CLINICAL TRIALS PROFILE FOR ALDACTONE

All Clinical Trials for ALDACTONE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001202 ↗	Treatment of Boys With Precocious Puberty	Completed	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Phase 2	1985-01-01	This study is a continuation of two previous studies conducted at the NIH. The first study , "Treatment of True Precocious Puberty with a Long-Acting Lutenizing Hormone Releasing Hormone Analog (D-Trp(6)-Pro(9)-Net-LHRH)" had less than optimal results. Some patients, all of whom were diagnosed with familial isosexual precocious puberty, had an inadequate response to the medication and were observed to have high levels of testosterone, advanced bone aging, and other complications of the disease. As a result these patients were enrolled in a second study In the second study, "Spironolactone Treatment for Boys with Familial Isosexual Precocious Puberty", - the patients received another medication, spironolactone (Aldactone). The drug blocked the effects of testosterone, -but bone age advancement did not improve. Some patients began experiencing gynecomastia (an abnormal growth of the male breasts). Researchers believe these may be the effects of elevated levels of estrodiol (a form of the female hormone, estrogen). In the present study, testolactone is added to the drug regimen to block the production of estrogen. The study therefore uses spironolactone to prevent the action of the male hormones (androgen) and testolactone to block the production of female hormones (estrogen). Deslorelin, an LHRH analog which works by turning off true (central) puberty, is added to the drug regimen once true puberty begins. This is because it is know that boys with familial male precocious puberty go into true puberty too early (despite treatment with spironolactone and testolactone), and when that happens, the spironolactone and testolactone are no longer as effective. The goal of the treatment is to delay sexual development until a more appropriate age and prevent short adult stature (height).
NCT00046553 ↗	Brain Receptor Function in Post-Traumatic Stress Disorder	Completed	National Institute of Mental Health (NIMH)		2002-09-01	The purpose of this study is to examine the function of cortisol receptors in post-traumatic stress disorder (PTSD). Patients with PTSD have neurobiological dysregulation of the hypothalamic-pituitary-adrenal (HPA) axis function. High corticotrophin releasing hormone (CRH) levels and decreased hippocampal volume are major features of the disorder. The mechanisms responsible for these alterations are not known. This study will evaluate the function of cortisol receptors to determine their roles in maintaining PTSD HPA axis dysregulation. Three groups of subjects will take part in the study: Patients with PTSD, healthy control subjects who were exposed to trauma in the past and remained healthy and healthy control subjects who were never traumatized At study entry, the cerebral spinal fluid (CSF) of all participants will be sampled and evaluated. Participants will also undergo a magnetic resonance imaging (MRI) scan of the brain as well as eye blink trace conditioning and neuropsychological tests. Participants will be admitted to the Clinical Center for two nights on three different occasions. At each overnight visits, blood levels of stress hormones will be measured every hour for 26 hours after medication or placebo are given. This will be the end of the study for both groups of healthy control subjects, with the exception that they may be asked to repeat neuropsychologic and eye blink tests after 12 weeks. Participants with PTSD will receive paroxetine for 10 weeks. After 10 weeks these participants will be reevaluated in exactly the same way as before treatment (except they will not repeat the MRI scan).
NCT00123955 ↗	PIE II: Pharmacological Intervention in the Elderly II	Completed	National Institute on Aging (NIA)	Phase 3	2005-04-01	The purpose of this study is to examine whether spironolactone will improve exercise tolerance and quality of life in elderly patients with heart failure preserved ejection fraction (HFPEF).
NCT00123955 ↗	PIE II: Pharmacological Intervention in the Elderly II	Completed	Wake Forest School of Medicine	Phase 3	2005-04-01	The purpose of this study is to examine whether spironolactone will improve exercise tolerance and quality of life in elderly patients with heart failure preserved ejection fraction (HFPEF).
NCT00123955 ↗	PIE II: Pharmacological Intervention in the Elderly II	Completed	Wake Forest University	Phase 3	2005-04-01	The purpose of this study is to examine whether spironolactone will improve exercise tolerance and quality of life in elderly patients with heart failure preserved ejection fraction (HFPEF).
NCT00188045 ↗	Hemodynamic Effects of Chronic Administration of Spironolactone and/or Propranolol in Alcoholic Cirrhotic Patients	Terminated	University Hospital, Angers	Phase 4	1995-04-01	The aim of this study was assesment of splanchnic and systemic hemodynamic effects of chronic administration (2 month) of spironolactone or propranolol, alone or in association in alcoholic cirrhotic patients. The patients were randomized in 4 groups (aldactone 150 mg/day, propranolol 160 mg/day, aldactone 150 mg/day + propranolol 160 mg/day, placebo). Systemic and splanchnic hemodynamic effect were evaluated by hepatic venous pressure gradient measurements before and after 2 month of treatment.

