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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 : Active, not recruiting
First Posted : January 31, 2017
Last Update Posted : August 27, 2019
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

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.

Condition or disease Intervention/treatment Phase
Mycobacterium Infections, Nontuberculous Mycobacteria, Atypical Drug: LAI plus multi-drug regimen Phase 2

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
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
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
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
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™




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

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. Number of Hospitalizations for pulmonary exacerbations [ Time Frame: Number of Hospitalizations for pulmonary exacerbations that occur between Baseline and 12 months ]
  5. Number of Adverse Events [ Time Frame: Number of Patient-reported and Investigator-reported Adverse Events at 12 months ]
  6. 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 ]

Other Outcome Measures:
  1. 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) ]
  2. Change from Baseline Chest CT at End of Treatment (EOT) [ Time Frame: CT scan examined for change from Baseline at EOT (12 months) ]
  3. 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) ]
  4. 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) ]


Information from the National Library of Medicine

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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
Criteria

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

Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Kevin Winthrop
Insmed Incorporated
The University of Texas Health Science Center at Tyler
Investigators
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Principal Investigator: Kevin L Winthrop, MD, MPH Oregon Health and Science University

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Responsible Party: Kevin Winthrop, Associate Professor, Oregon Health and Science University
ClinicalTrials.gov Identifier: NCT03038178     History of Changes
Other Study ID Numbers: LAI/INS-IIR-01
First Posted: January 31, 2017    Key Record Dates
Last Update Posted: August 27, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Kevin Winthrop, Oregon Health and Science University:
Mycobacterium Infections, Nontuberculous
Mycobacteria, Atypical
Amikacin
Inhalation
Additional relevant MeSH terms:
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Mycobacterium Infections
Mycobacterium Infections, Nontuberculous
Lung Diseases
Respiratory Aspiration
Respiratory Tract Diseases
Respiration Disorders
Pathologic Processes
Actinomycetales Infections
Gram-Positive Bacterial Infections
Bacterial Infections
Amikacin
Anti-Bacterial Agents
Anti-Infective Agents