Table of contents

• Trial overview
• Studies in lung infections
• Studies in ICU infection and sepsis
• Studies in children and adolescents
• Trial phases and study size
• Outcomes measured in the trials

Trial overview

The trial data show Amikacin being studied in different infection settings, not as a general medicine article but as part of clinical research programs.^[1] The studies include people with mycobacterial lung infections, ventilator-related airway infection, sepsis, and serious bacterial infections in children.^[1][2]

Most of the trials are interventional studies, which means researchers assign a treatment or compare treatment groups.^{[1][2] The trial goals are mainly to test effectiveness, safety, and treatment outcomes in specific patient groups.[1][2]}

Studies in lung infections

Several trials focus on mycobacterial lung infection, which means lung disease caused by certain slow-growing bacteria.^{[1][6] One large Phase 3 study looked at adults newly diagnosed with nontuberculous mycobacterial lung infection caused by Mycobacterium avium complex (MAC) and compared Amikacin Liposome Inhalation Suspension plus background treatment with an empty liposome control plus the same background treatment.[1]}

That study measured the change in respiratory symptom score from the start of the trial to Month 13, so it was focused on how patients felt over time.^[1] The study was completed and included 783 participants.^[1]

Another Phase 2 trial studied Mycobacterium abscessus pulmonary disease and included several treatment options, including Amikacin by inhalation and by intravenous use.^[6] Its main outcome was microbiological clearance, meaning the germ could no longer be found in sputum or bronchoalveolar lavage samples, together with good tolerance of the treatment.^[6]

A Phase 3 trial in Mycobacterium xenopi pulmonary infection studied ARIKAYCE liposomal added to standard treatment and compared it with standard treatment alone.^[3] The main result was the 3-month sputum conversion rate, which checks whether the lung sample becomes negative for the germ.^[3]

Studies in ICU infection and sepsis

Amikacin is also being studied in critically ill patients with ventilator-associated tracheobronchitis, a breathing-tube related airway infection seen in intensive care.^[4] This Phase 3 trial compared a 5-day course of inhaled Amikacin with placebo and looked at whether treatment reduced progression from ventilator-associated tracheobronchitis to ventilator-associated pneumonia by Day 28.^[4]

Another Phase 3 study included Amikacin among many antibiotic options in patients with sepsis in the ICU.^[5] The main endpoint was 30-day mortality, which means whether patients were alive 30 days after treatment assignment.^[5]

These trials show that Amikacin is being tested in severe hospital infections where fast treatment choices matter.^[4][5]

Studies in children and adolescents

One Phase 2 trial studied children and adolescents 9 months to younger than 18 years with serious gram-negative bacterial infections, including bloodstream infection, complicated urinary tract infection, complicated intra-abdominal infection, hospital-acquired pneumonia, and ventilator-associated pneumonia.^[2] In this study, Amikacin was one of the treatment options compared with best available therapy.^[2]

The study looked at pharmacokinetics, which means how the body handles the medicine over time, and also safety and tolerability.^[2] The planned enrollment was 54 participants.^[2]

A separate Phase 3 study in pediatric cancer patients with febrile neutropenia tested whether antibiotics could be stopped early in children who were improving and had low infection markers.^[8] Amikacin was one of the antibiotics listed in the treatment options for this study.^[8] The main outcome was uncomplicated resolution at 28 days, meaning no death, no ICU admission, no sepsis, and no proven bacterial infection.^[8]

Trial phases and study size

The listed Amikacin trials are mainly Phase 2 and Phase 3 studies, showing that the drug is being tested in both mid-stage and larger confirmatory research.^{[1][2][3][4][5][6][8]}

One Phase IV study is also listed, but it does not specifically include Amikacin in its intervention list.^[7] The enrollment sizes vary widely, from 54 participants in a pediatric study to 783 participants in the MAC lung infection study.^[1][2]

These sizes matter because larger studies can better show whether a treatment works across many patients, while smaller studies are often used to learn more about safety or dosing behavior.^[2][6]

Outcomes measured in the trials

The main outcomes vary by disease but usually focus on either improvement in infection or safety.^{[1][2][3][4][5][6][8]}

• Respiratory symptom score in the MAC lung infection study measured how symptoms changed over 13 months.^[1]

• Sputum conversion in the Mycobacterium xenopi study measured whether the lung samples became negative after 3 months.^[3]

• Progression to pneumonia in the ICU airway infection study measured whether ventilator-associated tracheobronchitis became ventilator-associated pneumonia by Day 28.^[4]

• Mortality in the sepsis study measured whether patients survived 30 days.^[5]

• Microbiological clearance and tolerance in the Mycobacterium abscessus study measured whether the germ was cleared and whether the treatment was tolerated.^[6]

• Uncomplicated resolution in the pediatric febrile neutropenia study measured recovery without major complications at 28 days.^[8]

Together, these endpoints show that the research on Amikacin is aimed at practical patient outcomes: symptom relief, infection control, survival, and safety.^{[1][2][3][4][5][6][8]}