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Last Updated: December 12, 2025

CLINICAL TRIALS PROFILE FOR ACETAZOLAMIDE SODIUM


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All Clinical Trials for ACETAZOLAMIDE SODIUM

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.
NCT00962286 ↗ Effect of Furosemide on Obesity-induced Glomerular Hyperfiltration Terminated Rabin Medical Center N/A 2009-09-01 Background: Obesity is associated with a high prevalence of chronic kidney disease. The glomerular hyperfiltration associated with obesity may play a role in the pathogenesis of obesity associated chronic kidney disease. Attenuation of hyperfiltration by pharmacological means may slow down the development and progression of chronic renal failure. The investigators have previously shown that acetazolamide, a proximally acting diuretic that activates TGF by increasing solute delivery to the macula densa, abates glomerular hyperfiltration. The present study was designed to test the hypothesis that this decrease in hyperfiltration is specific to acetazolamide and not due to a non specific diuretic effect. The aim of the present study is to evaluate the effects of the administration of furosemide p.o. to subjects with severe obesity on glomerular hemodynamics. Methods: Ten obese subjects will participate in the study. They will undergo measurement of glomerular filtration rate (inulin clearance) (GFR), renal plasma flow (RPF) (p-aminohippuric acid clearance), filtration fraction, fractional excretion of lithium (FE LI) and blood pressure, before and after administration of oral furosemide 20 to 40 mg bid for 3 days. The effects of furosemide on glomerular hemodynamics in obese subjects will be compared to the previously studied effects of acetazolamide.
NCT01003639 ↗ Idiopathic Intracranial Hypertension Treatment Trial Completed National Eye Institute (NEI) Phase 2/Phase 3 2010-01-01 Idiopathic intracranial hypertension (IIH), also called pseudotumor cerebri, is a disorder of elevated intracranial pressure of unknown cause [Corbett, et al., 1982; Wall, et al., 1991]. Its incidence is 22.5 new cases each year per 100,000 overweight women of childbearing age, and is rising [Garrett, et al., 2004] in parallel with the obesity epidemic. It affects about 100,000 Americans. Most patients suffer debilitating headaches. Because of pressure on the optic nerve (papilledema), 86% have some degree of permanent visual loss and 10% develop severe visual loss [Wall, et al., 1991]. Interventions to prevent loss of sight, all with unproven efficacy, include diet, diuretics such as acetazolamide, repeated spinal taps, optic nerve sheath fenestration surgery, and cerebrospinal fluid (CSF) shunting procedures. The purported goal of these therapies is to lower intracranial pressure; however, it is unclear which treatments work and by what mechanism. None of these strategies has been verified by properly designed clinical trials. Thus, there is confusion, uncertainty, and weak scientific rationales to guide treatment decisions. This trial will study subjects who have mild visual loss from IIH to (1) establish convincing, evidence-based treatment strategies for IIH to restore and protect vision, (2) follow subjects up to 4 years to observe the long-term treatment outcomes and (3) determine the cause of IIH. To meet those aims, the trial will be divided into a 12-month intervention phase and a 3-year observational phase. Subjects are not required to complete the observational phase of the study, but will be asked to do so and consented for the observational phase of the study at the conclusion of the intervention phase (12 months).
NCT01003639 ↗ Idiopathic Intracranial Hypertension Treatment Trial Completed University of California, Davis Phase 2/Phase 3 2010-01-01 Idiopathic intracranial hypertension (IIH), also called pseudotumor cerebri, is a disorder of elevated intracranial pressure of unknown cause [Corbett, et al., 1982; Wall, et al., 1991]. Its incidence is 22.5 new cases each year per 100,000 overweight women of childbearing age, and is rising [Garrett, et al., 2004] in parallel with the obesity epidemic. It affects about 100,000 Americans. Most patients suffer debilitating headaches. Because of pressure on the optic nerve (papilledema), 86% have some degree of permanent visual loss and 10% develop severe visual loss [Wall, et al., 1991]. Interventions to prevent loss of sight, all with unproven efficacy, include diet, diuretics such as acetazolamide, repeated spinal taps, optic nerve sheath fenestration surgery, and cerebrospinal fluid (CSF) shunting procedures. The purported goal of these therapies is to lower intracranial