Clinical trial NCT04685720: 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
Overall status: COMPLETED
Phase: NA
Conditions: Non-Tuberculous Mycobacterial Pneumonia
Cystic Fibrosis
Mycobacterial Pneumonia
Mycobacterium Abscessus Infection
Mycobacterium Avium Complex
Lead sponsor: Beyond Air Inc.
Sponsor class: INDUSTRY
First posted: 2020-12-28
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Treatment-Emergent SAEs
timeFrame: Day 1 to Day 84
Secondary outcomes:
measure: Changes in NTM bacterial load from baseline to Day 174
timeFrame: Day 1 to Day 174
measure: Number of patients with culture conversion at Day 174
timeFrame: Day 1 to Day 174
measure: Changes in quality of life assessed by CFQ-R for or QOL-B with NTM module
timeFrame: Day 1 to Day 174
measure: Changes in FEV1 from baseline to Day 174
timeFrame: Day 1 to Day 174
measure: Changes in activity tracker data as assessed by changes in distance from baseline to Day 174.
timeFrame: Day 1 to Day 174
measure: Change in 6 Minute Walking Test
timeFrame: Day 1 to Day 84
Other outcomes:
Locations:
city: Greenslopes
country: Australia
facility: Gallipoli Medical Research Foundation
state: Queensland
Study officials:
References: