A Pilot Study to Assess the Effect of Intermittent iNO on the Treatment of NTM Lung Infection in CF and Non-CF Patients

Not Applicable

Completed

• Conditions: Cystic Fibrosis; Mycobacterial Pneumonia; Mycobacterium Avium Complex; Mycobacterium Abscessus Infection; Non-Tuberculous Mycobacterial Pneumonia
• Interventions: Device: LungFit

• Registration Number: NCT04685720
• Lead Sponsor: Beyond Air Inc.

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.

Study Timeline

• Study Start: 2020-12-07
• Study First Submitted: 2020-12-07
• Study First Submitted QC: 2020-12-24
• Study First Posted: 2020-12-28
• Primary Completion: 2022-07-28
• Study Completion: 2022-10-10
• Status Verified: 2022-12
• Last Update Submitted: 2022-12-14
• Last Update Posted: 2022-12-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Inhaled NO delivered using LungFit	LungFit	Inhaled Nitric Oxide in doses up to 250 ppm

Primary Outcome Measures

Name	Time	Method
Treatment-Emergent SAEs	Day 1 to Day 84	The primary endpoint of the study is the number of patients with treatment-emergent SAEs

Secondary Outcome Measures

Name	Time	Method
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 FEV1 from baseline to Day 174	Day 1 to Day 174	Respiratory function will be assessed by spirometry including FEV1.
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
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 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.

Trial Locations

Locations (1)

Gallipoli Medical Research Foundation; 🇦🇺 Greenslopes, Queensland, Australia