pressure; however, it is unclear which treatments work and by what mechanism. None of these strategies has been verified by properly designed clinical trials. Thus, there is confusion, uncertainty, and weak scientific rationales to guide treatment decisions. This trial will study subjects who have mild visual loss from IIH to (1) establish convincing, evidence-based treatment strategies for IIH to restore and protect vision, (2) follow subjects up to 4 years to observe the long-term treatment outcomes and (3) determine the cause of IIH. To meet those aims, the trial will be divided into a 12-month intervention phase and a 3-year observational phase. Subjects are not required to complete the observational phase of the study, but will be asked to do so and consented for the observational phase of the study at the conclusion of the intervention phase (12 months).
NCT01003639 ↗ Idiopathic Intracranial Hypertension Treatment Trial Completed University of Iowa Phase 2/Phase 3 2010-01-01 Idiopathic intracranial hypertension (IIH), also called pseudotumor cerebri, is a disorder of elevated intracranial pressure of unknown cause [Corbett, et al., 1982; Wall, et al., 1991]. Its incidence is 22.5 new cases each year per 100,000 overweight women of childbearing age, and is rising [Garrett, et al., 2004] in parallel with the obesity epidemic. It affects about 100,000 Americans. Most patients suffer debilitating headaches. Because of pressure on the optic nerve (papilledema), 86% have some degree of permanent visual loss and 10% develop severe visual loss [Wall, et al., 1991]. Interventions to prevent loss of sight, all with unproven efficacy, include diet, diuretics such as acetazolamide, repeated spinal taps, optic nerve sheath fenestration surgery, and cerebrospinal fluid (CSF) shunting procedures. The purported goal of these therapies is to lower intracranial pressure; however, it is unclear which treatments work and by what mechanism. None of these strategies has been verified by properly designed clinical trials. Thus, there is confusion, uncertainty, and weak scientific rationales to guide treatment decisions. This trial will study subjects who have mild visual loss from IIH to (1) establish convincing, evidence-based treatment strategies for IIH to restore and protect vision, (2) follow subjects up to 4 years to observe the long-term treatment outcomes and (3) determine the cause of IIH. To meet those aims, the trial will be divided into a 12-month intervention phase and a 3-year observational phase. Subjects are not required to complete the observational phase of the study, but will be asked to do so and consented for the observational phase of the study at the conclusion of the intervention phase (12 months).
NCT01003639 ↗ Idiopathic Intracranial Hypertension Treatment Trial Completed University of Rochester Phase 2/Phase 3 2010-01-01 Idiopathic intracranial hypertension (IIH), also called pseudotumor cerebri, is a disorder of elevated intracranial pressure of unknown cause [Corbett, et al., 1982; Wall, et al., 1991]. Its incidence is 22.5 new cases each year per 100,000 overweight women of childbearing age, and is rising [Garrett, et al., 2004] in parallel with the obesity epidemic. It affects about 100,000 Americans. Most patients suffer debilitating headaches. Because of pressure on the optic nerve (papilledema), 86% have some degree of permanent visual loss and 10% develop severe visual loss [Wall, et al., 1991]. Interventions to prevent loss of sight, all with unproven efficacy, include diet, diuretics such as acetazolamide, repeated spinal taps, optic nerve sheath fenestration surgery, and cerebrospinal fluid (CSF) shunting procedures. The purported goal of these therapies is to lower intracranial pressure; however, it is unclear which treatments work and by what mechanism. None of these strategies has been verified by properly designed clinical trials. Thus, there is confusion, uncertainty, and weak scientific rationales to guide treatment decisions. This trial will study subjects who have mild visual loss from IIH to (1) establish convincing, evidence-based treatment strategies for IIH to restore and protect vision, (2) follow subjects up to 4 years to observe the long-term treatment outcomes and (3) determine the cause of IIH. To meet those aims, the trial will be divided into a 12-month intervention phase and a 3-year observational phase. Subjects are not required to complete the observational phase of the study, but will be asked to do so and consented for the observational phase of the study at the conclusion of the intervention phase (12 months).
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for ACETAZOLAMIDE SODIUM

