Clinical trial NCT05906316: A Prospective, Multicenter, Single-arm MAC-PD Cohort: a NTM-NET and ESGMYC Collaborative Study
Official title: A Prospective, Multicenter, Single-arm MAC-PD Cohort: a NTM-NET and ESGMYC Collaborative Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Mycobacterium Avium Complex Pulmonary Disease
Lead sponsor: Radboud University Medical Center
Sponsor class: OTHER
First posted: 2023-06-15
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Mycobacterium avium complex pulmonary disease that (MAC-PD) requires guideline-based three-drug antimycobacterial therapy. MAC is the most common causative species of pulmonary NTM infections.

Brief summary: 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: 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.

Eligibility criteria: 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;

Primary outcomes:
measure: Culture conversion rate
timeFrame: 6 months
Secondary outcomes:
measure: Change in mycobacterial load compared to baseline
timeFrame: 6 months
measure: Change in mycobacterial load compared to baseline
timeFrame: 6 months
measure: Change in symptoms compared to baseline
timeFrame: 6 months
measure: Adverse drug reactions to antimycobacterial treatment
timeFrame: 6 months
measure: Antimycobacterial treatment adjustments
timeFrame: 6 months
measure: Other treatment adjustments
timeFrame: 6 months
Other outcomes:
Locations:
city: Portland
country: United States
facility: Oregon Science and Health University
state: Oregon
city: Leuven
country: Belgium
facility: UZ Leuven
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Zagreb
country: Croatia
facility: University Hospital Center Zagreb
city: Arhus
country: Denmark
facility: Aarhus University Hospital
city: Hellerup
country: Denmark
facility: Rasmus Rude Laub
city: Tokyo
country: Japan
facility: Fukujuji Hospital
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Center
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
Study officials:
affiliation: Radboud University Medical Center
name: Jakko van Ingen, MSc, PhD
role: PRINCIPAL_INVESTIGATOR
References: