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

Phase 2

• Conditions: Cystic Fibrosis
• Interventions: Drug: AIR DNase™

• Registration Number: NCT02722122
• Lead Sponsor: Protalix

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.

Study Timeline

• Study First Submitted: 2016-03-23
• Study First Submitted QC: 2016-03-28
• Study First Posted: 2016-03-29
• Study Start: 2016-05
• Status Verified: 2016-09
• Last Update Submitted: 2016-09-26
• Last Update Posted: 2016-09-28
• Primary Completion: 2017-02

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

1. Age ≥ 12 years of age (inclusive) at the time of screening
2. Weight ≥ 36 kg
3. Prior confirmed diagnosis of CF
4. At least 4 months treatment with Pulmozyme® prior to screening.
5. The subject is medically stable for at least one month prior to the screening visit.
6. Stable inhaled regimen of either: antibiotics, steroids, hypertonic saline at least four months prior to screening visit.
7. FEV1 of >40% and <90% and FVC ≥ 40% of predicted normal for age, gender, and height at screening .
8. 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.
9. Be willing and able to adhere to the study visit schedule and other protocol requirements
10. Be willing and able to provide voluntary written informed consent

Main

Exclusion Criteria

1. Has a history of lung transplantation.
2. Female subjects who are pregnant or lactating.
3. History of severe or unexplained adverse reactions during aerosol delivery of any medicinal product.
4. History or presence of hypersensitivity or reaction to inhaled proteins.
5. Participation in another clinical trial within 60 days prior to screening.
6. 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.
7. Have positive serology for human immunodeficiency virus (HIV) or Hepatitis B or Hepatitis C infection.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Adverse events following daily administration of AIR DNase™	56 days	Adverse events from subject reporting or other assessments

Secondary Outcome Measures

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

Trial Locations

Locations (5)

Carmel MC; 🇮🇱 Haifa, Israel

Rambam MC; 🇮🇱 Haifa, Israel

Schneider MC; 🇮🇱 Petah Tikva, Israel

Hadassah MC; 🇮🇱 Jerusalem, Israel

Sheba MC; 🇮🇱 Ramat Gan, Israel