Clinical trial NCT02722122: Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of AIR DNase™in Patients With Cystic Fibrosis Previously Treated With Pulmozyme®
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®
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: Cystic Fibrosis
CF
AIR DNase™
Digestive System Diseases
Genetic Diseases, Inborn
Infant, New born, Diseases
Lung diseases
Pancreatic Diseases
Respiratory Tract Diseases
Lead sponsor: Protalix
Sponsor class: INDUSTRY
First posted: 2016-03-29
Enrollment: 15
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Adverse events following daily administration of AIR DNase™
timeFrame: 56 days
Secondary outcomes:
measure: Area under the curve
timeFrame: Up to 4 hours
measure: Change from baseline to end of AIR DNase™ treatment in FEV1
timeFrame: Baseline and 28 days
measure: Change from baseline to end of AIR DNase™ treatment in FVC
timeFrame: Baseline and 28 days
Other outcomes:
Locations:
city: Haifa
country: Israel
facility: Carmel MC
city: Haifa
country: Israel
facility: Rambam MC
city: Jerusalem
country: Israel
facility: Hadassah MC
city: Petah Tikva
country: Israel
facility: Schneider MC
city: Ramat Gan
country: Israel
facility: Sheba MC
Study officials:
affiliation: Sr. Director Clinical Development
name: Einat Dekel, DVM
role: STUDY_DIRECTOR
References: