Summary
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. ClinicalTrials.gov lists 2 registered trials linking Acetazolamide to Epilepsy: 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00552526). It plans or enrolled 60 participants. Registration activity spans 2007 to 2016. The literature layer holds 5 publications for this pair: 2 Cochrane reviews, 1 systematic review and 2 clinical trial publications. Publication years run from 1994 to 2023. 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 Epilepsy ranks 187 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: 60-85% achieve seizure freedom on medication; of those, 50-75% remain seizure-free after withdrawal (varies by syndrome and duration of freedom).
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 40.5 (trials 3.5, literature 17.0, tier 15, approved bonus 5.0) |
| Registered trials | 2 total: 0 recruiting, 0 active / not yet recruiting, 0 completed, 2 other |
| Linked publications | 5 (2 Cochrane reviews, 2 clinical trial publications, 1 systematic review) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | AARS1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT00552526 | Ketogenic Diet vs.Antiepileptic Drug Treatment in Drug Resistant Epilepsy 2007 | status unknown | Phase 4 | 60 |
| NCT02904265 | Efficacy Study of Acetazolamide Versus Diazepam in Continuous Spike and Wave/Landau-Kleffner Syndrome 2016 | terminated | PHASE2, PHASE3 | 3 |
Published literature
- Systematic review Episodic Ataxias: Primary and Secondary Etiologies, Treatment, and Classification ApproachesHassan A · Tremor and other hyperkinetic movements (New York, N.Y.) · 2023 · PMID 37008993
- Cochrane review Treatments for seizures in catamenial (menstrual-related) epilepsyMaguire MJ, Nevitt SJ · The Cochrane database of systematic reviews · 2021 · PMID 34528245
- Clinical trial publication AZATAX: Acetazolamide safety and efficacy in cerebellar syndrome in PMM2 congenital disorder of glycosylation (PMM2-CDG)Martínez-Monseny AF, Bolasell M, Callejón-Póo L et al. · Annals of neurology · 2019 · PMID 30873657
- Cochrane review Treatments for seizures in catamenial (menstrual-related) epilepsyMaguire MJ, Nevitt SJ · The Cochrane database of systematic reviews · 2019 · PMID 31608992
- Clinical trial publication Acetazolamide in bipolar affective disordersHayes SG · Annals of clinical psychiatry : official journal of the American Academy of Clinical Psychiatrists · 1994 · PMID 7804393
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 AARS1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Acetazolamide approved for Epilepsy?
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Epilepsy would be drug repurposing rather than first-in-human development. This does not mean it is approved for Epilepsy. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Acetazolamide in clinical trials for Epilepsy?
ClinicalTrials.gov lists 2 registered trials linking Acetazolamide to Epilepsy: 2 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT00552526). It plans or enrolled 60 participants. Registration activity spans 2007 to 2016.
What does the evidence show for Acetazolamide in Epilepsy?
Trials of Acetazolamide in Epilepsy 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 AARS1. 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.