UMIN-CTR Clinical Trial

Basic information
Public title	An observational study on host immunity in patients with refractory Mycobacterium avium complex pulmonary disease
Acronym	An observational study on host immunity in patients with refractory MAC-PD
Scientific Title	An observational study on host immunity in patients with refractory Mycobacterium avium complex pulmonary disease: a multicenter observational study
Scientific Title:Acronym	An observational study on host immunity in patients with refractory MAC-PD
Region	Japan

Condition
Condition	Mycobacterium avium complex pulmonary disease
Classification by specialty	Pneumology Infectious disease
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	-To determine the host immune characteristics in patients with refractory MAC-PD.
Basic objectives2	Others
Basic objectives -Others	-To make recommendations that will lead to the establishment of novel treatment strategies for refractory MAC-PD.
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Not applicable

Assessment
Primary outcomes	1) Clinical outcome Treatment failure of MAC-PD under the standard treatment. 2) Immunological outcome Host immune characteristics in treatment failure of MAC-PD under standard treatment.
Key secondary outcomes

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	20 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	1. Patients with MAC-PD Patients who have been on multidrug therapy for MAC-PD for at least 12 months and meet the following criteria. -Age: 20 and older -Written consent has been obtained. The diagnostic definition of MAC-PD should meet both of the following criteria 1) and 2). 1) Clinical criteria (Both required) -Patients with respiratory or systemic symptoms. -Nodular or cavitary opacities on chest radiograph, or a computed tomography scan that shows bronchiectasis with multiple small nodules. 2) Microbiologic criteria (One of the following is met) -Positive culture results from at least two separate expectorated sputum samples. -Positive culture results from at least one bronchial wash or lavage. -Transbronchial or other lung biopsy with mycobacterial histologic features and one or more positive culture results from sputum, bronchial washing, or lung biopsy In this study, it is assumed that patients are still on multidrug therapy at the time of study registration, but patients who are discontinued after 12 months of therapy due to difficulty in controlling the disease of MAC-PD can be enrolled. 2. Healthy subjects Healthy people who meet the following criteria: -Age: 20 and older -Written consent has been obtained. -People without respiratory or systemic symptoms. -No obvious abnormality on chest radiograph, or a computed tomography scan. -No underlying disease under treatment or follow-up.
Key exclusion criteria	-Patients with multiple detections of mycobacteria other than MAC. -Patients with macrolide-resistant MAC detected. -Patients whose therapy was discontinued due to adverse events, drug interaction, etc. -Patients who have been treated for less than 12 months. (If the MAC detected is macrolide susceptible, this criterion is not required.) -Patients within 1 year of completing prior treatment for MAC-PD. -Patients who underwent surgery for MAC-PD within 1 year. -Patients within 1 months of recovery from an acute infection. -Patients with chronic liver damage due to HBV or HCV. -HIV-positive patients. -Patients previously treated with immune checkpoint inhibitors for malignant tumors. -Patients with active hematologic malignancies. -Patients after blood stem cell transplants, including bone marrow transplants. -Patients treated for co-morbidities with high-dose glucocorticoid or immunosuppressive drugs. -Super-elderly people over 90 years old. -Pregnant women.
Target sample size	40

Research contact person
Name of lead principal investigator	1st name Yuichiro Middle name Last name Shindo
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Respiratory Medicine
Zip code	466-8550
Address	65 Tsurumai-cho, Showa-ku, Nagoya-shi, Aichi, 466-8550, Japan
TEL	052-744-2167
Email	yshindo@med.nagoya-u.ac.jp

Public contact
Name of contact person	1st name Akinobu Middle name Last name Matsuura
Organization	Nagoya University Graduate School of Medicine
Division name	Department of Respiratory Medicine
Zip code	466-8550
Address	65 Tsurumai-cho, Showa-ku, Nagoya-shi, Aichi, 466-8550, Japan
TEL	052-744-2167
Homepage URL
Email	akinobu@med.nagoya-u.ac.jp

Sponsor
Institute	Department of Respiratory Medicine, Nagoya University Graduate School of Medicine
Institute
Department

Funding Source
Organization	Japan Society for the Promotion of Science, Grants-in-Aid for Scientific Research
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	National Hospital Organization, Higashi-Nagoya Hospital
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Bioethics Review Committee, Nagoya University Hospital
Address	65 Tsurumai-cho, Showa-ku, Nagoya-shi, Aichi, 466-8550, Japan
Tel	052-744/2973
Email	ethics@med.nagoya-u.ac.jp

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2021 Year 03 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Enrolling by invitation
Date of protocol fixation	2020 Year 10 Month 26 Day
Date of IRB	2021 Year 02 Month 22 Day
Anticipated trial start date	2021 Year 03 Month 01 Day
Last follow-up date	2025 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other

Other related information

-This study is an observational study. -Collection of clinical information and blood samples are performed in both Nagoya University Hospital and Higashi-Nagoya Hospital. Immunological analysis of blood samples is performed in Nagoya University Graduate School of Medicine. -The protocol was revised to modify the exclusion criteria and to add the healthy subject group, and was re-approved by Institutional Review Board, Nagoya University Hospital on March 24, 2022.

Management information
Registered date	2021 Year 02 Month 24 Day
Last modified on	2022 Year 03 Month 31 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000049473