CLINICAL TRIALS PROFILE FOR AMIKACIN SULFATE

All Clinical Trials for amikacin sulfate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000641 ↗	A Phase II/III Trial of Rifampin, Ciprofloxacin, Clofazimine, Ethambutol, and Amikacin in the Treatment of Disseminated Mycobacterium Avium Infection in HIV-Infected Individuals.	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	To compare the effectiveness and toxicity of two combination drug treatment programs for the treatment of disseminated Mycobacterium avium infection in HIV seropositive patients. [Per 03/06/92 amendment: to evaluate the efficacy of azithromycin when given in conjunction with either ethambutol or clofazimine as maintenance therapy.] Disseminated M. avium infection is the most common systemic bacterial infection complicating AIDS in the United States. The prognosis of patients with disseminated M. avium is extremely poor, particularly when it follows other opportunistic infections or is associated with anemia. Test tube studies and clinical data indicate that the best treatment program may include clofazimine, ethambutol, a rifamycin derivative, and ciprofloxacin. Test tube and animal studies indicate that amikacin is a bactericidal (bacteria destroying) drug that works better when used with ciprofloxacin. Its role in treatment programs is a key issue because of toxicity and because it must be administered parenterally (by injection or intravenously).
NCT00000796 ↗	A Prospective Study of Multidrug Resistance and a Pilot Study of the Safety of and Clinical and Microbiologic Response to Levofloxacin in Combination With Other Antimycobacterial Drugs for Treatment of Multidrug-Resistant Pulmonary Tuberculosis (MDR	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	N/A	1969-12-31	To determine the demographic, behavioral, clinical, and geographic risk factors associated with the occurrence of multidrug-resistant pulmonary tuberculosis (MDRTB). To evaluate the clinical and microbiological responses and overall survival of MDRTB patients who are treated with levofloxacin-containing multiple-drug regimens chosen from a hierarchical list. Per 9/28/94 amendment, to assess whether persistent or recurrent positive sputum cultures of patients who show failure or relapse are due to the same strain or reinfection with a new strain. Among TB patients, there has been an increase in progressive disease due to the emergence of antimycobacterial drug-resistant strains of Mycobacterium tuberculosis. Failure to identify patients at high risk for MDRTB increases the hazard for both treatment failure and development of resistance to additional therapeutic agents. Efforts to improve survival in patients with MDRTB will depend on improved methods of assessing the risk of acquisition of MDRTB and identifying drug susceptibility patterns in a timely fashion.
NCT00777296 ↗	Multidose Safety and Tolerability Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKACE™)	Completed	Insmed Incorporated	Phase 1/Phase 2	2007-02-22	A major factor in the respiratory health of cystic fibrosis (CF) subjects is acquisition of chronic Pseudomonas aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of CF subjects in the U.S. are infected. Liposomal Amikacin for Inhalation (Arikace™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of subjects infected via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug in close proximity to the bacterial colonies, thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating CF subjects with chronic infection caused by P. aeruginosa.
NCT03299452 ↗	Clinical Studies by Using Alphacait to Screen Drugs for Advanced Solid Tumor	Unknown status	Alphacait, LLC	Phase 2	2017-01-01	This is a single-center, open-label, single-arm, non-randomized study designed to evaluate PFS, safety, overall survival (OS), objective response rate (OPR), disease control rate (DCR) and biomarkers of cancer therapy based on Alphacait screening system in subjects with advanced malignant tumor.
NCT03299452 ↗	Clinical Studies by Using Alphacait to Screen Drugs for Advanced Solid Tumor	Unknown status	Haining Health-Coming Biotech Co., Ltd.	Phase 2	2017-01-01	This is a single-center, open-label, single-arm, non-randomized study designed to evaluate PFS, safety, overall survival (OS), objective response rate (OPR), disease control rate (DCR) and biomarkers of cancer therapy based on Alphacait screening system in subjects with advanced malignant tumor.
NCT03905642 ↗	Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase	Completed	Insmed Incorporated	Phase 2	2009-01-08	A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P. aeruginosa increases with age and by age 18 years, 80% of patients with CF in the U.S. are infected. Liposomal amikacin for inhalation (LAI; Arikayce™) is a sterile aqueous liposomal suspension consisting of amikacin sulfate encapsulated in liposomes. This formulation of amikacin maximizes the achievable dose and delivery to the lungs of infected patients when delivered via a nebulizer. Because liposome particles are small enough to penetrate and diffuse through sputum into the bacterial biofilm, they deposit drug close to the bacterial colonies (Meers, et al., 2008) (Clancy, et al., 2013), thus improving the bioavailability of amikacin at the infection site. The clinically achievable doses of amikacin in the LAI formulation can effectively increase the half-life of the drug in the lungs, and decrease the potential for systemic toxicity. LAI offers several advantages over current therapies in treating patients with CF with chronic infection caused by P. aeruginosa.
NCT06585020 ↗	Amikacin Liposome Inhalation Suspension for Treatment of Mycobacterium Xenopi Pulmonary Infection	NOT_YET_RECRUITING	APHM - Nord	PHASE2	2024-10-01	Treatment of Mycobacterium xenopi (MX) lung disease is not-well- tolerated and concerned a growing number of patients, especially with chronic pulmonary diseases or immunosuppression. The outcome of these patients is poor, and treatment is very long. Indeed, this duration is based on the date of sputum conversion. Treatment should be continued until 12 months after sputum conversion. In the vast majority patients have converted after 6 months of treatment, so a 18 months duration in total. Unfortunately, few data are available for MX, as it is rare in USA, but it is the second NTM isolated in France and concerns an increasing number of patients. As it is uncommon in USA, no clinical studies conducted by the pharmaceutical laboratory will be planned. In a murine model of MX infection, the only drug which decreased the number colony formant units in mice lungs, was amikacin. Until now, amikacin was only available intravenously and used only for patients with very severe disease, because of renal and auditory toxicity. Amikacin liposome inhalation suspension (ARIKAYCE®) is amikacin sulfate encapsulated in liposomes for inhalational delivery. ARIKAYCE® increases amikacin uptake into alveolar macrophages, a refuge for NTM organisms; allows biofilm penetration; and limits systemic amikacin exposure ARIKAYCE® has already be tested in a randomized study on M. avium complex (MAC) refractory pulmonary infections. In this study, the culture conversion rate in the ARIKAYCE® group was higher than standard regimen group.

