Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®

Study Overview
=================
Brief Summary
-----------------
This study evaluates the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients with Cystic Fibrosis previously treated with Pulmozyme®.

Detailed Description
-----------------
This is a proof-of-concept, open label study, to evaluate the safety, tolerability, pharmacokinetics and exploratory efficacy of 2.5 mg AIR DNase TM administered once daily for 28 days via inhalation to Cystic Fibrosis subjects who have previously been treated with Pulmozyme®. This multicenter study will be conducted in approximately 10 sites and will enroll up to 15 subjects.

Official Title
-----------------
A Proof-of-Concept, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®

Conditions
-----------------
Cystic Fibrosis

Intervention / Treatment
-----------------
* Drug: AIR DNase™


Participation Criteria
=================
Eligibility Criteria
-----------------
Main Inclusion Criteria: Age ≥ 12 years of age (inclusive) at the time of screening Weight ≥ 36 kg Prior confirmed diagnosis of CF At least 4 months treatment with Pulmozyme® prior to screening. The subject is medically stable for at least one month prior to the screening visit. Stable inhaled regimen of either: antibiotics, steroids, hypertonic saline at least four months prior to screening visit. FEV1 of >40% and <90% and FVC ≥ 40% of predicted normal for age, gender, and height at screening . Female and male subjects whose co-partners are of child bearing potential must agree to use two medically acceptable methods of contraception, not including the rhythm method. Be willing and able to adhere to the study visit schedule and other protocol requirements Be willing and able to provide voluntary written informed consent Main Exclusion Criteria: Has a history of lung transplantation. Female subjects who are pregnant or lactating. History of severe or unexplained adverse reactions during aerosol delivery of any medicinal product. History or presence of hypersensitivity or reaction to inhaled proteins. Participation in another clinical trial within 60 days prior to screening. Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's safety or compliance with the requirements of the study. Have positive serology for human immunodeficiency virus (HIV) or Hepatitis B or Hepatitis C infection.

Ages Eligible for Study
-----------------
Minimum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AIR DNase™ 2.5 mg<br>2.5 mg of AIR DNase™ administered once daily via inhalation for 28 days | Drug: AIR DNase™<br> <br> |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events following daily administration of AIR DNase™ | Adverse events from subject reporting or other assessments | 56 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Area under the curve | AIR DNase concentrations measured at 0, 0.25, 0.5, 0.75, 1, 1.5, 2, and 4 hours | Up to 4 hours | 
| Change from baseline to end of AIR DNase™ treatment in FEV1 |  | Baseline and 28 days | 
| Change from baseline to end of AIR DNase™ treatment in FVC |  | Baseline and 28 days | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Cystic Fibrosis, CF, AIR DNase™, Digestive System Diseases, Genetic Diseases, Inborn, Infant, New born, Diseases, Lung diseases, Pancreatic Diseases, Respiratory Tract Diseases