A Prospective, Multicenter, Single-arm MAC-PD Cohort: a NTM-NET and ESGMYC Collaborative Study

Study Overview
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Brief Summary
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To gain insight in the effectiveness of, adherence and adjustments to guideline-based three-drug antimycobacterial therapy in patients with Mycobacterium avium complex pulmonary disease (MAC-PD) during the first 6 months of treatment.

Detailed Description
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Mycobacterium avium complex (MAC) is the most frequent causative species that results in nontuberculous mycobacterial pulmonary disease (NTM-PD). Current treatment outcomes for MAC-PD are unsatisfactory with sputum culture conversion rates between 50-70%.The high prevalence of drug toxicity and low adherence to guideline-based treatment (GBT) are key reasons for the limited treatment success. Hence, there is an unmet need for prospective data on effectiveness of, adherence and adjustments to MAC-PD treatment regimens. By collaborating with NTM-NET and ESGMYC affiliated medical centers, we enable systematic, prospective data collection of an international MAC-PD cohort.

Official Title
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A Prospective, Multicenter, Single-arm MAC-PD Cohort: a NTM-NET and ESGMYC Collaborative Study

Conditions
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Mycobacterium Avium Complex Pulmonary Disease

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Newly diagnosed MAC-PD (primary or recurrent) as per the international NTM guideline that requires three-drug antimycobacterial treatment Signed and dated informed consent Exclusion Criteria: The participant is in poor general condition where participation in the study cannot be accepted per discretion of the Investigator The participant has a known or suspected, current drug or alcohol abuse, that is, in the opinion of the Investigator, sufficient to compromise the safety or cooperation of the patient HIV-infection; Cystic fibrosis; >1 month antibiotic treatment for current MAC infection; < 6 months between previous antimycobacterial NTM-PD treatment and antimycobacterial treatment for current MAC-PD Disseminated MAC infection; Active pulmonary tuberculosis, fungal or nocardial disease requiring treatment; Active pulmonary malignancy (primary or metastatic) or any other malignancy requiring chemotherapy or radiotherapy within 6 months before screening or anticipated during the study period;

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Culture conversion rate | Conversion from positive cultures at baseline to negative cultures after 6 months of antimycobacterial treatment, defined by three or more negative sputum cultures sampled a month apart or one negative culture from a bronchial lavage. | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in mycobacterial load compared to baseline | Change in acid fast bacilli (AFB) smear determined by auramine staining | 6 months | 
| Change in mycobacterial load compared to baseline | Change in time-to-positivity of positive cultures | 6 months | 
| Change in symptoms compared to baseline | Change in symptoms as judged by the treating physician | 6 months | 
| Adverse drug reactions to antimycobacterial treatment | Adverse drug reactions and the (assumed) causative antimycobacterial drug | 6 months | 
| Antimycobacterial treatment adjustments | Any change in the antimycobacterial treatment including the reason | 6 months | 
| Other treatment adjustments | Changes in concomitant medication, supportive care measures or a surgical intervention | 6 months |