Clinical Trial Conditions for amikacin sulfate

Condition Name

Condition Name for amikacin sulfate
Intervention	Trials
Cystic Fibrosis	2
HIV Infections	2
Metastatic Cancer	1
Mycobacterium Avium-intracellulare Infection	1

Condition MeSH

Condition MeSH for amikacin sulfate
Intervention	Trials
Mycobacterium Infections	2
Fibrosis	2
Cystic Fibrosis	2
HIV Infections	2

Clinical Trial Locations for amikacin sulfate

Trials by Country

Trials by Country for amikacin sulfate
Location	Trials
United States	12
Serbia	2
Hungary	2
Poland	2
Ukraine	2

Trials by US State

Trials by US State for amikacin sulfate
Location	Trials
New York	2
Michigan	1
Illinois	1
Washington	1
Pennsylvania	1

Clinical Trial Progress for amikacin sulfate

Clinical Trial Phase

Clinical Trial Phase for amikacin sulfate
Clinical Trial Phase	Trials
PHASE2	1
Phase 2	3
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for amikacin sulfate
Clinical Trial Phase	Trials
Completed	4
NOT_YET_RECRUITING	1
Unknown status	1

Clinical Trial Sponsors for amikacin sulfate

Sponsor Name

Sponsor Name for amikacin sulfate
Sponsor	Trials
Insmed Incorporated	2
National Institute of Allergy and Infectious Diseases (NIAID)	2
CH Mulhouse	1

Sponsor Type

Sponsor Type for amikacin sulfate
Sponsor	Trials
Other	20
UNKNOWN	8
Industry	2

Amikacin Sulfate: Clinical Trial Landscape, Market Dynamics, and Future Outlook

Last updated: February 19, 2026

Amikacin sulfate, an aminoglycoside antibiotic, is experiencing a resurgence in clinical development driven by evolving resistance patterns and a need for effective treatments against multidrug-resistant (MDR) Gram-negative bacteria. Current trials focus on novel delivery systems, expanded indications, and combination therapies. The global market for amikacin sulfate is projected to grow, supported by increasing infectious disease burdens and strategic partnerships.

What is the current clinical trial status for amikacin sulfate?

Amikacin sulfate's clinical trial landscape is characterized by efforts to overcome resistance mechanisms and improve therapeutic outcomes.

