Disease × agent evidence record

Acetazolamide for Heart Failure: evidence, trials and status

Acetazolamide has both completed registered trials and synthesis-level publications linked to Heart Failure. 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.

8 registered trials 3 recruiting 13 publications Evidence tier A · Strong Score 78.5
Research Tracker › Pairs › Heart Failure › Acetazolamide
Research map, not treatment advice. This page aggregates registry and literature records. It does not evaluate efficacy, dosing or safety for any individual. Discuss any treatment decision with a qualified clinician.

Summary

Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Heart Failure would be drug repurposing rather than first-in-human development. This does not mean it is approved for Heart Failure. ClinicalTrials.gov lists 8 registered trials linking Acetazolamide to Heart Failure: 3 are currently recruiting; 1 has completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT04062760). The largest enrolment is 320 participants (NCT07199088). Registration activity spans 2007 to 2025. The literature layer holds 13 publications for this pair: 5 Cochrane reviews, 3 meta-analyses and 5 clinical trial publications. Publication years run from 2023 to 2026. 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 Heart Failure ranks 616 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: Annual mortality ~22% on standard care; only 5-11% of HFrEF patients achieve normalisation of ejection fraction (HFrecEF) with GDMT; HFpEF has no disease-modifying therapy.

Open in Repurposing ExplorerHeart Failure hubHeart Failure biomarkers & interventionsChEMBLOpen Targets

Evidence table

Evidence tierA · Strong
Evidence score78.5 (trials 28.5, literature 30.0, tier 15, approved bonus 5.0)
Registered trials8 total: 3 recruiting, 0 active / not yet recruiting, 1 completed, 4 other
Linked publications13 (5 Cochrane reviews, 5 clinical trial publications, 3 meta-analyses)
Agent typeDrug (Small molecule)
Development stage (any indication)Approved
Mechanism / classNot recorded
Data sourcesOpen Targets, ChEMBL
Linked via biomarker / targetABO
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 IDTitleStatusPhaseEnrolment
NCT00517426Effects of Acetazolamide and CO2 Inhalation on Exercise-induced Periodic Breathing in Heart Failure
2007
completedPhase 420
NCT06414759Efficacy and Safety of Combination Diuretic Therapy in Patients With Acute Decompensated Heart Failure and Volume Overload
2024
recruitingPhase 4110
NCT06475105Combination Oral Acetazolamide and Intravenous Furosemide on Acute Decompensated Heart Failure Outcomes
2024
recruitingPhase 466
NCT05986773Diuretic Strategies in Acute Heart Failure Patients at High Risk for Diuretic Resistance
2023
terminatedPhase 413
NCT04062760Safety and Efficacy of Early, seQUential Oral dIuretic Nephron blockAde In Acute Heart Failure
2019
status unknownPhase 4206
NCT00746954Buspirone as a Potential Treatment for Recurrent Central Apnea
2008
terminatedPhase 38
NCT05940220Efficacy of Oral Acetazolamide in Decongestion in Patients With Heart Failure
2023
status unknownPhase 3130
NCT07199088Comparative Efficacy and Safety of Acetazolamide Versus Metolazone as an Adjunct to Standard Therapy in Patients With Acute Decompensated Heart Failure
2025
recruitingNot applicable320

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Published literature

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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 ABO. The tier describes how well the drug-disease link is documented, not how well the drug works.

Frequently asked questions

Is Acetazolamide approved for Heart Failure?

Acetazolamide has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Heart Failure would be drug repurposing rather than first-in-human development. This does not mean it is approved for Heart Failure. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.

Is Acetazolamide in clinical trials for Heart Failure?

ClinicalTrials.gov lists 8 registered trials linking Acetazolamide to Heart Failure: 3 are currently recruiting; 1 has completed; 4 are terminated, withdrawn, suspended or of unknown status. The most advanced is Phase 4 (NCT04062760). The largest enrolment is 320 participants (NCT07199088). Registration activity spans 2007 to 2025.

What does the evidence show for Acetazolamide in Heart Failure?

Acetazolamide has both completed registered trials and synthesis-level publications linked to Heart Failure. 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 ABO. The tier describes how well the drug-disease link is documented, not how well the drug works.

Cite this page

Open Source Medicine Foundation. Acetazolamide for Heart Failure: evidence, trials and status. OSMF Research Tracker. Updated 2026-07-05. https://research.opensourcemed.info/pairs/heart-failure/acetazolamide.html

Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-05. Page built 2026-10-07.

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This is a research map, not treatment advice. Evidence tiers and scores summarise what has been studied, not whether a treatment works or is safe for you.