Back to resultsA Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF Patients
Primary Purpose
Non-Tuberculous Mycobacterial Pneumonia, Cystic Fibrosis, Mycobacterial Pneumonia
Status
Completed
Phase
Not Applicable
Locations
Australia
Study Type
Interventional
Intervention
LungFit
Sponsored by
About this trial
This is an interventional treatment trial for Non-Tuberculous Mycobacterial Pneumonia
Eligibility Criteria
Inclusion Criteria:
- Subjects diagnosed with refractory NTM (MAC or MAbs) pulmonary infection
- CF and Non-CF patients
Exclusion Criteria:
- Diagnosis of methemoglobinemia or MetHb ≥2% at screening; treatment with any drug known to increase MetHb; known or suspected hemoglobinopathy.
- History of or current myeloproliferative disease, leukemia or other hematological malignancy; known or suspected immunodeficiency disease.
- Subjects with advanced cardiovascular disease or CHF
- Use of an investigational drug during the 30 days prior to enrollment.
- History of frequent epistaxis (>1 episode/month); significant hemoptysis (during the 30 days prior to enrollment.
- Subject on non-constant dose of systemic steroids within 30 days prior to enrollment; subjects on constant systemic steroids if the daily dose is higher than 10 mg/d prednisolone or equivalent.
- Active pulmonary malignancy (primary or metastatic) or any malignancy; history of lung transplantation.
- Pulmonary tuberculosis requiring treatment or treated within 2 years prior to screening.
- Uncontrolled hypertension within 3 months prior to or at screening
- Diagnosis of significant pulmonary hypertension indicated on echocardiogram at screening
- Clinically significant renal or liver laboratory abnormalities
- History of daily, continuous oxygen supplementation.
- Women of childbearing potential - pregnant or breastfeeding, or not on medically acceptable double methods of contraception from enrollment until Day 84.
- Smoking tobacco or any substance within 6 months prior to screening or anticipated inability to refrain from smoking throughout the study.
- Patient receiving drugs that have a contraindication with NO
Sites / Locations
- Gallipoli Medical Research Foundation
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Inhaled NO delivered using LungFit
Arm Description
Inhaled Nitric Oxide in doses up to 250 ppm
Outcomes
Primary Outcome Measures
Treatment-Emergent SAEs
The primary endpoint of the study is the number of patients with treatment-emergent SAEs
Secondary Outcome Measures
Changes in NTM bacterial load from baseline to Day 174
Changes in NTM bacterial load will be assessed by sputum culture in liquid and solid media.
Number of patients with culture conversion at Day 174
NTM culture conversion will be defined as having at least three consecutive negative NTM cultures
Changes in quality of life assessed by CFQ-R for or QOL-B with NTM module
Changes in quality of life assessed by Cystic Fibrosis Questionnaire Revised [CFQ R] for CF patients or Quality of Life Questionnaire-Bronchiectasis [QOL-B] with NTM module for non-CF patients.
Changes in FEV1 from baseline to Day 174
Respiratory function will be assessed by spirometry including FEV1.
Changes in activity tracker data as assessed by changes in distance from baseline to Day 174.
Patients will collect activity tracker data from 2 weeks before treatment and from Day 1 to Day 174.
Change in 6 Minute Walking Test
Change in 6 Minute Walking Test will be assessed by changes in distance between baseline and Day 84
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04685720
Brief Title
A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF Patients
Official Title
A Pilot Study to Assess the Effect of Intermittent Inhaled Nitric Oxide on the Treatment of Nontuberculous Mycobacteria (NTM) Lung Infection in Cystic Fibrosis and Non-Cystic Fibrosis Patients
Study Type
Interventional
2. Study Status
Record Verification Date
December 2022
Overall Recruitment Status
Completed
Study Start Date
December 7, 2020 (Actual)
Primary Completion Date
July 28, 2022 (Actual)
Study Completion Date
October 10, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Beyond Air Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this open-label, multicenter, non-randomized, pilot study is to assess the safety of high dose intermittent iNO for treatment of NTM infection in CF and non-CF patients.
