Clinical Trials

Numerous clinical trials evaluate the therapeutic efficacy and pharmacokinetics of amiloride across diverse indications, primarily targeting cardiovascular and renal disorders—such as hypertension, proteinuria, diabetic nephropathies, and pre-hypertension—alongside cystic fibrosis, bipolar disorder, and malignancies including triple-negative and HER2-low breast cancer. Spanning Early Phase 1 through Phase 4, these studies encompass completed, active, recruiting, and terminated recruitment statuses. The trials are sponsored by academic medical centers, university health networks, and clinical research foundations, including Assistance Publique - Hôpitaux de Paris and Georgetown University.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT06224673 RECRUITING
HER2 Low Breast Carcinoma; Triple Negative Breast Cancer; Hormone-receptor-positive Breast Cancer; Hormone Receptor Positive Breast Carcinoma
Laura Huppert, MD, BA
 2026-06-05 PHASE2
NCT05753059 RECRUITING
Heart Failure
Yale University
 2023-08-10 PHASE1
NCT04063540 RECRUITING
Migraine With Aura
Centre Hospitalier Universitaire de Nice
 2020-08-11 PHASE2
NCT07634068 NOT_YET_RECRUITING
Vertebrobasilar Dolichoectasia
Huashan Hospital
 2026-06 PHASE2
NCT06923709 RECRUITING
Chronic Kidney Disease(CKD); Proteinuria
Odense University Hospital
 2024-10-10 PHASE2
NCT05228574 COMPLETED
Autosomal Dominant Polycystic Kidney
Erasmus Medical Center
 2022-03-11 PHASE4
NCT05044611 ACTIVE_NOT_RECRUITING
Bipolar Disorder
Assistance Publique - Hôpitaux de Paris
 2023-01-11 PHASE4
NCT03837626 COMPLETED
Overweight and Obesity; Insulin Resistance
University of Missouri-Columbia
 2019-07-15 PHASE2; PHASE3
NCT04331691 COMPLETED
Resistant Hypertension
Yonsei University
 2020-11-16 PHASE4
NCT02847338 COMPLETED
Hypertension
Cambridge University Hospitals NHS Foundation Trust
 2016-11 PHASE4
NCT05044611 Recruiting
Bipolar Disorder
Assistance Publique - Hôpitaux de Paris
 2023-01-11 Phase 4
NCT05079789 TERMINATED
Nephrotic Syndrome; Edema; Sodium Retention
University Hospital Tuebingen
 2020-06-08 PHASE3
NCT04181008 COMPLETED
Pharmacokinetics
University of Utah
 2020-09-28 EARLY_PHASE1
NCT02525796 COMPLETED
Primary Hyperparathyroidism
Brigham and Women's Hospital
 2016-01 PHASE2; PHASE3
NCT02522650 UNKNOWN
Proteinuria
Georgetown University
 2013-07 PHASE4
NCT03036748 COMPLETED
Kidney Transplant; Complications
University of Southern Denmark
 2017-01-01
NCT03928145 UNKNOWN
Hypertension
Hospital de Clinicas de Porto Alegre
 2019-11-13 PHASE3
NCT02497300 COMPLETED
Chronic Kidney Disease; Albuminuria
University of Alabama at Birmingham
 2015-03 PHASE2
NCT04181008 Completed
Pharmacokinetics
University of Utah|Center for Addiction and Mental Health
 2020-09-28 Early Phase 1
NCT03170336 COMPLETED
Chronic Kidney Diseases
Guangdong Provincial People's Hospital
 2018-03-01 PHASE4
NCT06416735 COMPLETED
Hypertension Essential; Salt Excess; Genetic Hypertension
Ospedale San Raffaele
 2018-09-28 PHASE3
NCT02323100 Terminated
Cystic Fibrosis
National Jewish Health|University of Alabama at Birmingham|Children''s Hospital of Philadelphia|Johns Hopkins University|Horizon Pharma Ireland Ltd. Dublin Ireland
 2018-12-02 Phase 1|Phase 2
NCT01910259 COMPLETED
Secondary Progressive Multiple Sclerosis
University College, London
 2014-12-18 PHASE2
NCT02875886 COMPLETED
Chronic Kidney Disease
Erasmus Medical Center
 2016-09 PHASE4
NCT02832973 COMPLETED
Hypertension Resistant to Conventional Therapy
Sao Jose do Rio Preto Medical School
 2015-09 PHASE4
NCT03031496 COMPLETED
Hypertension
GlaxoSmithKline
 2017-03-17 PHASE1
NCT01879527 UNKNOWN
Optic; Neuritis, With Demyelination
