Clinical Trial: Liposomal Amikacin for Inhalation (LAI) in the Treatment of Mycobacterium Abscessus Lung Disease

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 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.


Sponsor: Kevin Winthrop

Current Primary Outcome: Change from Baseline sputum culture at 12 months [ Time Frame: Sputum examined for culture change from Baseline at 12 months ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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 ]


Original Secondary Outcome: Same as current

Information By: Oregon Health and Science University

Dates:
Date Received: January 3, 2017
Date Started: October 2016
Date Completion: December 2019
Last Updated: January 26, 2017
Last Verified: January 2017