Summary
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Glaucoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Glaucoma. ClinicalTrials.gov lists 6 registered trials linking Acetazolamide to Glaucoma: 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT01535768). It plans or enrolled 150 participants. Registration activity spans 2005 to 2015. The literature layer holds 7 publications for this pair: 1 Cochrane review, 1 meta-analysis and 5 clinical trial publications. Publication years run from 2017 to 2025. 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 Glaucoma ranks 339 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: Progressive optic nerve damage without treatment; average visual field loss 0.5-1.0 dB/year; blindness in 10-20% without treatment; no spontaneous recovery of lost vision.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 57.0 (trials 18.0, literature 19.0, tier 15, approved bonus 5.0) |
| Registered trials | 6 total: 0 recruiting, 0 active / not yet recruiting, 3 completed, 3 other |
| Linked publications | 7 (5 clinical trial publications, 1 Cochrane review, 1 meta-analysis) |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | ABCA1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT01228149 | COSOPT-S® Treatment Versus Acetazolamide Before Trabeculectomy 2010 | completed | Phase 4 | 62 |
| NCT01535768 | Effect of Prophylactic Aqueous Suppression on Hyperencapsulation of Ahmed Glaucoma Valves 2012 | status unknown | Phase 4 | 150 |
| NCT00485238 | ALPI vs Medical Therapy Effects on Optic Nerve Structure & Function 2007 | status unknown | Phase 4 | 60 |
| NCT02390284 | Stop Retinal Ganglion Cell Dysfunction Study 2015 | terminated | Phase 3 | 28 |
| NCT01923454 | Immediate Anterior Chamber Paracentesis With A 30-Gauge Needle for Acute Primary Angle - Closure 2005 | completed | Not applicable | 15 |
| NCT01028664 | Use of an Ocular Telemetry Sensor in Diamox Treated Patients 2009 | completed | Not applicable | 20 |
Published literature
- Clinical trial publication Initial Results of the Paul Ahmed Comparison (PAC) Study in Refractory Childhood GlaucomaElhusseiny AM, Khaled OM, Chauhan MZ et al. · American journal of ophthalmology · 2025 · PMID 39510370
- Clinical trial publication Comparison between Surgical Techniques Used for Revision of the Filter Bleb: A Randomized Controlled Clinical TrialZantut F, P B Gracitelli C, Teixeira SH et al. · Ophthalmic research · 2023 · PMID 37285822
- Clinical trial publication Various modalities of cyclodestruction in non-refractory glaucoma: a comparative studyAbdullatif AM, Ahmed El-Saied HM · International ophthalmology · 2021 · PMID 34009517
- Clinical trial publication Effect of antiglaucoma agents on short-term intraocular pressure fluctuations after intravitreal bevacizumab injectionsShoeibi N, Ghosi Z, Jafari H et al. · International ophthalmology · 2021 · PMID 33389369
- Meta-analysis Side effects of acetazolamide: a systematic review and meta-analysis assessing overall risk and dose dependenceSchmickl CN, Owens RL, Orr JE et al. · BMJ open respiratory research · 2020 · PMID 32332024
- Clinical trial publication Efficacy and safety of preoperative IOP reduction using a preservative-free fixed combination of dorzolamide/timolol eye drops versus oral acetazolamide and dexamethasone eye drops and assessment of the clinical outcome of trabeculectomy in glaucomaLorenz K, Wasielica-Poslednik J, Bell K et al. · PloS one · 2017 · PMID 28199397
- Cochrane review Perioperative medications for preventing temporarily increased intraocular pressure after laser trabeculoplastyZhang L, Weizer JS, Musch DC · The Cochrane database of systematic reviews · 2017 · PMID 28231380
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 ABCA1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Acetazolamide approved for Glaucoma?
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Glaucoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Glaucoma. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Acetazolamide in clinical trials for Glaucoma?
ClinicalTrials.gov lists 6 registered trials linking Acetazolamide to Glaucoma: 3 have completed; 3 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT01535768). It plans or enrolled 150 participants. Registration activity spans 2005 to 2015.
What does the evidence show for Acetazolamide in Glaucoma?
Acetazolamide has both completed registered trials and synthesis-level publications linked to Glaucoma. 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, in this case via the biomarker or target ABCA1. 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.