CLINICAL TRIALS PROFILE FOR ACETAZOLAMIDE

All Clinical Trials for acetazolamide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000115 ↗	Randomized Trial of Acetazolamide for Uveitis-Associated Cystoid Macular Edema	Completed	National Eye Institute (NEI)	Phase 2	1990-12-01	To test the efficacy of acetazolamide for the treatment of uveitis-associated cystoid macular edema.
NCT00004802 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders	Completed	Ohio State University	Phase 3	1992-06-01	OBJECTIVES: I. Assess the efficacy of dichlorphenamide in the treatment of episodic weakness attacks in patients with hyperkalemic periodic paralysis, paramyotonia congenita with periodic paralysis, and hypokalemic periodic paralysis.
NCT00004802 ↗	Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders	Completed	National Center for Research Resources (NCRR)	Phase 3	1992-06-01	OBJECTIVES: I. Assess the efficacy of dichlorphenamide in the treatment of episodic weakness attacks in patients with hyperkalemic periodic paralysis, paramyotonia congenita with periodic paralysis, and hypokalemic periodic paralysis.
NCT00108602 ↗	Sleep Apnea: Mechanism and Cerebrovascular Consequences	Withdrawn	US Department of Veterans Affairs	N/A	2007-05-01	This study will determine the effect of ventilatory stimulation on obstructive sleep apnea in selected patients with stroke. We will select a subset of patients with stroke in the prior 3 months who are most likely to have ventilatory instability as a cause of the upper airway obstruction as indicated by the absence of obesity, an abnormal hypocapnic apnea threshold and resolution of obstructive apnea during CO2 administration. Following baseline polysomnography, patients will be randomly assigned to acetazolamide (250 mg hs [at bedtime]) versus placebo for 7 days. Polysomnography will be repeated and then subjects will cross-over therapies for another 7 days followed by a final nocturnal polysomnography.
NCT00108602 ↗	Sleep Apnea: Mechanism and Cerebrovascular Consequences	Withdrawn	VA Office of Research and Development	N/A	2007-05-01	This study will determine the effect of ventilatory stimulation on obstructive sleep apnea in selected patients with stroke. We will select a subset of patients with stroke in the prior 3 months who are most likely to have ventilatory instability as a cause of the upper airway obstruction as indicated by the absence of obesity, an abnormal hypocapnic apnea threshold and resolution of obstructive apnea during CO2 administration. Following baseline polysomnography, patients will be randomly assigned to acetazolamide (250 mg hs [at bedtime]) versus placebo for 7 days. Polysomnography will be repeated and then subjects will cross-over therapies for another 7 days followed by a final nocturnal polysomnography.
NCT00120731 ↗	Effects of Potassium Citrate in Urine of Children With Elevated Calcium in Urine and Kidney Stones	Withdrawn	Children's Mercy Hospital Kansas City	N/A	2005-07-01	High amounts of calcium in the urine (hypercalciuria) can cause development of kidney stones in children. Treatment for these children includes plenty of fluids, a low-salt diet and medications such as potassium citrate. A major advantage of potassium citrate, as compared to hydrochlorothiazide, is its lack of side effects. One problem the researchers and others have observed is that some children continue to form kidney stones despite correction of hypercalciuria with potassium citrate. One possible explanation is that in some individuals potassium citrate therapy results in an excessive elevation of urine pH, a situation that may predispose to calcium phosphate stone formation. In this study, the researchers will study the effects of potassium citrate on urine chemistries and acid-base balance in three groups of children aged 5-17 years: - children who are hypercalciuric stone formers; - healthy children without a history of hypercalciuria or kidney stones. Particular attention will be paid to try to identify those who develop a very high urine pH (>8) and the factors leading to this metabolic reaction. The researchers will try to learn whether it is the child's characteristics, the disease manifestations, the dose of the drug, or a combination of the above which may be the cause of the development of very alkaline urine. Based on the results, the researchers hope to be able to better "tailor" the individual treatment for each child with kidney stones.
NCT00222534 ↗	Acetazolamide for Respiratory Failure in Combination With Metabolic Alkalosis	Completed	University of Oslo School of Pharmacy	Phase 4	2002-01-01	Respiratory failure is a common consequence of chronic obstructive pulmonary disease (COPD). A concurrent metabolic alkalosis may worsen the respiratory failure, as a higher pH in blood (and thus in cerebrospinal fluid) results in a weaker respiratory drive. Use of diuretics is the most common cause of metabolic alkalosis. When a patient with an acute exacerbation of a respiratory failure is also alkalotic, there are (at least theoretical) reasons to lower the pH in order to increase the respiratory drive. Among other alternatives, the drug acetazolamide can be used for this purpose. In some hospitals there is a tradition for the use of acetazolamide on this indication, but any evidence for the effect of such a treatment is rather weak. Thus, the aim of this trial is to evaluate the effect of acetazolamide as an adjuvant treatment for hospitalized patients with acute exacerbation of respiratory failure in combination with metabolic alkalosis.

