Liposomal Amikacin for Inhalation (LAI) in the Treatment of Mycobacterium Abscessus Lung Disease
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ClinicalTrials.gov Identifier: NCT03038178 |
Recruitment Status :
Completed
First Posted : January 31, 2017
Last Update Posted : March 4, 2020
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Condition or disease | Intervention/treatment | Phase |
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Mycobacterium Infections, Nontuberculous Mycobacteria, Atypical | Drug: LAI plus multi-drug regimen | Phase 2 |
This is an open-label study of efficacy, safety and tolerability of once daily dosing of Liposomal-Amikacin for Inhalation (LAI), in addition to a standard multi-drug antibiotic therapy in accordance with the 2007 ATS/ IDSA guidelines, in patients with Mycobacterium abscessus lung disease. The multi-drug therapy is determined at the discretion of the Investigator. After screening, all eligible patients will enter the trial and will receive LAI 590 mg once daily for 12 months. If deemed necessary by the investigator, dose may be adjusted from a minimum of three times per week up to daily, in cases of adverse events relating to tolerability.
All patients who enter the study will have subsequent study visits at Months 1,2,4,6,9, at End-of-Study or Month 12, and at 1 and 3 months post study drug discontinuation. At each visit (including screening), review of concomitant medications, review of adverse events, and physical exam, will be performed. Chest CT scan will be performed at baseline, 6 months, and 12 months, unless a chest CT scan has already been performed within 6 months on this time point. The 6 minute walk test and QOL-B-NTM will be performed at baseline, 6 months, 12 months, and 3 months post study drug discontinuation. All patients will have inducted sputum collected at each study visit, and patients will self-collect expectorated sputum during intervening monthly time-points until study completion to determine changes in mycobacterial smear and culture status.
Unscheduled visits will occur as needed should subjects' symptoms worsen between visits.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 30 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-label Study of Efficacy, Safety and Tolerability of Liposomal Amikacin for Inhalation (LAI) Once Daily in Addition to Standard Multi-antibiotic Therapy in the Treatment of Mycobacterium Abscessus Lung Disease |
Actual Study Start Date : | October 2016 |
Actual Primary Completion Date : | October 31, 2019 |
Actual Study Completion Date : | December 31, 2019 |

Arm | Intervention/treatment |
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Experimental: LAI plus multi-drug regimen
once daily dosing of Liposomal-Amikacin for Inhalation (LAI) 590 mg for 12 months plus standard of care (SOC) mycobacterial multi-drug regimen in accordance with the 2007 ATS/ IDSA guidelines
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Drug: LAI plus multi-drug regimen
Liposomal Amikacin for Inhalation (LAI) is the experimental treatment which, in this single arm will be taken in conjunction with standard of care multi-drug treatment regimen
Other Name: ARIKAYCE™ |
- Change from Baseline sputum culture at 12 months [ Time Frame: Sputum examined for culture change from Baseline at 12 months ]
- Change from Baseline 6-minute Walk Test at 6 months [ Time Frame: 6-minute Walk Test results examined for change from Baseline at 6 months ]
- Change from Baseline 6-minute Walk Test at 12 months [ Time Frame: 6-minute Walk Test results examined for change from Baseline at 12 months ]
- Change from End of Treatment (EOT) sputum culture at 3 months post EOT [ Time Frame: Sputum examined for culture change from EOT at 3 months post EOT ]
- Number of Hospitalizations for pulmonary exacerbations [ Time Frame: Number of Hospitalizations for pulmonary exacerbations that occur between Baseline and 12 months ]
- Number of Adverse Events [ Time Frame: Number of Patient-reported and Investigator-reported Adverse Events at 12 months ]
- Number of subjects discontinuing study drug due to Adverse Event [ Time Frame: Number of subjects who discontinue study drug before 12 months due to Adverse Event will be examined at 12 months ]
- Change from Baseline reported nontuberculous mycobacterium (NTM) symptoms at End of Treatment (EOT) [ Time Frame: Patient-reported NTM symptoms examined for change from Baseline at EOT (12 months) ]
- Change from Baseline Chest CT at End of Treatment (EOT) [ Time Frame: CT scan examined for change from Baseline at EOT (12 months) ]
- Change from Baseline body weight at End of Treatment (EOT) [ Time Frame: body weight of patient examined for change from Baseline at EOT (12 months) ]
- Change from Baseline Body Mass Index (BMI) at End of Treatment (EOT) [ Time Frame: BMI of patient examined for change from Baseline at EOT (12 months) ]

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Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female patients 12 years and older
- Diagnosis of M. abscessus, including all subspecies, abscessus, bolleti, and massiliense lung disease according to the 2007 ATS/IDSA criteria
- Both newly diagnosed and currently on treatment or previously treated patients will be included
- Culture positive (either sputum or bronchoscopy) for M. abscessus at time of screening
- Willingness to adhere to a treatment regimen, study visits, and study procedures during the course of the study.
- Ability to produce at approximately 3.0 mL of sputum or be willing to undergo an induction that produces approximately 3.0 mL of sputum for culture collection
- Female of childbearing potential agrees to practice an acceptable method of birth control (e.g., abstinence, hormonal or barrier methods, partner sterilization, or IUD)
- Written informed consent or assent obtained from the patient, parent or legal guardian prior to the performance of any study related procedures
Exclusion Criteria:
- Active pulmonary tuberculosis requiring treatment at screening
- Treatment with inhaled or intravenous Amikacin within 14 days prior to baseline
- Known hypersensitivity to aminoglycosides
- Aspartate aminotransferase or alanine aminotransferase ≥ 3 times the upper limit of normal or total bilirubin ≥ 2 times the upper limit of normal (ULN) at screening
- Current addiction to alcohol or illicit drug abuse
- Any condition which in the opinion of the Investigator interferes with ability to adhere to study requirements
- Primary immunodeficiency syndromes and acquired immunodeficiency syndromes (e.g., HIV-positive patients regardless of CD4 counts)
- Absolute neutrophil count ≤500/μL at Screening
- Significant (as determined by the investigator) hearing loss, vestibular dysfunction, neuromuscular weakness or a diagnosis of myasthenia gravis, where the potential risk of aminoglycoside toxicity outweighs the potential benefit
- Serum creatinine >2 times ULN at Screening
- History of lung transplantation
- Any condition that, in the judgment of the Investigator, would compromise the ability of the subject to complete the study

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03038178
United States, Oregon | |
Oregon Health & Science University | |
Portland, Oregon, United States, 97239 | |
United States, Texas | |
University of Texas Health Science Center | |
Tyler, Texas, United States, 75708 |
Principal Investigator: | Kevin L Winthrop, MD, MPH | Oregon Health and Science University |
Responsible Party: | Kevin Winthrop, Associate Professor, Oregon Health and Science University |
ClinicalTrials.gov Identifier: | NCT03038178 |
Other Study ID Numbers: |
LAI/INS-IIR-01 |
First Posted: | January 31, 2017 Key Record Dates |
Last Update Posted: | March 4, 2020 |
Last Verified: | March 2020 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Mycobacterium Infections, Nontuberculous Mycobacteria, Atypical Amikacin Inhalation |
Mycobacterium Infections Mycobacterium Infections, Nontuberculous Lung Diseases Respiratory Aspiration Respiratory Tract Diseases |
Respiration Disorders Pathologic Processes Actinomycetales Infections Gram-Positive Bacterial Infections Bacterial Infections |