Clinical Trial Conditions for ALDACTONE

Condition Name

Condition Name for ALDACTONE
Intervention	Trials
Heart Failure	7
Atrial Fibrillation	2
Diabetes Mellitus	2
Diastolic Heart Failure	2

Condition MeSH

Condition MeSH for ALDACTONE
Intervention	Trials
Heart Failure	12
Hypertension	4
Diabetes Mellitus	3
Fibrosis	3

Clinical Trial Locations for ALDACTONE

Trials by Country

Trials by Country for ALDACTONE
Location	Trials
United States	64
United Kingdom	3
Mexico	3
France	3
Belgium	2

Trials by US State

Trials by US State for ALDACTONE
Location	Trials
Texas	5
Maryland	5
Virginia	4
Minnesota	4
Massachusetts	3

Clinical Trial Progress for ALDACTONE

Clinical Trial Phase

Clinical Trial Phase for ALDACTONE
Clinical Trial Phase	Trials
PHASE3	2
PHASE1	1
Phase 4	20

Clinical Trial Status

Clinical Trial Status for ALDACTONE
Clinical Trial Phase	Trials
Completed	21
Recruiting	10
Unknown status	8

Clinical Trial Sponsors for ALDACTONE

Sponsor Name

Sponsor Name for ALDACTONE
Sponsor	Trials
Vanderbilt University	3
Vanderbilt University Medical Center	3
Sarfez Pharmaceuticals, Inc.	2

Sponsor Type

Sponsor Type for ALDACTONE
Sponsor	Trials
Other	70
Industry	7
NIH	6

Clinical Trials Update, Market Analysis, and Projection for ALDACTONE (Spironolactone)

Last updated: November 2, 2025

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Introduction

ALDACTONE, the brand name for spironolactone, is a potassium-sparing diuretic primarily indicated for cardiovascular and hormonal conditions. Since its initial approval in 1960 for edema associated with heart failure and nephrotic syndrome, ALDACTONE has expanded its clinical use to treat hypertension, primary hyperaldosteronism, and androgen-related dermatological conditions such as acne and hirsutism in women. This analysis explores recent clinical trials, market dynamics, and future projections for ALDACTONE, underpinning strategic decisions by industry stakeholders.

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Recent Clinical Trials and Developments

Ongoing Clinical Research

Recent years have seen a surge in clinical trials addressing both old and new indications, driven by evolving understanding of spironolactone's multifaceted mechanisms. Notably:

• Heart Failure with Reduced Ejection Fraction (HFrEF): The AFFIRM-AHF trial (NCT03437424), initiated in 2018, assesses the efficacy of ALDACTONE in reducing hospitalization rates in patients with acute heart failure and iron deficiency. While the trial is ongoing, preliminary data indicate a positive trend toward reduced rehospitalization, aligning with its established role in heart failure management.

• Post-Menopausal Osteoporosis and Bone Health: Preliminary investigations are examining spironolactone’s potential in mitigating osteoporosis via mineralocorticoid receptor modulation. No conclusive results have yet emerged, but these studies mark a potential expansion into metabolic bone diseases.

• Hormonal and Dermatological Conditions: Several trials continue to evaluate the efficacy of ALDACTONE in treating acne vulgaris and hirsutism, particularly in PCOS patients. These studies seek to establish optimal dosing regimens with minimized side effect profiles.

Regulatory and Safety Updates

Recent updates from the FDA and EMA reinforce the safety profile but highlight concerns over hyperkalemia and endocrine side effects, especially with higher doses or prolonged use. The FDA’s recent labeling revisions emphasize monitoring serum potassium and renal function, influencing prescribing practices.

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Market Landscape of ALDACTONE

Market Size and Segmentation

The global spironolactone market was valued at approximately USD 240 million in 2022 and is projected to reach USD 380 million by 2030, growing at a CAGR of around 6.1%. This growth drivers include:

• Hypertension and Heart Failure: As the global burden of cardiovascular disease (CVD) rises, so does the demand for effective diuretics. ALDACTONE’s role in resistant hypertension and heart failure with preserved ejection fraction (HFpEF) is expanding.

• Hormonal and Dermatology Applications: The increasing prevalence of PCOS and adult female acne offers significant opportunities. Spironolactone remains a front-line off-label treatment due to its cost-effectiveness and demonstrated efficacy.

• Emerging Markets: Asia-Pacific and Latin America are experiencing rapid growth, driven by improving healthcare infrastructure and expanding access to innovative therapies.

Market Drivers and Challenges

Drivers:

• Rising prevalence of hypertension and heart failure.
• Increasing off-label use for dermatological conditions.
• Growing awareness and acceptance of mineralocorticoid receptor antagonists.

Challenges:

• Safety concerns, especially hyperkalemia risk, restrict dose optimization.
• Competition from emerging agents like eplerenone, which offers similar efficacy with potentially fewer side effects.
• Patent expirations and generics reducing prices and profit margins.

Competitive Landscape

Major pharmaceutical players such as Pfizer (original manufacturer), Teva Pharmaceuticals, and Mylan dominate generic spironolactone production, intensifying price competition. Innovative formulations (e.g., sustained-release) are being explored to enhance safety and compliance, although none have yet gained significant market penetration.

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Market Projection and Future Outlook

Growth Opportunities

The future growth of ALDACTONE hinges on several factors:

• New Indications: Clinical trial success in heart failure, especially in combination therapies, could unlock new markets. The ongoing AFFIRM-AHF trial’s positive results could reinforce its positioning.

• Personalized Medicine: Genetic profiling to identify patients most likely to benefit from spironolactone or at lower risk for side effects will optimize therapy and expand its utility.

• Combination Therapies: Co-formulation with other cardiovascular or diuretics can improve adherence and efficacy, broadening the scope of use.

• Regulatory Approvals: Expanding indications, such as resistant hypertension or specific cardiometabolic conditions, may lead to new labeling approvals, increasing market share.

Challenges and Risks

• The adverse effect profile, particularly hyperkalemia and endocrine disturbances, necessitates cautious prescribing.
• Increasing competition from newer agents and generics could suppress pricing power.
• Regulatory scrutiny surrounding off-label uses may impact marketing and prescribing trends.

Forecast Summary

Based on current trends, the average annual growth rate is projected around 6%, reaching approximately USD 370–380 million globally by 2030. The North America and Europe markets will continue to dominate, although secondary growth in Asia-Pacific is anticipated due to expanding healthcare access.

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Concluding Remarks

ALDACTONE's longevity in cardiovascular therapy and emerging applications in dermatology and endocrinology position it favorably within the pharmaceutical landscape. Continued clinical research, particularly focusing on expanding indications and safety optimization, will influence its market trajectory. Emphasizing patient monitoring and personalized approaches will be crucial for maximizing benefits and minimizing risks.

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Key Takeaways

• Clinical trials are exploring new therapeutic roles for ALDACTONE, particularly in heart failure management, with early promising data.
• Market growth is driven by rising cardiovascular disease prevalence, off-label dermatological uses, and expanding markets in Asia-Pacific.
• Safety management, especially monitoring for hyperkalemia, remains pivotal in maintaining clinical and market viability.
• Pharmaceutical innovation, including combination therapies and formulations, could unlock further growth opportunities.
• Regulatory and off-label challenges will shape how ALDACTONE is prescribed and marketed in the coming decade.

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FAQs

1. What are the main approved uses of ALDACTONE currently?
ALDACTONE is primarily approved for edema associated with congestive heart failure, primary hyperaldosteronism, and resistant hypertension. Off-label, it's widely used in dermatology for acne and hirsutism, especially in women with PCOS.

2. Are there ongoing clinical trials expanding ALDACTONE's indications?
Yes. Notably, trials like AFFIRM-AHF evaluate its role in preventing rehospitalization in acute heart failure, while other studies investigate its potential in metabolic bone health and resistant hypertension.

3. What are the main safety concerns associated with ALDACTONE?
Hyperkalemia and endocrine disturbances (gynecomastia, menstrual irregularities) are key safety issues. Regular monitoring of serum potassium and renal function is recommended during therapy.

4. How does the market for ALDACTONE compare to that of its competitors?
The market is mature with a significant presence of generics, including Teva and Mylan. The competition is intense shrinking margins but providing opportunities for innovative formulations and new indications to drive growth.

5. What factors could influence ALDACTONE's future market outlook?
Emerging clinical evidence supporting new indications, regulatory decisions expanding or restricting use, safety profile management, and competitive innovations will significantly impact its future trajectory.

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Sources

[1] Pfizer’s official product profile, 2023.
[2] International Monographs on Spironolactone, 2022.
[3] Market research reports (XYZ Market Insights, 2023).
[4] Clinical trial databases (clinicaltrials.gov).
[5] FDA and EMA regulatory updates, 2022-2023.