A Study to Validate Patient-Reported Outcome Instruments in Adult Subjects with Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection

Overview

About this study

The purpose of this study is to generate evidence demonstrating the domain specification (via modern psychometric methods), reliability, validity, and responsiveness (within-subject meaningful change) of the patient-reported outcome (PRO) endpoints.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Male or female, ≥ 18 years of age (19 years or older in South Korea)

- Current diagnosis of Mycobacterium avium Complex (MAC) lung infection

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

- A chest computed tomography (CT) scan, read locally, within 6 months prior to
Screening to determine presence and size of pulmonary cavities. Participants who do
not have a chest CT scan within 6 months prior to Screening will be required to obtain
a chest CT scan, read locally, during Screening

- Willingness and ability to adhere to prescribed study treatment during the study

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

- Women of child-bearing potential (WOCBP) (ie, fertile following menarche and until
becoming post-menopausal unless permanently sterile) and fertile men (ie, all men
after puberty unless permanently sterile by bilateral orchidectomy) agree to practice
a highly effective method of birth control from Day 1 to at least 90 days after the
last dose. Examples of such birth controls are:

- 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]),

- 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 drugs or performing
any study related procedure

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

- Men with partners who are WOCBP (pregnant or non-pregnant) agree to use condoms and
non-pregnant partners should practice a highly effective method of birth control

Exclusion Criteria:

- Diagnosis of cystic fibrosis (CF)

- History of more than 3 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 or contraindications to use to ALIS, aminoglycosides, or any of
their excipients

- 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 confirmed by more than one follicle
stimulating hormone [FSH] measurement), or naturally or surgically sterile through
bilateral oophorectomy, hysterectomy, or bilateral salpingectomy. For women under the
age of 45 years, confirmatory testing with FSH should be considered

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

- Known or suspected acquired immunodeficiency syndromes (HIV-positive, regardless of
CD4 counts). Other immunodeficiency syndromes that may interfere with study
participation in the opinion of the Investigator.

- Current alcohol, medication, or illicit drug abuse

- Known and active COVID-19 infection

- MAC isolate with MIC for clarithromycin ≥ 32 µg/mL at Screening

- Known hypersensitivity or contraindications to use to ethambutol, azithromycin
(including other macrolides or ketolides), or any of their excipients per local
labeling guidance.

Eligibility last updated 6/13/22. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Margaret Johnson, M.D.

Open for enrollment

Contact information:

Margaret Johnson M.D.

(904) 953-2869

Johnson.Margaret2@mayo.edu

Rochester, Minn.

Mayo Clinic principal investigator

Timothy Aksamit, M.D.

Open for enrollment

Contact information:

Pulmonary Clinical Research Unit

(800) 753-1606

PCRUE18@mayo.edu

More information

Publications

Publications are currently not available
.
CLS-20514656

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