Cystic Fibrosis, Mycobacterial Pneumonia, Mycobacterium Abscessus Infection, Mycobacterium Avium Complex, Non-Tuberculous Mycobacterial Pneumonia
Conditions
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.
Interventions
DEVICELungFit
LungFit for NTM is an experimental device that produces Nitric Oxide from the ambient air.
Sponsors
Beyond Air Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-Emergent SAEs | Day 1 to Day 84 | The primary endpoint of the study is the number of patients with treatment-emergent SAEs |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of patients with culture conversion at Day 174 | Day 1 to 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 | Day 1 to Day 174 | 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 NTM bacterial load from baseline to Day 174 | Day 1 to Day 174 | Changes in NTM bacterial load will be assessed by sputum culture in liquid and solid media. |
| Changes in activity tracker data as assessed by changes in distance from baseline to Day 174. | Day 1 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 | Day 1 to Day 84 | Change in 6 Minute Walking Test will be assessed by changes in distance between baseline and Day 84 |
| Changes in FEV1 from baseline to Day 174 | Day 1 to Day 174 | Respiratory function will be assessed by spirometry including FEV1. |
Countries
Australia
Outcome results
None listed