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
  Purpose

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.


Condition Intervention Phase
Mycobacterium Infections, Nontuberculous
Drug: Liposomal amikacin for inhalation (LAI)
Drug: placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled Study of Liposomal Amikacin for Inhalation (Arikayce®) in Patients With Recalcitrant Nontuberculous Mycobacterial Lung Disease

Resource links provided by NLM:


Further study details as provided by Insmed Incorporated:

Primary Outcome Measures:
  • Change in semi-quantitative mycobacterial culture results from baseline to end of treatment [ Time Frame: 84 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 89
Study Start Date: May 2012
Estimated Study Completion Date: June 2015
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Arikayce 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.
Placebo Comparator: Placebo 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.

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01315236

  Show 19 Study Locations
Sponsors and Collaborators
Insmed Incorporated
Investigators
Study Director: Gina Eagle Insmed Incorporated
  More Information

No publications provided

Responsible Party: Insmed Incorporated
ClinicalTrials.gov Identifier: NCT01315236     History of Changes
Other Study ID Numbers: TR02-112
Study First Received: March 11, 2011
Last Updated: February 12, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mycobacterium Infections
Mycobacterium Infections, Nontuberculous
Actinomycetales Infections
Bacterial Infections
Gram-Positive Bacterial Infections
Amikacin
Anti-Bacterial Agents
Anti-Infective Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on August 02, 2015