfellows. A Systematic Review With Meta-AnalysisPanin F, Peana AT · Frontiers in pharmacology · 2019 · PMID 31680952
- Systematic review Smartphone Apps Targeting Alcohol and Illicit Substance Use: Systematic Search in in Commercial App Stores and Critical Content AnalysisTofighi B, Chemi C, Ruiz-Valcarcel J et al. · JMIR mHealth and uHealth · 2019 · PMID 31008713
- Clinical trial publication Patient predictors of substance use disorder treatment initiation in primary careOber AJ, Watkins KE, McCullough CM et al. · Journal of substance abuse treatment · 2018 · PMID 29866385
- Clinical trial publication An open-label pilot study of icariin for co-morbid bipolar and alcohol use disorderXiao H, Wignall N, Brown ES · The American journal of drug and alcohol abuse · 2016 · PMID 26809351
- Cochrane review Disulfiram efficacy in the treatment of alcohol dependence: a meta-analysisSkinner MD, Lahmek P, Pham H et al. · PloS one · 2014 · PMID 24520330
- Clinical trial publication Pharmacological challenge with naloxone and cue exposure in alcohol dependence: results of a randomized, double-blind placebo-controlled trialLieb M, Palm U, Chiang S et al. · The world journal of biological psychiatry : the official journal of the World Federation of Societies of Biological Psychiatry · 2013 · PMID 24020866
- Randomized controlled trial Integrated Management of Physician-delivered Alcohol Care for Tuberculosis Patients: Design and ImplementationGreenfield SF, Shields A, Connery HS et al. · Alcoholism, clinical and experimental research · 2010 · PMID 19930235
- Systematic review Identification of molecular targets associated with ethanol toxicity and implications in drug developmentWang LL, Yang AK, He SM et al. · Current pharmaceutical design · 2010 · PMID 20184550
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 DGIdb, in this case via the biomarker or target ADH1C. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Naltrexone approved for Alcohol Use Disorder?
Naltrexone has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Alcohol Use Disorder would be drug repurposing rather than first-in-human development. This does not mean it is approved for Alcohol Use Disorder. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Naltrexone in clinical trials for Alcohol Use Disorder?
ClinicalTrials.gov lists 8 registered trials linking Naltrexone to Alcohol Use Disorder: 5 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00145847). The largest enrolment is 7500 participants (NCT00439049). Registration activity spans 2003 to 2023.
What does the evidence show for Naltrexone in Alcohol Use Disorder?
Naltrexone has both completed registered trials and synthesis-level publications linked to Alcohol Use Disorder. 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 DGIdb, in this case via the biomarker or target ADH1C. 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.