Detailed Description
The study will include 20 patients from up to four clinical sites in Australia. The overall treatment plan includes 2 weeks of inhalation treatments (intensive phase) of iNO four times per day at 4.5-hour intervals followed by 10 weeks of inhalation treatments (maintenance phase) at the maximum tolerated dose (a maximum of 250 ppm NO) inhaled twice daily.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Non-Tuberculous Mycobacterial Pneumonia, Cystic Fibrosis, Mycobacterial Pneumonia, Mycobacterium Abscessus Infection, Mycobacterium Avium Complex
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Inhaled NO delivered using LungFit
Arm Type
Experimental
Arm Description
Inhaled Nitric Oxide in doses up to 250 ppm
Intervention Type
Device
Intervention Name(s)
LungFit
Intervention Description
LungFit for NTM is an experimental device that produces Nitric Oxide from the ambient air.
Primary Outcome Measure Information:
Title
Treatment-Emergent SAEs
Description
The primary endpoint of the study is the number of patients with treatment-emergent SAEs
Time Frame
Day 1 to Day 84
Secondary Outcome Measure Information:
Title
Changes in NTM bacterial load from baseline to Day 174
Description
Changes in NTM bacterial load will be assessed by sputum culture in liquid and solid media.
Time Frame
Day 1 to Day 174
Title
Number of patients with culture conversion at Day 174
Description
NTM culture conversion will be defined as having at least three consecutive negative NTM cultures
Time Frame
Day 1 to Day 174
Title
Changes in quality of life assessed by CFQ-R for or QOL-B with NTM module
Description
Changes in quality of life assessed by Cystic Fibrosis Questionnaire Revised [CFQ R] for CF patients or Quality of Life Questionnaire-Bronchiectasis [QOL-B] with NTM module for non-CF patients.
Time Frame
Day 1 to Day 174
Title
Changes in FEV1 from baseline to Day 174
Description
Respiratory function will be assessed by spirometry including FEV1.
Time Frame
Day 1 to Day 174
Title
Changes in activity tracker data as assessed by changes in distance from baseline to Day 174.
Description
Patients will collect activity tracker data from 2 weeks before treatment and from Day 1 to Day 174.
Time Frame
Day 1 to Day 174
Title
Change in 6 Minute Walking Test
Description
Change in 6 Minute Walking Test will be assessed by changes in distance between baseline and Day 84
Time Frame
Day 1 to Day 84
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subjects diagnosed with refractory NTM (MAC or MAbs) pulmonary infection
CF and Non-CF patients
Exclusion Criteria:
Diagnosis of methemoglobinemia or MetHb ≥2% at screening; treatment with any drug known to increase MetHb; known or suspected hemoglobinopathy.
History of or current myeloproliferative disease, leukemia or other hematological malignancy; known or suspected immunodeficiency disease.
Subjects with advanced cardiovascular disease or CHF
Use of an investigational drug during the 30 days prior to enrollment.
History of frequent epistaxis (>1 episode/month); significant hemoptysis (during the 30 days prior to enrollment.
Subject on non-constant dose of systemic steroids within 30 days prior to enrollment; subjects on constant systemic steroids if the daily dose is higher than 10 mg/d prednisolone or equivalent.
Active pulmonary malignancy (primary or metastatic) or any malignancy; history of lung transplantation.
Pulmonary tuberculosis requiring treatment or treated within 2 years prior to screening.
Uncontrolled hypertension within 3 months prior to or at screening
Diagnosis of significant pulmonary hypertension indicated on echocardiogram at screening
Clinically significant renal or liver laboratory abnormalities
History of daily, continuous oxygen supplementation.
Women of childbearing potential - pregnant or breastfeeding, or not on medically acceptable double methods of contraception from enrollment until Day 84.
Smoking tobacco or any substance within 6 months prior to screening or anticipated inability to refrain from smoking throughout the study.
Patient receiving drugs that have a contraindication with NO
Facility Information:
Facility Name
Gallipoli Medical Research Foundation
City
Greenslopes
State/Province
Queensland
ZIP/Postal Code
4120
Country
Australia
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF Patients
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