Liposomal Amikacin for Inhalation (LAI) in the Treatment of Mycobacterium Abscessus Lung Disease

• Status: Completed
• Trial end date: 2019-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Kevin Winthrop
• Collaborators: Insmed Incorporated; The University of Texas Health Science Center at Tyler

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