Clinical Trial Conditions for acetazolamide

Condition Name

Condition Name for acetazolamide
Intervention	Trials
Obstructive Sleep Apnea	12
Acute Mountain Sickness	9
Altitude Hypoxia	8
Chronic Obstructive Pulmonary Disease	8

Condition MeSH

Condition MeSH for acetazolamide
Intervention	Trials
Altitude Sickness	31
Sleep Apnea Syndromes	23
Hypoxia	17
Sleep Apnea, Obstructive	17

Clinical Trial Locations for acetazolamide

Trials by Country

Trials by Country for acetazolamide
Location	Trials
United States	133
Kyrgyzstan	22
Belgium	8
Israel	7
Switzerland	6

Trials by US State

Trials by US State for acetazolamide
Location	Trials
California	17
Massachusetts	12
Texas	8
Florida	7
Pennsylvania	7

Clinical Trial Progress for acetazolamide

Clinical Trial Phase

Clinical Trial Phase for acetazolamide
Clinical Trial Phase	Trials
PHASE4	7
PHASE3	2
PHASE2	8

Clinical Trial Status

Clinical Trial Status for acetazolamide
Clinical Trial Phase	Trials
Completed	65
Recruiting	39
Not yet recruiting	16

Clinical Trial Sponsors for acetazolamide

Sponsor Name

Sponsor Name for acetazolamide
Sponsor	Trials
University of Zurich	27
National Center of Cardiology and Internal Medicine named after academician M.Mirrakhimov	20
University of California, San Diego	6

Sponsor Type

Sponsor Type for acetazolamide
Sponsor	Trials
Other	230
NIH	17
U.S. Fed	8

Acetazolamide Market Analysis and Financial Projection

Last updated: April 26, 2026

Acetazolamide: clinical trials landscape, market analysis, and projection

What does the current clinical trials signal for acetazolamide?

Acetazolamide is an established small-molecule carbonic anhydrase inhibitor with a long clinical history. Recent “clinical trials update” for a legacy, off-patent product is typically dominated by:

• New indications (most commonly ophthalmology, neurology, and respiratory/acid-base disorders) rather than first-in-class replication.
• Formulation and dose-optimization studies (including dosing regimens, tolerability, and comparator-controlled designs).
• Real-world evidence and prospective observational work, especially where randomized data is sparse or older.
• Pediatric cohorts and special populations (to characterize safety, exposure, and efficacy endpoints).

Implication for R&D: the dominant investment question is not “is the mechanism viable,” but “does the evidence package support label expansion in a way payers and clinicians accept,” given that acetazolamide is widely used off-label and often priced as a generic.

What trials are actively driving evidence right now?

No complete, current, trial-by-trial public record can be reliably produced from the information in this prompt. A correct “update” requires current registry pulls (ClinicalTrials.gov/WHO ICTRP), including study identifiers, status, endpoints, and enrollment. Without those feed-level data, producing a trial list would risk inaccuracies.

How does the regulatory and IP posture shape clinical development?

Acetazolamide’s IP status is a core constraint on how sponsors structure trials and marketing. For a legacy generic, the practical paths to meaningful commercial differentiation are:

• Route/formulation (extended release, ophthalmic delivery, fixed-dose combinations).
• Geography-specific lifecycle management (where brand differentiation depends on supply chain and regulatory filings rather than patent exclusivity).
• Indication-specific evidence to support reimbursement and guideline uptake.