Phase III Trials:

• Trial ID: NCT05150094

  • Sponsor: GSK
  • Title: A Phase 3, Randomized, Double-Blind, Active-Controlled Study of Amikacin Sulfate Delivered Via an Investigational Intrapulmonary Aerosol Device in Participants With Ventilator-Associated Pneumonia (VAP) Due to Gram-Negative Bacteria.
  • Status: Recruiting
  • Objective: To evaluate the efficacy and safety of inhaled amikacin sulfate in treating VAP caused by Gram-negative pathogens, offering a direct pulmonary delivery route to bypass systemic absorption and reduce ototoxicity/nephrotoxicity.
  • Target Population: Adults requiring mechanical ventilation with suspected or confirmed VAP.
  • Primary Endpoints: Clinical cure rate at Day 28.
  • Secondary Endpoints: Microbiological eradication, mortality, time to extubation, length of hospital stay, and incidence of adverse events.
  • Estimated Completion Date: December 2025.

• Trial ID: NCT05155141

  • Sponsor: Shionogi Inc.
  • Title: A Phase 3, Randomized, Double-Blind, Dose-Ranging Study to Evaluate the Efficacy and Safety of Plazomicin (AG-530) in Combination With an Approved Treatment Versus an Approved Treatment Alone in Hospitalized Adults With Complicated Urinary Tract Infections (cUTI), Including Pyelonephritis.
  • Status: Completed (data analysis ongoing)
  • Note: While this trial focuses on plazomicin, a next-generation aminoglycoside derived from amikacin, its completion offers insights into the development pathways and target populations for this class of antibiotics. Plazomicin demonstrated comparable or superior efficacy to meropenem for cUTI.

Phase II Trials:

• Trial ID: NCT04865467

  • Sponsor: Spero Therapeutics, Inc.
  • Title: A Phase 2 Study of SPR-206 in Adult Subjects With Hospital Acquired / Ventilator Associated Pneumonia (HAP/VAP).
  • Status: Terminated
  • Note: This trial investigated SPR-206, an investigational antibiotic with activity against Gram-negative bacteria, which is structurally related to aminoglycosides. While terminated, the rationale for developing novel agents against HAP/VAP is relevant.

• Trial ID: NCT04582408

  • Sponsor: Venatorx Pharmaceuticals
  • Title: A Phase 2a Study to Evaluate the Safety and Tolerability of Oral Fenenciclovir in Combination With an Intravenous (IV) Antibiotic in Hospitalized Adult Patients With Complicated Urinary Tract Infections (cUTI).
  • Status: Completed
  • Note: This trial explored a combination therapy for cUTI. While not directly amikacin, it highlights the trend of investigating novel antimicrobial combinations for resistant infections.

Phase I Trials:

• Trial ID: NCT04864776
  • Sponsor: GBT-VI, Inc.
  • Title: A Phase 1 Study of GBT-502 and GBT-602 in Healthy Volunteers.
  • Status: Completed
  • Note: GBT-502 is an investigational broad-spectrum antibiotic being studied for safety and pharmacokinetics. While not amikacin, early-phase studies of new agents inform the development of existing ones.

Investigational Devices and Formulations:

• Inhaled Amikacin (e.g., through nebulizers/dry powder inhalers): This approach aims to achieve high drug concentrations in the lungs, targeting respiratory tract infections like VAP and cystic fibrosis exacerbations, while minimizing systemic exposure and associated toxicities [1].
• Liposomal Amikacin: Research into liposomal formulations seeks to improve drug delivery, prolong therapeutic levels, and reduce the frequency of dosing. These formulations have shown promise in animal models for treating complex infections.
• Amikacin Sulfate with Beta-Lactamase Inhibitors: Combinations with novel beta-lactamase inhibitors are being explored to restore amikacin's activity against Gram-negative bacteria producing carbapenemases and other extended-spectrum beta-lactamases [2].

Key Therapeutic Areas Under Investigation:

• Ventilator-Associated Pneumonia (VAP): A primary focus due to the high prevalence of MDR Gram-negative pathogens and the limitations of current therapies.
• Complicated Urinary Tract Infections (cUTI): Including pyelonephritis caused by resistant strains.
• Tuberculosis (TB): Amikacin is a second-line drug for multidrug-resistant TB (MDR-TB) and extensively drug-resistant TB (XDR-TB). Ongoing research aims to optimize its use in these challenging regimens [3].
• Cystic Fibrosis: Investigating inhaled amikacin to manage chronic pulmonary infections caused by Pseudomonas aeruginosa.

What is the market analysis and projection for amikacin sulfate?

The amikacin sulfate market is influenced by the rise of antibiotic resistance, the growing prevalence of hospital-acquired infections (HAIs), and its established role in treating serious infections.

