GM-CSF Inhalation for Nontuberculous Mycobacterial Lung Disease Pilot Trial

JRCT ID: jRCTs031210062

Registered date:30/04/2021

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Nontuberculous mycobacterial lung disease
• Date of first enrollment: 22/09/2021
• Target sample size: 15
• Study type: Interventional

Intervention(s)

1) Dosage
High dosage group: Sargramostim 250 microgram at a time, twice a day
Intermediate dosage group: Sargramostim 125 microgram at a time, twice a day
Low dosage group: Sargramostim 125 microgram at a time, once a day
2) Administration
In high dosage group, 1 vial is dissolved in 2 mL of normal saline followed by setting all solution in designated nebulizer.
In intermediate and low dosage groups, 1 vial is dissolved in 4 mL followed by setting 2 mL solution in designated nebulizer.
In all groups, subjects inhalate with designated compressor until running out of inhalation solution. Inhalation takes 6 - 15 minutes at a time, twice a day (morning and evening) in high and intermediate dosage groups and once a day (morning) in low dose group. 1 course is 2 weeks which consists of inhalation for 7 days followed by withdrawal for 7 days. 1 course is repeated 8 times, 16 weeks in total.

Outcome(s)

• Primary Outcome: Rate of patients who show negative mycobacterial culture test in sputume 3 times in a row after initiation of intervention

Secondary Outcome

1. Number of patients who show negative mycobacterial culture test in sputum after initiation of intervention at 12-week and 16-week in a row or at 16-week
2. Improvement in mycobacterial smear test in sputum at 4, 8, 12, and 16-week from baseline
3. Change in QOL score at 16-week from baseline
4. Improvement in chest radiograph
5. Change in anti-GM-CSF antibody titer
6. Change in serum cytokine levels

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

Subjects meeting all of the conditions below:
1. Age 20 or more at the time of obtaining informed consent, regardless of gender
2. Information about the name and condition of the disease is appropriately provided in advance
3. Ability to understand the explanation of this trial and give written voluntary consent
4. Meet the diagnostic criteria of nontuberculous mycobacterial lung disease. <Positive mycobacterial culture test>, <Negative, +-, or 1+ mycobacterial smear test>, and Mycobacterium avium or M. intracellulare is detected in sputum twice within 2 years.
5. Meet a) or b) below:
a) Sustained positive mycobacterial culture test after chemotherapy against nontuberculous mycobacterial lung disease for 6 months or more.
b) Withdrawal of chemotherapy against nontuberculous mycobacterial lung disease due to insufficient effectiveness or adverse effects 28 days or more before assessment for eligibility

Exclude criteria

Subjects meeting one or more conditions below:
1. 12000/mm3 or more of WBC on enrollment
2. Previously treated with GM-CSF inhalation
3. 60 ml or more per 24 hours of Hemoptysis within 4 weeks
4. Cardiovascular diseases, congestive heart failure/angina pectoris/hemorrhagic diathesis etc., with severe symptoms
5. History of malignant tumor with in 5 years except treated uterine carcinoma in situ and local basal cell carcinoma
6. Systemic corticosteroid therapy with 10 mg/day or more on a prednisolone conversion basis within 3 months or the therapy is expected during this trial
7. Change of chemotherapy regimen against nontuberculous mycobacterial lung disease wihtin 28 days
8. Pregnancy, breast-feeding, or desire for childbearing during this trial
9. Liver dysfunction <AST and/or ALT is 100 IU/L>
10. Renal dysfunction <CCr by Cockcroft & Gault equation < 30>
11. History of severe or unexplainable adverse effect by inhalatoin therapy
12. Incapable of informed consent, subject whose informed consent may not be voluntary
13. Ineligible for this trial at the medical discretion of the principal investigator

Related Information

• Primary Sponsor: Kikuchi Toshiaki
• Source(s) of Monetary Support: Nobelpharma Co., Ltd.,Japan Agency for Medical Research and Development