Validation of Patient Reported Outcome Measures in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex

• Status: Recruiting
• Trial end date: 2022-02-28
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to generate evidence demonstrating the domain specification via modern psychometric methods, reliability, validity, and responsiveness of Quality of Life - Bronchiectasis (QoL-B) and Patient-Reported Outcome Measurement Information System - Fatigue-Short Form (PROMIS F-SF 7a) in participants with newly diagnosed nontuberculous mycobacterial (NTM) lung infection caused by Myobaxterium avium Complex (MAC).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Insmed Incorporated
• Treatments: Amikacin; Azithromycin; Ethambutol

Criteria

Inclusion Criteria:

• Male or female, ≥ 18 years of age (20 years or older in Japan)

• Current diagnosis of Myobacterium avium Complex (MAC) lung infection (initial, second, or third infection event)

• Positive sputum culture for MAC within 6 months prior to screening

• A high-resolution chest computerized tomography (CT) scan or chest CT scan with contrast, read locally, within 6 months prior to Screening and provided to the Sponsor. Participants who do not have a chest CT scan within 6 months prior to Screening will be required to obtain a high resolution chest CT scan or chest CT scan with contrast, read locally, during Screening

• Adherence to a predefined multidrug antimycobacterial regimen during the study

• Ability to produce (spontaneously or with induction) approximately 2mL of sputum for mycobacteriology at screening

• Women of child-bearing potential (WOCBP) agree to practice an acceptable method of birth control (eg, true abstinence [refraining from heterosexual intercourse during the entire study], copper intrauterine device (IUD), hormonal methods (levonorgestrel-releasing intrauterine system, progestogen implant, combined oral contraceptive pill [combined with barrier method] or double barrier method plus a spermicidal agent, exclusive homosexual relationship, or sole male partner who has undergone surgical sterilization with confirmation of azoospermia at least 3 months post procedure) while participating in the study

• Provide signed informed consent prior to administration of study drug or performing any study related procedure

• Be able to comply with study drug use, study visits, and study procedures as defined by the protocol

Exclusion Criteria:

• Diagnosis of cystic fibrosis (CF)

• History of 3 or more prior MAC lung infections

• Received any mycobacterial antibiotic treatment for current MAC lung infection

• Refractory MAC lung infection, defined as having positive MAC cultures while being treated with a multidrug mycobacterial antibiotic treatment regimen for a minimum of 6 consecutive months and no documented successful treatment, defined as negative sputum culture for MAC and cessation of treatment

• Relapse of prior MAC lung infection, defined as positive sputum culture for MAC ≤ 6 months of cessation of prior successful treatment

• Evidence of any pulmonary cavity ≥ 2 cm in diameter, as determined by chest CT scan, read locally, within 6 months prior to Screening

• Radiographic finding of new lobar consolidation, atelectasis, significant pleural effusion, or pneumothorax during routine clinical care within 2 months prior to Screening

• Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within 1 year prior to Screening or anticipated during the study

• Acute pulmonary exacerbation (eg, chronic obstructive pulmonary disease [COPD] or bronchiectasis) requiring treatment with antibiotics, or corticosteroids (intravenous [IV] or oral), within 4 weeks prior to and during Screening

• Current smoker

• History of lung transplantation

• Prior exposure to amikacin liposome inhalation suspension (ALIS) (including clinical study)

• Known hypersensitivity to aminoglycosides

• Disseminated MAC infection

• Positive pregnancy test or lactation at Screening. All WOCBP will be tested. Women not of childbearing potential are defined as postmenopausal (ie, amenorrheic for 12 months without an alternative medical cause), or naturally or surgically sterile through bilateral oophorectomy, hysterectomy, or bilateral salpingectomy. For women under the age of 45, confirmatory testing with follicle stimulating hormone should be considered.)

• Administration of any investigational drug within 8 weeks prior to Screening

• Acquired and primary immunodeficiency syndromes (eg, HIV-positive, regardless of CD4 counts)

• Current alcohol, medication, or illicit drug abuse