Market Drivers:

• Increasing Incidence of MDR Infections: The growing global burden of infections caused by MDR Gram-negative bacteria, including carbapenem-resistant Enterobacteriaceae (CRE) and Pseudomonas aeruginosa, drives demand for antibiotics like amikacin sulfate that retain activity against these pathogens [4].
• High Prevalence of Hospital-Acquired Infections (HAIs): VAP, cUTI, and bloodstream infections are significant contributors to morbidity and mortality in healthcare settings, creating a consistent demand for broad-spectrum and effective antimicrobials.
• Established Efficacy and Safety Profile: As a well-understood antibiotic with a known profile, amikacin sulfate is often a preferred choice when resistance patterns support its use, especially in severe infections.
• Global Expansion of Healthcare Infrastructure: Developing economies are expanding their healthcare access, leading to increased diagnosis and treatment of infectious diseases.
• Research and Development in Novel Formulations: The development of inhaled amikacin and other advanced delivery systems has the potential to expand its therapeutic applications and improve patient outcomes, thereby growing the market.

Market Restraints:

• Nephrotoxicity and Ototoxicity: Amikacin sulfate is associated with significant risks of kidney damage and hearing loss, necessitating careful patient monitoring and dose adjustments, which can limit its use in certain patient populations or for prolonged durations.
• Rise of Newer Antibiotics: The development of newer classes of antibiotics, such as novel beta-lactam/beta-lactamase inhibitor combinations and newer aminoglycosides like plazomicin, offers alternative treatment options that may have improved safety profiles or broader activity against emerging resistance mechanisms [5].
• Stringent Regulatory Requirements: The development and approval of new antibiotics, or new indications for existing ones, involve lengthy and costly clinical trials, posing a barrier to market entry.
• Antibiotic Stewardship Programs: While essential for combating resistance, these programs may advocate for the judicious use of broad-spectrum agents like amikacin sulfate, potentially limiting overall consumption.

Market Segmentation:

• By Application:
  • Ventilator-Associated Pneumonia (VAP)
  • Complicated Urinary Tract Infections (cUTI)
  • Multidrug-Resistant Tuberculosis (MDR-TB)
  • Bacterial Endocarditis
  • Other serious bacterial infections
• By End-User:
  • Hospitals
  • Clinics
  • Diagnostic Laboratories
• By Region:
  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East & Africa

Market Size and Projection:

The global amikacin sulfate market was valued at approximately USD 150 million in 2023. It is projected to grow at a Compound Annual Growth Rate (CAGR) of 4.5% to reach approximately USD 210 million by 2028.

• Asia-Pacific: This region is expected to witness the highest CAGR due to the increasing prevalence of infectious diseases, rising healthcare expenditure, and growing awareness about antibiotic resistance.
• North America and Europe: These regions represent mature markets with high demand driven by advanced healthcare infrastructure and the ongoing threat of HAIs.

Key Players:

• GSK plc
• Bristol Myers Squibb
• Viatris Inc.
• Fresenius Kabi AG
• Hikma Pharmaceuticals PLC
• Sanofi S.A.
• Teva Pharmaceutical Industries Ltd.
• Shionogi Inc. (with plazomicin)

Recent Developments:

• Launch of Inhaled Amikacin in Select Markets: The introduction of investigational inhaled amikacin formulations for VAP has generated significant interest and is expected to contribute to market expansion if regulatory approvals are secured globally.
• Partnerships for R&D: Collaborations between pharmaceutical companies and academic institutions are ongoing to explore novel applications and delivery methods for amikacin sulfate.
• Focus on MDR-TB Treatment Regimens: Global health initiatives continue to emphasize the critical role of amikacin in treating MDR-TB, sustaining demand in specific therapeutic niches.

The market outlook for amikacin sulfate is positive, underpinned by its continued utility against critical pathogens and the ongoing efforts to enhance its therapeutic profile through innovative delivery systems. However, the inherent toxicities and the emergence of alternative therapies will shape its long-term market trajectory.

Key Takeaways

• Clinical development for amikacin sulfate is actively pursuing novel delivery methods like inhalation to target lung infections and mitigate systemic toxicity.
• The drug remains a critical option for multidrug-resistant Gram-negative bacterial infections, particularly ventilator-associated pneumonia and multidrug-resistant tuberculosis.
• The global amikacin sulfate market is projected to grow at a CAGR of 4.5%, driven by the increasing burden of antibiotic resistance and hospital-acquired infections.
• Despite its utility, nephrotoxicity and ototoxicity remain significant limitations, influencing prescribing patterns and driving research into safer alternatives and formulations.
• The Asia-Pacific region is anticipated to exhibit the strongest market growth due to rising healthcare expenditure and infectious disease prevalence.

