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

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.

Study Overview

• Status: Recruiting
• Conditions: Mycobacterium Avium Complex Pulmonary Disease

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.

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Arthur Lemson, MSc; Phone Number: +31634265743; Email: arthur.lemson@radboudumc.nl
• Study Contact Backup: Name: Wouter Hoefsloot, MSc, PhD; Phone Number: +31611072569; Email: wouter.hoefsloot@radboudumc.nl

Study Locations

• Croatia
  • Zagreb, Croatia
    • Recruiting
    • University Hospital Center Zagreb
    • Contact: Mateja Janković Makek, MD, PhD
• Japan
  • Tokyo, Japan
    • Recruiting
    • Fukujuji Hospital
    • Contact: Masashi Ito, MD, PhD
• Korea, Republic of
  • Seoul, Korea, Republic of
    • Recruiting
    • Seoul National University Hospital
    • Contact: Nakwon Kwak; Email: n.kwak@snu.ac.kr
• Netherlands
  • Nijmegen, Netherlands, 6511LK
    • Recruiting
    • Radboud University Medical Center
    • Contact: Arthur Lemson, MD; Phone Number: +31634265743; Email: arthur.lemson@radboudumc.nl
    • Contact: Wouter Hoefsloot, MD, PhD; Phone Number: +31611072569; Email: wouter.hoefsloot@radboudumc.nl
• United States
  • Oregon
    • Portland, Oregon, United States, 97239
      • Recruiting
      • Oregon Science and Health University
      • Contact: Kevin Winthrop, MD, MPH

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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;

Study Plan

How is the study designed?

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Radboud University Medical Center
• Investigators: Principal Investigator: Jakko van Ingen, MSc, PhD, Radboud University Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2023
• Primary Completion (Estimated): January 1, 2025
• Study Completion (Estimated): March 1, 2025

Study Registration Dates

• First Submitted: June 7, 2023
• First Submitted That Met QC Criteria: June 7, 2023
• First Posted (Actual): June 15, 2023

Study Record Updates

• Last Update Posted (Estimated): November 17, 2023
• Last Update Submitted That Met QC Criteria: November 16, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Infections; Bacterial Infections; Bacterial Infections and Mycoses; Gram-Positive Bacterial Infections; Actinomycetales Infections; Mycobacterium Infections; Mycobacterium Infections, Nontuberculous; Mycobacterium avium-intracellulare Infection
• Other Study ID Numbers: 2022-13742

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No