CLINICAL TRIALS PROFILE FOR AMILORIDE HYDROCHLORIDE

505(b)(2) Clinical Trials for AMILORIDE HYDROCHLORIDE

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT00590538 ↗	Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)	Terminated	Cystic Fibrosis Foundation	Phase 1/Phase 2	2003-02-01	The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most common genetic mutation found in patients with CF is called Delta F508. Due to this mutation, there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines, pancreas and sweat glands. This lack of movement causes the clinical manifestations of the disease. Although Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein is a naturally occurring substance that is found in food products such as soy and tofu, but is also an investigational drug for this study. When used together, both drugs may be able to restore normal chloride and salt (water) movements in body organs and glands in people with CF. We will be studying salt and water movement in the nose by a technique called nasal transepithelial potential difference (NPD).
New Combination	NCT00590538 ↗	Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)	Terminated	Cystic Fibrosis Foundation Therapeutics	Phase 1/Phase 2	2003-02-01	The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most common genetic mutation found in patients with CF is called Delta F508. Due to this mutation, there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines, pancreas and sweat glands. This lack of movement causes the clinical manifestations of the disease. Although Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein is a naturally occurring substance that is found in food products such as soy and tofu, but is also an investigational drug for this study. When used together, both drugs may be able to restore normal chloride and salt (water) movements in body organs and glands in people with CF. We will be studying salt and water movement in the nose by a technique called nasal transepithelial potential difference (NPD).
New Combination	NCT00590538 ↗	Phenylbutyrate/Genistein Duotherapy in Delta F508-Heterozygotes (for Cystic Fibrosis)	Terminated	Children's Hospital of Philadelphia	Phase 1/Phase 2	2003-02-01	The purpose of this research study is to test a new combination of medicines, Phenylbutyrate and Genistein, to determine if they could be used to treat cystic fibrosis (CF). The most common genetic mutation found in patients with CF is called Delta F508. Due to this mutation, there is a lack of salt (chloride) movement in your nose, sinuses, lungs, intestines, pancreas and sweat glands. This lack of movement causes the clinical manifestations of the disease. Although Phenylbutyrate has been extensively used to treat patients with rare metabolic diseases, Phenylbutyrate is an investigational drug for the purpose of this study. Genistein is a naturally occurring substance that is found in food products such as soy and tofu, but is also an investigational drug for this study. When used together, both drugs may be able to restore normal chloride and salt (water) movements in body organs and glands in people with CF. We will be studying salt and water movement in the nose by a technique called nasal transepithelial potential difference (NPD).

All Clinical Trials for AMILORIDE HYDROCHLORIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00224549 ↗	PHARES Study: Management of Resistant Hypertension	Completed	Assistance Publique - Hôpitaux de Paris	Phase 4	2005-04-01	The purpose of this study is to assess the efficacy of two different treatment regimens for treating resistant hypertension previously uncontrolled with at least 3 antihypertensive treatments. The study hypothesis is that these two regimens (one based on increasing diuretics and the other based on increasing renin angiotensin system blockage) may not differ in terms of efficacy.
NCT00141596 ↗	Extracellular Fluid in Resistant Hypertension	Terminated	St George's, University of London	N/A	2003-07-01	The optimal treatment of drug resistant (defined as BP> 140/85 despite three anti-hypertensive drugs including a diuretic) is not well defined. This study aims to test the hypothesis that resistant hypertension is caused by excessive expansion of extracellular fluid volume. A secondary objective is to study which of three different antihypertensive drugs would be most useful in drug resistant hypertension.
NCT00274391 ↗	Efficacy of Amiloride and Hypertonic Saline in Cystic Fibrosis	Completed	Cystic Fibrosis Foundation	Phase 2	2001-07-01	The purpose of this research study is to determine whether the combination of inhaled amiloride and a concentrated salt solution is better than the salt solution itself for cystic fibrosis (CF) patients. In CF, airway secretions are thick and dehydrated. Many patients use inhaled salt solutions to help draw water into their secretions so that they are easier to get rid of with chest physiotherapy ("chest PT") and cough. Unfortunately, these salt solutions are reabsorbed very quickly by the airways, so the beneficial effects may not last very long. In the hopes of prolonging their effects, the drug amiloride could be used in combination to slow salt and water reabsorption from airways. Amiloride is a medication that has been given by mouth for high blood pressure for many years. It is possible that the combination of salt solutions and inhaled amiloride may significantly improve the clearance of secretions in CF, which would be expected to improve lung function in CF.
NCT00004705 ↗	Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis	Completed	University of North Carolina	N/A	1996-09-01	OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II. Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.
NCT00004705 ↗	Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis	Completed	FDA Office of Orphan Products Development	N/A	1996-09-01	OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II. Determine the acute safety and efficacy of aerosolized UTP in children with cystic fibrosis.
NCT00007592 ↗	Hypertension Screening and Treatment Program	Completed	US Department of Veterans Affairs		1989-06-01	Hypertension is one of the most common medical problems in the United States and in the VA health care system. It has been well-documented that hypertension can be effectively treated. However, there remain important unresolved clinical questions in the area of antihypertensive treatment. For example, how much is mortality affected by visit compliance, blood pressure control and type of antihypertensive agent? Or, are some regimens associated with more morbidity than others? Or, are there inexpensive regimens that are as effective as more expensive regimens? The amount of data that is available from this demonstration project (currently 6,100 patients) will help address these questions. The answers to these questions should result in better care for veterans with hypertension.
NCT00007592 ↗	Hypertension Screening and Treatment Program	Completed	VA Office of Research and Development		1989-06-01	Hypertension is one of the most common medical problems in the United States and in the VA health care system. It has been well-documented that hypertension can be effectively treated. However, there remain important unresolved clinical questions in the area of antihypertensive treatment. For example, how much is mortality affected by visit compliance, blood pressure control and type of antihypertensive agent? Or, are some regimens associated with more morbidity than others? Or, are there inexpensive regimens that are as effective as more expensive regimens? The amount of data that is available from this demonstration project (currently 6,100 patients) will help address these questions. The answers to these questions should result in better care for veterans with hypertension.

Clinical Trial Conditions for AMILORIDE HYDROCHLORIDE

Condition Name

Condition Name for AMILORIDE HYDROCHLORIDE
Intervention	Trials
Hypertension	18
Cystic Fibrosis	7
Bipolar Disorder	2
Cardiovascular Disease	2

Condition MeSH

Condition MeSH for AMILORIDE HYDROCHLORIDE
Intervention	Trials
Hypertension	23
Fibrosis	7
Cystic Fibrosis	7
Kidney Diseases	4

Clinical Trial Locations for AMILORIDE HYDROCHLORIDE

Trials by Country

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−

Trials by Country for AMILORIDE HYDROCHLORIDE
Location	Trials
United States	31
Brazil	14
India	8
France	8
United Kingdom	7

Trials by US State

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−

Trials by US State for AMILORIDE HYDROCHLORIDE
Location	Trials
North Carolina	3
Pennsylvania	3
Alabama	2
Maryland	2
Utah	2

Clinical Trial Progress for AMILORIDE HYDROCHLORIDE

Clinical Trial Phase

Clinical Trial Phase for AMILORIDE HYDROCHLORIDE
Clinical Trial Phase	Trials
Phase 4	17
Phase 3	9
Phase 2/Phase 3	5

Clinical Trial Status

Clinical Trial Status for AMILORIDE HYDROCHLORIDE
Clinical Trial Phase	Trials
Completed	34
Recruiting	9
Unknown status	8

Clinical Trial Sponsors for AMILORIDE HYDROCHLORIDE

Sponsor Name

Sponsor Name for AMILORIDE HYDROCHLORIDE
Sponsor	Trials
Assistance Publique - Hôpitaux de Paris	6
Hospital de Clinicas de Porto Alegre	6
Cystic Fibrosis Foundation	3

Sponsor Type

Sponsor Type for AMILORIDE HYDROCHLORIDE
Sponsor	Trials
Other	126
Industry	7
U.S. Fed	4

Amiloride Hydrochloride: Clinical Trials, Market Analysis, and Projections

Introduction to Amiloride Hydrochloride

Amiloride hydrochloride is a potassium-sparing diuretic commonly used in the treatment of hypertension, heart failure, and edema. Its mechanism of action involves inhibiting the sodium channels in the collecting ducts of the kidneys, thereby reducing sodium reabsorption and potassium excretion.

Clinical Trials Update

Treatment of Nephrogenic Diabetes Insipidus in Bipolar Disorder Patients

One significant clinical trial currently underway is investigating the use of amiloride for the treatment of nephrogenic diabetes insipidus (NDI) in patients with bipolar disorder who are treated with lithium. This trial involves randomizing patients into two groups: one receiving 5mg of amiloride twice daily and the other receiving a placebo for two months. The trial also includes an open-label phase where the treatment allocation is revealed, and the experimental group continues with amiloride while the control group is followed up without treatment. The primary focus is on assessing renal functions and the safety of amiloride, particularly monitoring for hyperkalemia, a potential risk in patients with severe renal insufficiency[1].

Antimyeloma Activity

Another notable study has revealed the potent antimyeloma activity of amiloride. This research, conducted using myeloma cell lines and a xenograft mouse model, showed that amiloride induces apoptosis in multiple myeloma cells and has a synergistic effect when combined with other myeloma treatments like dexamethasone and melphalan. The study highlights the potential of amiloride as a therapeutic agent for multiple myeloma, especially in high-risk patients with TP53 abnormalities[3].

Market Analysis

Current Market Size and Growth

The amiloride hydrochloride market has experienced significant growth in recent years. As of 2022, the market size was valued at USD 0.85 billion and is projected to reach USD 1.35 billion by 2030, growing at a CAGR of 6.1% from 2024 to 2030. This growth is driven by the increasing prevalence of hypertension and heart failure, as well as the rising awareness of the therapeutic benefits of amiloride hydrochloride[4].

Key Drivers and Trends

The key drivers of the amiloride hydrochloride market include:

• Increasing Prevalence of Hypertension and Heart Failure: These conditions are becoming more common, leading to a higher demand for effective treatments like amiloride hydrochloride.
• Growing Elderly Population: The elderly are more prone to cardiovascular diseases, which in turn increases the demand for amiloride hydrochloride.
• Advancements in Clinical Research: Expanding indications for the drug and new formulations are creating new opportunities in the market[4].

Market Segments

The amiloride hydrochloride market can be segmented by:

• Dosage Form: Tablets and solutions are the primary forms in which amiloride hydrochloride is available.
• Application: The drug is primarily used for hypertension and heart failure, but its use in other conditions like nephrogenic diabetes insipidus and multiple myeloma is being explored.
• Distribution Channel: Hospital pharmacies and retail pharmacies are the main distribution channels[4].

Market Projections

Future Scope and Trends

The future of the amiloride hydrochloride market looks promising, with a forecasted CAGR that indicates robust growth. Key factors driving this growth include:

• Rising Consumer Demand: Increasing awareness and the need for effective treatments will drive demand.
• Developing Technologies: New formulations and delivery methods are expected to enhance market growth.
• Growing Applications: The potential use of amiloride hydrochloride in treating multiple myeloma and other conditions will expand its market scope[4].

Regional Outlook

The preferred regions for market expansion include North America, Europe, and Asia Pacific, due to the high prevalence of hypertension and heart failure in these areas. Emerging economies with growing healthcare infrastructure are also expected to contribute significantly to the market growth[4].

Challenges and Opportunities

Challenges

Despite the positive outlook, the amiloride hydrochloride market faces several challenges:

• Alternative Treatment Options: The presence of other diuretics and treatment options can compete with amiloride hydrochloride.
• Potential Side Effects: Hyperkalemia and other side effects need to be carefully managed to ensure patient safety[4].

Opportunities

The market also presents several opportunities:

• Expanding Indications: The potential use of amiloride hydrochloride in treating conditions like multiple myeloma and nephrogenic diabetes insipidus opens up new market avenues.
• Advancements in Drug Formulations: Improvements in drug delivery methods and new formulations can enhance patient compliance and efficacy[4].

Key Takeaways

• Clinical Trials: Amiloride hydrochloride is being investigated for new indications, including nephrogenic diabetes insipidus in bipolar disorder patients and multiple myeloma.
• Market Growth: The market is expected to grow significantly, driven by the increasing prevalence of hypertension and heart failure.
• Key Drivers: Growing elderly population, advancements in clinical research, and expanding indications are key drivers.
• Challenges and Opportunities: Managing side effects and exploring new indications are crucial for market growth.

FAQs

1. What is Amiloride Hydrochloride Used For?

Amiloride hydrochloride is primarily used to treat hypertension, heart failure, and edema. It is also being explored for other conditions such as nephrogenic diabetes insipidus and multiple myeloma.

2. What Are the Key Drivers of the Amiloride Hydrochloride Market?

The key drivers include the increasing prevalence of hypertension and heart failure, growing elderly population, and advancements in clinical research.

3. What Are the Potential Side Effects of Amiloride Hydrochloride?

One of the main risks associated with amiloride hydrochloride is hyperkalemia, particularly in patients with severe renal insufficiency.

4. What New Indications Are Being Explored for Amiloride Hydrochloride?

Amiloride hydrochloride is being investigated for the treatment of nephrogenic diabetes insipidus in bipolar disorder patients and for its antimyeloma activity.

5. What Is the Projected Market Size of Amiloride Hydrochloride by 2030?

The market size is projected to reach USD 1.35 billion by 2030, growing at a CAGR of 6.1% from 2024 to 2030.

Sources

1. CenterWatch: AMIloride for the Treatment of Nephrogenic Diabetes Insipidus for Patients With Bipolar Disorder Treated With Lithium.
2. Market Research Intellect: Global Amiloride Hydrochloride Market Size, Scope And Forecast Report.
3. AACR Journals: Amiloride, An Old Diuretic Drug, Is a Potential Therapeutic Agent for Multiple Myeloma.
4. Verified Market Reports: Amiloride Hydrochloride Market Key Drivers and Forecast 2025-2032.
5. EU Clinical Trials Register: Can the oral glucose tolerance test detect changes after 4 weeks treatment with the potassium sparing diuretic − amiloride hydrochloride?