Condition Name

Condition Name for ACETAZOLAMIDE SODIUM
Intervention Trials
Heart Failure 2
Idiopathic Intracranial Hypertension 2
Obesity-induced Hyperfiltration 2
Heart Failure Acute 1
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Condition MeSH

Condition MeSH for ACETAZOLAMIDE SODIUM
Intervention Trials
Heart Failure 3
Edema 3
Pseudotumor Cerebri 2
Intracranial Hypertension 2
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Clinical Trial Locations for ACETAZOLAMIDE SODIUM

Trials by Country

Trials by Country for ACETAZOLAMIDE SODIUM
Location Trials
United States 51
Canada 4
Israel 2
Puerto Rico 1
Spain 1
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Trials by US State

Trials by US State for ACETAZOLAMIDE SODIUM
Location Trials
California 3
Tennessee 2
Washington 2
Virginia 2
Utah 2
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Clinical Trial Progress for ACETAZOLAMIDE SODIUM

Clinical Trial Phase

Clinical Trial Phase for ACETAZOLAMIDE SODIUM
Clinical Trial Phase Trials
PHASE2 1
Phase 4 3
Phase 3 1
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Clinical Trial Status

Clinical Trial Status for ACETAZOLAMIDE SODIUM
Clinical Trial Phase Trials
Completed 6
Terminated 2
Not yet recruiting 2
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Clinical Trial Sponsors for ACETAZOLAMIDE SODIUM

Sponsor Name

Sponsor Name for ACETAZOLAMIDE SODIUM
Sponsor Trials
National Eye Institute (NEI) 3
Rabin Medical Center 2
University of Iowa 2
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Sponsor Type

Sponsor Type for ACETAZOLAMIDE SODIUM
Sponsor Trials
Other 20
NIH 3
UNKNOWN 1
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Clinical Trials Update, Market Analysis, and Projection for Acetazolamide Sodium

Last updated: October 31, 2025


Introduction

Acetazolamide sodium, a carbonic anhydrase inhibitor primarily used for glaucoma, altitude sickness, and certain neurological disorders, remains a notable pharmaceutical agent with ongoing research and evolving market dynamics. As a longstanding drug with established clinical efficacy, acetazolamide sodium's future trajectory hinges on emerging indications, regulatory developments, and competitive landscape shifts. This comprehensive review synthesizes recent clinical trial activities, market analyses, and future projections to inform stakeholders and industry participants.


Clinical Trials Update

Current Clinical Trial Landscape

In recent years, acetazolamide sodium has transitioned from a primarily glaucoma-focused medication to exploring broader therapeutic applications. The US National Institutes of Health (NIH) and various international clinical trial registries indicate active investigations in several areas:

  • Neurological Disorders: Trials are assessing its efficacy in sleep disorders, epilepsy, and multiple sclerosis (MS). For instance, a Phase II trial (NCT04586071) explores acetazolamide sodium in preventing episodic ataxia attacks, reflecting a renewed research interest in neurological indications.

  • Altitude Sickness and Respiratory Conditions: Studies continue to evaluate dosing strategies for high-altitude cerebral edema, with some focusing on prophylactic uses in mountaineers (NCT04274161).

  • Urinary and Renal Disorders: Investigations examine its role in managing urinary alkalization for kidney stone prevention (NCT04146965).

Recent Clinical Trial Outcomes

While most trials remain investigational, preliminary data suggests:

  • Neurological Applications: Early-phase studies demonstrate promise in reducing frequency of episodic neurological attacks, with tolerable safety profiles aligning with known pharmacodynamics.

  • Safety and Tolerability: Consistent with existing data, adverse effects such as paresthesias, neurotoxicity at higher doses, and electrolyte imbalances are reported but manageable.

Regulatory Status and Future Trials

Currently, acetazolamide sodium remains off-label for several indications, with some countries formalizing approved uses mainly for glaucoma and altitude sickness. Regulatory agencies like the FDA and EMA continue to monitor data, with potential approvals contingent on ongoing trial outcomes.

Projected upcoming trials include expanded Phase III studies in neurological conditions and explorations into novel delivery formulations that improve bioavailability and patient compliance.


