Inhaled Nitric Oxide for Patients With MABSC

A Prospective, Open Labeled, Multi-Center, Evaluation of the Efficacy Safety and Tolerability of Nitric Oxide Given Intermittently Via Inhalation to Subjects With NTM (Specifically MABSC)

A Prospective, Open labeled, multi-Center, Evaluation of the Efficacy Safety and Tolerability of Nitric Oxide Given Intermittently via Inhalation to Subjects with NTM (specifically MABSC)

Nitric Oxide (NO) has been shown to play a critical role in various biological functions. In the airways, NO is considered to play a key role in the innate immune system in which the first-line of host defense against microbes is built.

In vitro studies suggested that NO, in part per million (ppm) concentrations, possesses anti-microbial activity against a wide variety of phyla including fungi, and antibiotic-resistance bacteria.

In this study the investigators wish to evaluate the efficacy, safety and tolerability of 160 ppm Nitric Oxide intermittently delivered via inhalation to subjects infected with Non-tuberculosis mycobacteria (NTM), specifically with MABSC.

Study Overview

• Status: Completed
• Conditions: Mycobacterium Abscessus Infection
• Intervention / Treatment: Drug: Nitric Oxide

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Haifa, Israel
    • Rambam Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subjects (Male or female) between 6 to 65 years old
2. Diagnosis of pulmonary non-tuberculosis mycobacterial lung disease in accordance with the 2007 ATS/IDSA criteria with evidence of nodular bronchiectasis and/or cavitary disease by chest computed tomography (CT) and by culture.
3. History of at least 6 months chronic infection with Mycobacterium abscessus.
4. Mycobacterium abscessus positive sputum sample at screening or prior to screening.
5. Receiving ATS/IDSA guidelines-based treatment regimen defined as: adherent to a multi-drug regimen for at least 6 months prior to screening with persistently positive mycobacterial abscessus cultures.
6. FEV1 ≥ 30% at screening as well as baseline.
7. Ability to produce sputum or be willing to undergo an induction of sputum for clinical evaluation.
8. Life expectancy ≥1 year
9. Ability to perform 6MWT, but not to exceed a distance of 700 meters during screening.
10. Ability to understand and comply with study requirements.
11. Approved and signed informed consent by subject and/or by parent/legal guardian (depending on subject's age).

Exclusion Criteria:

1. Breastfeeding or pregnancy as evidenced by a positive pregnancy test.
2. Subjects diagnosed with methemoglobinemia, immunodeficiency, heart disease, pulmonary hypertension and/or high blood pressure.
3. Use of an investigational drug during the last 30 days prior enrollment and/or the subject is expected to participate in a new study within five months from enrollment to this study.
4. History of frequent epistaxis (>1 episode/month).
5. Significant hemoptysis during the last 30 days prior to enrolment (>30 Ml of blood in a 24-hour period).
6. Methemoglobin level >2% at screening.
7. Patients on inconsistant dosage of systemic steroids (any formulation) 30 days prior to enrolment.
8. History of illicit drug or medication abuse within 1 year of screening.
9. History of lung transplantation.
10. History of daily, continuous oxygen supplementation.
11. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the subject or the quality of the data.
12. Pulmonary tuberculosis requiring treatment or treated within 2 years prior to screening.
13. Any change in chronic NTM multi-drug regimen within 28 days prior to study day 1.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Nitric Oxide treatment	Drug: Nitric Oxide; Subjects will be treated with 91 thirty minute sessions of 160 ppm Nitric Oxide with at least 3 hours between each session.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety measured by NO-related Serious Adverse Events	21 days

Secondary Outcome Measures

Outcome Measure	Time Frame
Six minute walk test	21 days
Mycobacterium Abscesuss load in sputum	81 days
NO-related AE's such as elevation of methemoglobin and elevation of NO2 during treatment	21 days
Type and frequency of occurrence of NO-related Adverse Events (AE's) and Serious AE's (SAE's)	51 days

Collaborators and Investigators

• Sponsor: Beyond Air Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 24, 2017
• Primary Completion (ACTUAL): May 1, 2018
• Study Completion (ACTUAL): April 11, 2019

Study Registration Dates

• First Submitted: July 3, 2017
• First Submitted That Met QC Criteria: July 3, 2017
• First Posted (ACTUAL): July 6, 2017

Study Record Updates

• Last Update Posted (ACTUAL): July 9, 2019
• Last Update Submitted That Met QC Criteria: July 8, 2019
• Last Verified: July 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Infections; Bacterial Infections; Bacterial Infections and Mycoses; Gram-Positive Bacterial Infections; Actinomycetales Infections; Mycobacterium Infections; Mycobacterium Infections, Nontuberculous; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Vasodilator Agents; Autonomic Agents; Peripheral Nervous System Agents; Protective Agents; Bronchodilator Agents; Anti-Asthmatic Agents; Respiratory System Agents; Antioxidants; Free Radical Scavengers; Endothelium-Dependent Relaxing Factors; Gasotransmitters; Nitric Oxide
• Other Study ID Numbers: AIT_ CP_ CFNTM_01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No