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Arikayce for Nontuberculous Mycobacteria

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ClinicalTrials.gov Identifier: NCT01315236
Recruitment Status : Completed
First Posted : March 15, 2011
Last Update Posted : October 3, 2016
Sponsor:
Collaborator:
National Institute of Allergy and Infectious Diseases (NIAID)
Information provided by (Responsible Party):
Insmed Incorporated

Tracking Information
First Submitted Date  ICMJE March 11, 2011
First Posted Date  ICMJE March 15, 2011
Last Update Posted Date October 3, 2016
Study Start Date  ICMJE May 2012
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 13, 2011)
Change in semi-quantitative mycobacterial culture results from baseline to end of treatment [ Time Frame: 84 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01315236 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 5, 2011)
  • Proportion of subjects with culture conversion to negative [ Time Frame: 84 days ]
  • Time to "rescue" anti-mycobacterial drugs [ Time Frame: 84 days ]
  • Change from baseline in 6-minute walk distance and oxygen saturation [ Time Frame: 84 days ]
  • Change from baseline in Patient Reported Outcomes [ Time Frame: 84 days ]
  • Evaluation of safety and tolerability [ Time Frame: For entire study duration ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 13, 2011)
  • Proportion of subjects with culture conversion to negative [ Time Frame: 84 days ]
  • Time to "rescue" anti-mycobacterial drugs [ Time Frame: 84 days ]
  • Change from baseline in computed tomography scan abnormalities [ Time Frame: 84 days ]
  • Change from baseline in 6-minute walk distance and oxygen saturation [ Time Frame: 84 days ]
  • Change from baseline in Patient Reported Outcomes [ Time Frame: 84 days ]
  • Evaluation of safety and tolerability [ Time Frame: For entire study duration ]
Current Other Outcome Measures  ICMJE Not Provided
Original Other Outcome Measures  ICMJE Not Provided
 
Descriptive Information
Brief Title  ICMJE Arikayce for Nontuberculous Mycobacteria
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin for Inhalation (Arikayce®) in Patients With Recalcitrant Nontuberculous Mycobacterial Lung Disease
Brief Summary The purpose of this study is to evaluate the efficacy, safety and tolerability of 84 days of daily dosing of 590 mg of LAI versus placebo in patients with recalcitrant NTM lung disease.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Mycobacterium Infections, Nontuberculous
Intervention  ICMJE
  • Drug: Liposomal amikacin for inhalation (LAI)
    • Liposomal amikacin for inhalation is provided as a sterile aqueous liposomal dispersion for inhalation via nebulization.
    • 590 mg of liposomal amikacin for inhalation is administered once daily using the PARI Investigational eFlow® Nebulizer.
    • Administration time is approximately 13 minutes.
    • Liposomal amikacin for inhalation will be administered for 84 days in the double-blind, randomized portion of the study.
    • Subjects can continue with 84 additional days of dosing in the open label extension.
  • Drug: placebo
    • Placebo is provided as a sterile aqueous lipid dispersion for inhalation via nebulization.
    • Administration procedures, volume and administration time is the same as for Arikayce.
    • Placebo will be administered for 84 days only during the double-blind, randomized portion of the study.
Study Arms
  • Experimental: Arikayce
    Intervention: Drug: Liposomal amikacin for inhalation (LAI)
  • Placebo Comparator: Placebo
    Intervention: Drug: placebo
Publications * Olivier KN, Griffith DE, Eagle G, McGinnis JP 2nd, Micioni L, Liu K, Daley CL, Winthrop KL, Ruoss S, Addrizzo-Harris DJ, Flume PA, Dorgan D, Salathe M, Brown-Elliott BA, Gupta R, Wallace RJ Jr. Randomized Trial of Liposomal Amikacin for Inhalation in Nontuberculous Mycobacterial Lung Disease. Am J Respir Crit Care Med. 2017 Mar 15;195(6):814-823. doi: 10.1164/rccm.201604-0700OC.

*   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
Actual Enrollment  ICMJE
 (submitted: December 8, 2015)
90
Original Estimated Enrollment  ICMJE
 (submitted: March 13, 2011)
100
Study Completion Date Not Provided
Actual Primary Completion Date June 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  1. Diagnosis of pulmonary nontuberculous mycobacterial lung disease in accordance with the 2007 ATS/IDSA criteria with evidence of nodular bronchiectasis and/or cavitary disease by chest computed tomography (CT).
  2. History of chronic infection with either Mycobacterium avium complex or Mycobacterium abscessus or mixed infection with both species (defined as at least 2 documented positive cultures in the prior 2 years, of which at least one was obtained in the 6 months prior to screening).
  3. Positive sputum culture obtained at screening visit with either Mycobacterium avium complex or Mycobacterium abscessus or mixed infection with one dominant species.
  4. Receiving ATS/IDSA guidelines-based treatment regimen defined as: adherent to a multi-drug regimen for at least 6 months prior to screening with persistently positive mycobacterial cultures.
  5. Ability to produce at least 3 mL of sputum or be willing to undergo an induction that produces at least 3 mL of sputum for clinical evaluation.
  6. Female of childbearing potential agrees to practice an acceptable method of birth control (e.g., abstinence, hormonal or barrier methods, partner sterilization, or IUD).

Key Exclusion Criteria:

  1. Forced Expiratory Volume in 1 second (FEV1) <30% of predicted at Screening.
  2. Presence of any clinically significant cardiac disease as determined by Investigator. The QTc criteria for Exclusion is QTc> 450 msec for males or QTc> 470 msec for females.
  3. Subjects with hemoptysis of ≥60 mL in a 24 hour period within 4 weeks prior to screening.
  4. Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within one year prior to screening or anticipated during the study period.
  5. Active allergic bronchopulmonary mycosis or any other condition requiring systemic steroids at a dose ≥ equivalent of 10 mg/day of prednisone within 3 months prior to screening or anticipated during the study period.
  6. Pulmonary tuberculosis requiring treatment or treated within 2 years prior to screening.
  7. History of lung transplantation.
  8. Hypersensitivity to aminoglycosides.
  9. Any change in chronic NTM multi-drug regimen within 28 days prior to Study Day 1.
  10. Evidence of biliary cirrhosis with portal hypertension.
  11. History of daily, continuous oxygen supplementation.
  12. Smoking tobacco or any substance within 6 months prior to screening or anticipated inability to refrain from smoking throughout the study.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01315236
Other Study ID Numbers  ICMJE TR02-112
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Insmed Incorporated
Study Sponsor  ICMJE Insmed Incorporated
Collaborators  ICMJE National Institute of Allergy and Infectious Diseases (NIAID)
Investigators  ICMJE
Study Director: Gina Eagle Insmed Incorporated
PRS Account Insmed Incorporated
Verification Date September 2016

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