Medical University of Vienna
 2014-03 PHASE2
NCT02896621 COMPLETED
Hypertension; Sleep Apnea
Hospital de Clinicas de Porto Alegre
 2014-12-07 PHASE3
NCT01802489 COMPLETED
Optic Neuritis; Multiple Sclerosis
University of Oxford
 2013-02 PHASE2
NCT01733680 TERMINATED
ADHD
State University of New York - Upstate Medical University
 2012-09 EARLY_PHASE1
NCT01195805 COMPLETED
Essential Hypertension
Regional Hospital Holstebro
 2010-08
NCT02351973 UNKNOWN
Hypertension
University of Cambridge
 2009-11 PHASE4
NCT01918488 UNKNOWN
Diabetic Nephropathies; Hypertension
University of Southern Denmark
 2013-10
NCT01804777 TERMINATED
Proteinuria; Hypertension; Type II Diabetes
University of New Mexico
 2013-03 EARLY_PHASE1
NCT00970931 COMPLETED
Pre-hypertension; Hypertension; Cardiovascular Disease
Hospital de Clinicas de Porto Alegre
 2010-07 PHASE3
NCT00971165 COMPLETED
Hypertension; Cardiovascular Disease
Hospital de Clinicas de Porto Alegre
 2010-07 PHASE3
NCT01635231 COMPLETED
Nephropathy
Regional Hospital Holstebro
 2012-07
NCT01191450 COMPLETED
Arterial Hypertension
Eurofarma Laboratorios S.A.
 2011-08 PHASE3
NCT01228214 UNKNOWN
Coronary Artery Disease
University of Carabobo
 2011-03 PHASE2; PHASE3
NCT01210365 TERMINATED
Congestive Heart Failure
Eurofarma Laboratorios S.A.
 2011-01-01 PHASE3
NCT01308983 UNKNOWN
Prehypertension
Augusta University
 2010-09 PHASE4
NCT00478335 COMPLETED
Nephrogenic Diabetes Insipidus
University of Colorado, Denver
 2007-05
NCT01011660 UNKNOWN
Essential Hypertension
Chinese Academy of Medical Sciences, Fuwai Hospital
 2007-10 PHASE4
NCT02122731 COMPLETED
Hypertension; Type 2 Diabetes Mellitus; Microalbuminuria
Ib Abildgaard Jacobsen
 2010-11 PHASE4
NCT01706887 TERMINATED
Hypertension
Assistance Publique - Hôpitaux de Paris
 2007-04
NCT01388088 COMPLETED
Hypertension
Erling Bjerregaard Pedersen
 2010-09 PHASE4
NCT01095133 COMPLETED
Gastroesophageal Reflux Disease; Heartburn
University of North Carolina, Chapel Hill
 2010-03 PHASE1
NCT01246180 COMPLETED
Physiological Conditions
Hospices Civils de Lyon
 2010-10
NCT00709137 WITHDRAWN
Hypertension
VA Salt Lake City Health Care System
 2008-10
NCT00857909 COMPLETED
High Blood Pressure
Regional Hospital Holstebro
 2009-01 PHASE1
NCT01231165 COMPLETED
Coronary Heart Disease
University of Carabobo
 2009-02 PHASE2; PHASE3
NCT00424801 TERMINATED
Microvascular Angina; Hypertension
University of Aarhus
 2007-01
NCT00547053 COMPLETED
Cystic Fibrosis
Boston Children's Hospital
 2006-12 PHASE1
NCT00285805 COMPLETED
Insulin Resistance
Radboud University Medical Center
 2006-02
NCT00274391 COMPLETED
Cystic Fibrosis
University of North Carolina
 2001-07 PHASE2
NCT00394394 COMPLETED
Hypertension
Hospital de Clinicas de Porto Alegre
 2005-02 PHASE2
NCT00141596 TERMINATED
Hypertension
St George's, University of London
 2003-07
NCT00004705 COMPLETED
Cystic Fibrosis
FDA Office of Orphan Products Development
 1996-09
NCT00429897 UNKNOWN
Low-Renin Hypertension
University of Cambridge
 2006-08

(data from https://clinicaltrials.gov, updated on 2026-06-11)

Check the Amiloride HCl product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Amiloride selectively inhibits epithelial sodium channels, T-type calcium channels, and urokinase plasminogen activator, thereby blocking transepithelial sodium reabsorption in renal tubule epithelial cells. This inhibition prevents luminal membrane hyperpolarization and decreases potassium ion excretion, establishing the functional rationale for its therapeutic application in hypertension, proteinuria, and cardiovascular disease.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.