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Trial record 1 of 6 for:    LIPOSOMAL AMIKACIN MYCOBACTERIUM
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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
Sponsor:
Collaborators:
Insmed Incorporated
The University of Texas Health Science Center at Tyler
Information provided by (Responsible Party):
Kevin Winthrop, Oregon Health and Science University

Tracking Information
First Submitted Date  ICMJE January 3, 2017
First Posted Date  ICMJE January 31, 2017
Last Update Posted Date March 4, 2020
Actual Study Start Date  ICMJE October 2016
Actual Primary Completion Date October 31, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 26, 2017)
Change from Baseline sputum culture at 12 months [ Time Frame: Sputum examined for culture change from Baseline at 12 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 26, 2017)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: January 26, 2017)
  • 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) ]
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Liposomal Amikacin for Inhalation (LAI) in the Treatment of Mycobacterium Abscessus Lung Disease
Official Title  ICMJE 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
Brief Summary The proposed study will assess the efficacy, safety and tolerability of 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, for treatment of mycobacterium abscessus lung disease.
Detailed Description

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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Mycobacterium Infections, Nontuberculous
  • Mycobacteria, Atypical
Intervention  ICMJE 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™
Study Arms  ICMJE 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
Intervention: Drug: LAI plus multi-drug regimen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: January 26, 2017)
30
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 31, 2019
Actual Primary Completion Date October 31, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03038178
Other Study ID Numbers  ICMJE LAI/INS-IIR-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Kevin Winthrop, Oregon Health and Science University
Study Sponsor  ICMJE Kevin Winthrop
Collaborators  ICMJE
  • Insmed Incorporated
  • The University of Texas Health Science Center at Tyler
Investigators  ICMJE
Principal Investigator: Kevin L Winthrop, MD, MPH Oregon Health and Science University
PRS Account Oregon Health and Science University
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP