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

Cystic Fibrosis Clinical Trial

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®

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02722122
• Other study ID #: PB-110-CF02
• Status: Recruiting
• Phase: Phase 2
• First received: March 23, 2016
• Last updated: September 26, 2016
• Start date: May 2016

Study information

• Verified date: September 2016
• Source: Protalix
• Contact: Einat Dekel, DVM
• Email: Einat.Dekel[@]protalix.com
• Is FDA regulated: No
• Health authority: Israel: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. primary completion date: February 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years and older

Eligibility

Main 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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• AIR DNase™

Locations

Country	Name	City	State
Israel	Carmel MC	Haifa
Israel	Rambam MC	Haifa
Israel	Hadassah MC	Jerusalem
Israel	Schneider MC	Petah Tikva
Israel	Sheba MC	Ramat Gan

Sponsors (1)

Lead Sponsor	Collaborator
Protalix

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adverse events following daily administration of AIR DNase™	Adverse events from subject reporting or other assessments	56 days	Yes
Secondary	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	No
Secondary	Change from baseline to end of AIR DNase™ treatment in FEV1		Baseline and 28 days	No
Secondary	Change from baseline to end of AIR DNase™ treatment in FVC		Baseline and 28 days	No