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®

Protalix5 sites in 1 country15 target enrollmentMay 2016

ConditionsCystic Fibrosis

InterventionsAIR DNase™

DrugsAIR DNase™

Overview

Phase: Phase 2
Intervention: AIR DNase™
Conditions: Cystic Fibrosis
Sponsor: Protalix
Enrollment: 15
Locations: 5
Primary Endpoint: Adverse events following daily administration of AIR DNase™
Last Updated: 9 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

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.

Registry

clinicaltrials.gov

Start Date

May 2016

End Date

February 2017

Last Updated

9 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Protalix

Responsible Party

Sponsor

Eligibility Criteria

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

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.

Arms & Interventions

AIR DNase™ 2.5 mg

2.5 mg of AIR DNase™ administered once daily via inhalation for 28 days

Intervention: AIR DNase™

Outcomes

Primary Outcomes

Adverse events following daily administration of AIR DNase™

Time Frame: 56 days

Adverse events from subject reporting or other assessments

Secondary Outcomes

Change from baseline to end of AIR DNase™ treatment in FEV1(Baseline and 28 days)
Area under the curve(Up to 4 hours)
Change from baseline to end of AIR DNase™ treatment in FVC(Baseline and 28 days)