Summary
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Melanoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Melanoma. ClinicalTrials.gov lists 2 registered trials linking Acetazolamide to Melanoma: 1 is currently recruiting; 1 is active or not yet recruiting. The most advanced is Phase 1 (NCT05470283). The largest enrolment is 208 participants (NCT06060613). Registration activity spans 2022 to 2023. No published literature item is linked to Acetazolamide and Melanoma in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Melanoma ranks 50 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 regression documented in ~0.4% of metastatic melanoma cases; early-stage surgical resection: 90%+ 5-year survival for Stage I; Stage IV (metastatic): historically <10% 5-year survival.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 26.5 (trials 6.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 2 total: 1 recruiting, 1 active / not yet recruiting, 0 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | Open Targets, ChEMBL |
| Linked via biomarker / target | AKT1 |
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trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5).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 |
|---|---|---|---|---|
| NCT05470283 | Phase I, Open-Label, Study of Tumor Infiltrating Lymphocytes Engineered With Membrane Bound IL15 Plus Acetazolamide in Adult Patients With Metastatic Melanoma 2022 | active, not recruiting | Phase 1 | 21 |
| NCT06060613 | Safety and Efficacy of OBX-115 in Advanced Solid Tumors 2023 | recruiting | PHASE1, PHASE2 | 208 |
Published literature
No publication is linked to this pair yet.
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 AKT1. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Acetazolamide approved for Melanoma?
Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Melanoma would be drug repurposing rather than first-in-human development. This does not mean it is approved for Melanoma. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Acetazolamide in clinical trials for Melanoma?
ClinicalTrials.gov lists 2 registered trials linking Acetazolamide to Melanoma: 1 is currently recruiting; 1 is active or not yet recruiting. The most advanced is Phase 1 (NCT05470283). The largest enrolment is 208 participants (NCT06060613). Registration activity spans 2022 to 2023.
What does the evidence show for Acetazolamide in Melanoma?
Trials of Acetazolamide in Melanoma 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 AKT1. 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 2026-07-06. Page built 2026-10-07.