Inhaled Nitric Oxide for Patients With MABSC

NCT03208764 | Completed Phase 2

• 1 other identifier: AIT_ CP_ CFNTM_01
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Mycobacterium Abscessus Infection

Outcome Measures

Primary Outcomes (1)

• Safety measured by NO-related Serious Adverse Events

  21 days

Secondary Outcomes (4)

• 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

Study Arms (1)

Nitric Oxide treatment

EXPERIMENTAL

Drug: Nitric Oxide

Interventions

Nitric Oxide DRUG

Subjects will be treated with 91 thirty minute sessions of 160 ppm Nitric Oxide with at least 3 hours between each session.

Nitric Oxide treatment

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects (Male or female) between 6 to 65 years old
• 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.
• History of at least 6 months chronic infection with Mycobacterium abscessus.
• Mycobacterium abscessus positive sputum sample at screening or prior to screening.
• 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.
• FEV1 ≥ 30% at screening as well as baseline.
• Ability to produce sputum or be willing to undergo an induction of sputum for clinical evaluation.
• Life expectancy ≥1 year
• Ability to perform 6MWT, but not to exceed a distance of 700 meters during screening.
• Ability to understand and comply with study requirements.
• Approved and signed informed consent by subject and/or by parent/legal guardian (depending on subject's age).

You may not qualify if:

• Breastfeeding or pregnancy as evidenced by a positive pregnancy test.
• Subjects diagnosed with methemoglobinemia, immunodeficiency, heart disease, pulmonary hypertension and/or high blood pressure.
• 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.
• History of frequent epistaxis (\>1 episode/month).
• Significant hemoptysis during the last 30 days prior to enrolment (\>30 Ml of blood in a 24-hour period).
• Methemoglobin level \>2% at screening.
• Patients on inconsistant dosage of systemic steroids (any formulation) 30 days prior to enrolment.
• History of illicit drug or medication abuse within 1 year of screening.
• History of lung transplantation.
• History of daily, continuous oxygen supplementation.
• 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.
• Pulmonary tuberculosis requiring treatment or treated within 2 years prior to screening.
• Any change in chronic NTM multi-drug regimen within 28 days prior to study day 1.

Sponsors & Collaborators

• Beyond Air Inc.: lead

Study Sites (1)

Rambam Medical Center

Haifa, Israel

Location

MeSH Terms

Conditions

Mycobacterium Infections, Nontuberculous

Interventions

Nitric Oxide

Condition Hierarchy (Ancestors)

Mycobacterium Infections; Actinomycetales Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections

Intervention Hierarchy (Ancestors)

Reactive Nitrogen Species; Free Radicals; Inorganic Chemicals; Nitrogen Oxides; Nitrogen Compounds; Oxides; Oxygen Compounds; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2017
• First Posted: July 6, 2017
• Study Start: July 24, 2017
• Primary Completion: May 1, 2018
• Study Completion: April 11, 2019
• Last Updated: July 9, 2019

Record last verified: 2017-07

Locations

IL (1)