Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis

Summary

Cystic Fibrosis is defined as a genetic disorder affecting approximately 100,000 individuals worldwide. CF is caused by mutations in the CF Transmembrane Conductance Regulator (CFTR) gene. CF patients are highly prone to environmental opportunistic bacterial infections leading to prolonged and chronic lung infections. This results in reduction in the life expectancy of CF patients due to excessive lung tissue destruction.

Nitric Oxide (NO) is a naturally produced antimicrobial agent which is part of the innate immune defense system of the lung. Both in vitro and in vivo studies had shown clearly that NO acts against a wide variety of microbes including drug resistant bacteria as well as viruses and fungi. Building on a successful phase I safety trial, the team aims to develop a combined drug-device strategy to combat lung infections caused by biofilm-forming bacteria. Unlike other inhaled drugs, NO is also a smooth muscle relaxant and avoids the concomitant bronchial constriction often associated with inhaled antibiotics. An added benefit of NO therapy is its mucolytic activity. We suggest that the combine broad spectrum antimicrobial activity, signaling and mucolytic properties of NO, delivered to the lungs of CF patients, will be directed at reducing bacterial resistance, microbial burden and biofilms as well as resulting in improved airway clearance of viscid sputum.

Primary Objectives: Assess the safety and the tolerability of NO intermittent inhalation treatment in ≥10 years old CF subjects. Secondary Objective: Assess the improvement in forced expiratory volume in 1 second (FEV1) before and after NO intermittent inhalation. Up to 10 subjects with Cystic Fibrosis will be enrolled into the study.

Treatment administration: The subjects will receive intermittent inhalation of NO in addition to standard treatment for 10 working days (no NO treatment will be given to the subjects during weekend days). The subjects will be asked to attend the CF clinic once a week for a period of two weeks in order to evaluate the parameters related to the study. Oxygen (O2), NO, nitrogen dioxide (NO2) and fraction of inspired oxygen (FiO2) delivered to the patient will be continuously monitored.

Conditions

• Cystic Fibrosis

Interventions

DRUG

Nitric oxide

Sponsors & Collaborators

• Soroka University Medical Center

  collaborator OTHER

• Schneider Children's Medical Center, Israel

  collaborator OTHER

• Beyond Air Inc.

  lead INDUSTRY

Principal Investigators

• Asher Tal, M.D · Soroka University Medical Center

• Hannah Blau, M.D · Schneider Children's Medical Center, Israel

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

10 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-12-31

Primary Completion

2015-11-30

Completion

2015-11-30

Countries

• Israel

Study Locations