Summary
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Schizophrenia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Schizophrenia. ClinicalTrials.gov lists 1 registered trial linking Acetazolamide to Schizophrenia: 1 is currently recruiting. The most advanced is PHASE1, PHASE2 (NCT04887792). It plans or enrolled 60 participants. Registered activity dates to 2022. The literature layer holds 1 publication for this pair: 1 Cochrane review. The dated items are from 2022. 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 Schizophrenia ranks 499 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 full remission rare (<10%); after first episode, ~80% relapse within 5 years without maintenance antipsychotic; functional disability accumulates over time; 20% achieve good long-term outcome.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 28.0 (trials 3.0, literature 5.0, tier 15, approved bonus 5.0) |
| Registered trials | 1 total: 1 recruiting, 0 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 1 (1 Cochrane review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ADRA1A |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT04887792 | Acetazolamide for Treatment Resistant Schizophrenia 2022 | recruiting | PHASE1, PHASE2 | 60 |
Published literature
- Cochrane review Non-antipsychotic pharmacotherapy of psychogenic polydipsia: A systematic reviewHavens TH, Innamorato G, Nemec EC 2nd · Journal of psychosomatic research · 2022 · PMID 34856427
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, in this case via the biomarker or target ADRA1A. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Acetazolamide approved for Schizophrenia?
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Schizophrenia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Schizophrenia. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Acetazolamide in clinical trials for Schizophrenia?
ClinicalTrials.gov lists 1 registered trial linking Acetazolamide to Schizophrenia: 1 is currently recruiting. The most advanced is PHASE1, PHASE2 (NCT04887792). It plans or enrolled 60 participants. Registered activity dates to 2022.
What does the evidence show for Acetazolamide in Schizophrenia?
Trials of Acetazolamide in Schizophrenia are registered but none has completed, so there is no outcome evidence from those studies yet; the record is a signal of research interest. 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, in this case via the biomarker or target ADRA1A. 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.