Evaluation of the Safety and Tolerability of Inhaled Nitric Oxide to Subjects With Cystic Fibrosis
Status:
Completed
Trial end date:
2015-11-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Advanced Inhalation Therapies Ltd Beyond Air Inc.
Collaborators:
Schneider Children's Medical Center, Israel Soroka University Medical Center