Clinical trial design trend to expect for generics: smaller, pragmatic studies or bridge trials that can support label language and payer coverage, rather than large phase 3 programs aimed at de novo drug-class establishment.

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How big is the acetazolamide market and where does demand come from?

What are the main use segments?

Market demand for acetazolamide is driven by a mix of on-label and off-label use across multiple specialty categories. Common clinical demand drivers include:

• Glaucoma (and related ocular hypertension): carbonic anhydrase inhibition reduces aqueous humor production.
• Altitude illness prophylaxis and treatment: historical and ongoing travel-medication use.
• Edema and heart failure adjuncts (in selected settings), where diuretic synergy or metabolic effects matter.
• Neurologic indications (examples often studied historically: idiopathic intracranial hypertension and periodic syndromes tied to acid-base balance).
• Metabolic acidosis-related disorders and respiratory alkalosis contexts, where the pharmacologic effect on acid-base handling is relevant.

Where does procurement and pricing usually sit?

For an older, widely available compound, the market structure is typically characterized by:

• Low price points vs newer branded specialty products.
• High sensitivity to supply continuity and reimbursement processes.
• Generic competition in most geographies, with limited room for premium pricing unless formulation or indication supports it.

Business takeaway: Revenue tends to track volume and formulary access more than patent-protected differentiation.

What is the market projection?

A numeric forecast requires current market sizing baselines (by geography, channel, and indication mix) and an assumption set (price erosion rate, CAGR, and volume growth). Those required figures cannot be generated accurately from the prompt alone, and producing a quantified projection without registry- and payer-grade inputs would not be a “hard-data” answer.

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What is the R&D and investment outlook for acetazolamide?

Does acetazolamide still have a credible development runway?

For generics, the runway depends on how sponsors can create a “commercial wedge.” The most actionable wedges tend to be:

• Label expansion with payer-friendly endpoints: fewer adverse events, clearer responder definitions, and outcomes aligned to coverage criteria.
• Formulation differentiation: improved tolerability or adherence can generate a modest premium and higher clinician acceptance.
• Combination products: fixed-dose co-administration can reduce pill burden and improve uptake, even when active ingredients are off-patent.

What risks most affect future value?

• Generic price compression if multiple suppliers compete on the same indication without meaningful differentiation.
• Evidence sufficiency: trials must support label language that clinicians and payers accept, not just biologic plausibility.
• Safety tolerability constraints: acetazolamide is limited by known carbonic anhydrase inhibitor class effects, which can constrain use in broader populations.

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Key Takeaways

• Acetazolamide’s clinical and commercial dynamics are dominated by legacy generic status, so trial and market value hinges on indication-specific evidence and formulation or access differentiation.
• A precise, current “clinical trials update” and a numeric market projection require live registry and market sizing data; those inputs are not present in this prompt.
• The most credible near-term R&D path for acetazolamide is label expansion or pragmatic evidence generation that aligns with reimbursement and clinician practice, not de novo class establishment.

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FAQs

1. Is acetazolamide currently protected by patents in major markets?
   Patent protection for acetazolamide is generally limited; commercial differentiation is typically achieved through formulation and indication-specific regulatory pathways rather than exclusivity.

2. What endpoints typically matter for acetazolamide evidence packages?
   Endpoints usually relate to clinical control measures (ocular pressure targets for glaucoma-related uses, neurologic outcome metrics for neurologic indications, and symptom and recurrence outcomes for prophylaxis settings), plus tolerability.

3. Why do new trials still get run for acetazolamide?
   Sponsors pursue guideline- and reimbursement-aligned evidence in narrower populations, often including special populations such as pediatrics, or they evaluate new formulations and dosing regimens.

4. How does generic competition affect the market projection?
   Competition drives pricing toward low levels; market growth tends to depend on volume expansion, formulary placement, and differentiation through evidence or formulation rather than sustained premium pricing.

5. What is the most likely commercial “wedge” for acetazolamide?
   Indication-specific label expansion supported by payer-friendly outcomes, paired with a practical tolerability or adherence advantage via formulation or dosing.

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References

[1] ClinicalTrials.gov. (n.d.). Acetazolamide search results and trial records. https://clinicaltrials.gov/
[2] WHO International Clinical Trials Registry Platform. (n.d.). Acetazolamide registry search. https://trialsearch.who.int/