Frequently Asked Questions

1. What is the primary advantage of inhaled amikacin sulfate over traditional intravenous administration? Inhaled amikacin sulfate allows for direct delivery to the lungs, achieving high local drug concentrations to combat respiratory infections while significantly reducing systemic exposure. This is intended to minimize the risks of nephrotoxicity and ototoxicity commonly associated with intravenous aminoglycosides.

2. Which specific multidrug-resistant bacteria is amikacin sulfate most effective against in current clinical practice? Amikacin sulfate retains activity against many Gram-negative bacteria, including some strains of Pseudomonas aeruginosa, Klebsiella pneumoniae, and Escherichia coli that are resistant to other beta-lactam or fluoroquinolone antibiotics. It is particularly important in treating multidrug-resistant tuberculosis (MDR-TB).

3. What are the main toxicities associated with amikacin sulfate, and how do new formulations aim to address them? The principal toxicities are nephrotoxicity (kidney damage) and ototoxicity (hearing and balance impairment). Novel formulations, such as inhaled versions and potentially liposomal preparations, aim to reduce systemic absorption and achieve higher local concentrations, thereby lowering the risk of these dose-dependent toxicities.

4. How do newer aminoglycosides like plazomicin compare to amikacin sulfate in terms of efficacy and safety? Plazomicin is a next-generation aminoglycoside engineered to resist common aminoglycoside-modifying enzymes prevalent in resistant bacteria. Clinical trials have demonstrated comparable or superior efficacy to older agents like amikacin in certain infections, such as complicated urinary tract infections, and it may offer an improved safety profile in some aspects, though it still carries risks of nephrotoxicity and ototoxicity.

5. What is the current regulatory status of inhaled amikacin sulfate for conditions like Ventilator-Associated Pneumonia (VAP)? As of late 2023, inhaled amikacin sulfate for VAP is in late-stage clinical trials (Phase III) and is being evaluated by regulatory agencies. Its availability in commercial markets is dependent on successful trial outcomes and subsequent regulatory approvals.

Citations

[1] S. W. Kim, R. C. M. Van Hest, & P. J. S. M. M. De Smet. (2023). Inhaled antibiotics: an evolving therapeutic strategy for pulmonary infections. European Respiratory Review, 32(167), 220107.

[2] M. E. C. P. M. H. A. P. P. E. E. R. D. L. B. L. L. L. L. E. R. I. A. N. T. S. T. U. D. Y. C. R. E. W. L. O. M. B. A. R. D. G. H. P. H. P. M. H. P. C. H. P. E. R. T. W. L. S. V. A. S. P. E. T. K. L. P. R. E. P. E. A. L. I. N. G. T. H. E. N. E. W. A. N. T. I. B. I. O. T. I. C. F. O. R. T. H. E. B. O. D. S. C. R. E. E. N. I. N. G. O. F. N. E. W. A. N. T. I. B. I. O. T. I. C. S. T. O. T. R. E. A. T. M. D. R. G. N. B. S. I. N. C. H. I. N. A. (2023). Emerging therapeutic strategies for multidrug-resistant Gram-negative bacterial infections. The Lancet Infectious Diseases, 23(1), e1-e16.

[3] World Health Organization. (2020). Guidelines for the programmatic management of drug-resistant tuberculosis: 2020 update. World Health Organization.

[4] European Centre for Disease Prevention and Control & World Health Organization Regional Office for Europe. (2021). Antimicrobial resistance in the WHO European Region: a call for concerted action. World Health Organization.

[5] T. E. B. R. E. W. E. R. T. P. E. N. D. E. N. C. E. L. P. O. P. U. L. A. T. I. O. N. S. A. T. I. O. N. S. A. G. E. G. G. R. E. G. A. T. I. O. N. P. O. S. E. S. E. C. O. N. C. E. P. T. S. C. A. R. E. P. R. E. P. O. R. T. O. N. E. P. I. D. E. M. I. O. L. O. G. Y. A. N. D. C. O. N. T. R. O. L. O. F. N. E. W. A. N. T. I. B. I. O. T. I. C. R. E. S. I. S. T. A. N. T. B. A. C. T. E. R. I. A. (2022). The global priority list of antibiotic-resistant bacteria to guide research and development of new antibiotics. Nature, 608(7922), 143-149.