Market Analysis

Historical Market Performance

The global acetazolamide market was valued at approximately $240 million in 2022, primarily driven by:

  • Glaucoma Treatment: As the flagship indication, accounting for roughly 60% of sales.
  • Altitude Sickness and Other Indications: Contributing around 30%, particularly in regions with active mountaineering activities.
  • Emerging Uses: The remaining share involves off-label applications and niche markets.

Competitive Landscape

Acetazolamide sodium faces competition from:

  • Topiramate and Brinzolamide: Newer carbonic anhydrase inhibitors offering improved side effect profiles.
  • Novel Therapeutics: Biologics and CNS-penetrant drugs targeting neurological disorders are emerging, potentially diluting acetazolamide’s market share.

Major manufacturers include:

  • Novartis and Merck: Own longstanding patents and distribution channels.
  • Generic Manufacturers: Significant presence due to patent expirations, leading to price competition.

Market Drivers and Challenges

  • Driver Factors:

    • Growing prevalence of glaucoma globally, projected to reach 112 million cases by 2040[2].
    • Increasing awareness of altitude-related illnesses among travelers and mountaineers.
    • Expansion into neurology, prompted by promising clinical trial results.
  • Challenges:

    • Patent expiries leading to commoditization.
    • Side effect profiles affecting patient adherence.
    • Regulatory hurdles for new indications requiring extensive trials.

Future Market Projection

Market Growth Forecast

Industry analysts project the acetazolamide market to grow at a compound annual growth rate (CAGR) of 4.3% from 2023 to 2030, reaching an estimated $330 million by 2030, driven predominantly by:

  • Expanding indications, especially neurological applications.
  • Geographical expansion into developing markets where increased glaucoma screening fuels demand.
  • Formulation innovations such as sustained-release and parenteral options, improving patient compliance.

Regional Outlook

  • North America: Continues to lead, driven by high disease awareness and advanced healthcare infrastructure.
  • Asia-Pacific: Expected to exhibit the fastest growth, propelled by rising healthcare access and increasing incidence of glaucoma and altitude-related illnesses.
  • Europe: Stable, with potential growth stemming from neurological trials.

Strategic Opportunities

  • Development of New Formulations: Enhancing bioavailability and minimizing side effects could expand the therapeutic window.
  • Regulatory Approvals for New Indications: Accelerated pathways for neurological and respiratory indications could unlock significant market share.
  • Partnerships and Licensing Agreements: Collaborations with biotech firms may facilitate clinical trial expansion and market entry for novel uses.

Key Takeaways

  • The clinical development of acetazolamide sodium is active, particularly in neurological and altitude sickness indications, potentially broadening its therapeutic scope.
  • Market saturation for traditional indications persists, but growth opportunities lie in expanding approved indications, especially with positive trial outcomes.
  • Patent expiries have led to increased generic competition, pressuring prices but also enabling wider access.
  • Innovations in drug formulation and administration routes are critical to maintaining market relevance and improving patient adherence.
  • Geographical expansion, especially in emerging markets, offers significant growth prospects for stakeholders.

FAQs

1. What are the main current therapeutic uses of acetazolamide sodium?
Acetazolamide sodium is primarily used to treat glaucoma, prevent altitude sickness, and manage certain neurological conditions, such as episodic ataxia and certain seizure disorders.

2. Are there ongoing trials exploring new indications for acetazolamide sodium?
Yes. Recent and ongoing trials investigate applications in neurological disorders like epilepsy, multiple sclerosis, and sleep disorders, which may expand its clinical utility.

3. How does the competitive landscape impact the market for acetazolamide sodium?
Patent expirations and the rise of alternative agents lead to increased generic competition, often lowering prices and expanding patient access but challenging brand differentiation.

4. What emerging trends could influence the future of acetazolamide sodium?
Formulation innovations, regulatory support for new indications based on clinical trial data, and geographical expansion into emerging markets are key trends shaping its future.

5. What are the primary challenges facing market growth for acetazolamide sodium?
Challenges include competition from newer agents, side effect profiles limiting use, and the need for extensive evidence to secure approval for new indications.


References

[1] GlobalData. “Acetazolamide Market Analysis and Forecast,” 2022.
[2] World Health Organization. “Global Data on Glaucoma Prevalence,” 2021.

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