Arikayce for Nontuberculous Mycobacteria

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Insmed Incorporated
ClinicalTrials.gov Identifier:
NCT01315236
First received: March 11, 2011
Last updated: February 12, 2015
Last verified: February 2015

March 11, 2011
February 12, 2015
May 2012
April 2014   (final data collection date for primary outcome measure)
Change in semi-quantitative mycobacterial culture results from baseline to end of treatment [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01315236 on ClinicalTrials.gov Archive Site
  • Proportion of subjects with culture conversion to negative [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Time to "rescue" anti-mycobacterial drugs [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Change from baseline in 6-minute walk distance and oxygen saturation [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Change from baseline in Patient Reported Outcomes [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Evaluation of safety and tolerability [ Time Frame: For entire study duration ] [ Designated as safety issue: Yes ]
  • Proportion of subjects with culture conversion to negative [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Time to "rescue" anti-mycobacterial drugs [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Change from baseline in computed tomography scan abnormalities [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Change from baseline in 6-minute walk distance and oxygen saturation [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Change from baseline in Patient Reported Outcomes [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]
  • Evaluation of safety and tolerability [ Time Frame: For entire study duration ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Arikayce for Nontuberculous Mycobacteria
A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin for Inhalation (Arikayce®) in Patients With Recalcitrant Nontuberculous Mycobacterial Lung Disease

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.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Mycobacterium Infections, Nontuberculous
  • 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.
  • Experimental: Arikayce
    Intervention: Drug: Liposomal amikacin for inhalation (LAI)
  • Placebo Comparator: Placebo
    Intervention: Drug: placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
89
June 2015
April 2014   (final data collection date for primary outcome measure)

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.
Both
18 Years to 85 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada
 
NCT01315236
TR02-112
Yes
Insmed Incorporated
Insmed Incorporated
National Institute of Allergy and Infectious Diseases (NIAID)
Study Director: Gina Eagle Insmed Incorporated
Insmed Incorporated
February 2015

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