Cystic Fibrosis
Conditions
Keywords
Cystic Fibrosis, CF, AIR DNase™, Digestive System Diseases, Genetic Diseases, Inborn, Infant, New born, Diseases, Lung diseases, Pancreatic Diseases, Respiratory Tract Diseases
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.
Interventions
DRUGAIR DNase™
Sponsors
Protalix
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events following daily administration of AIR DNase™ | 56 days | Adverse events from subject reporting or other assessments |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 FEV1 | Baseline and 28 days | — |
| Change from baseline to end of AIR DNase™ treatment in FVC | Baseline and 28 days | — |
Countries
Israel
Contacts
Primary ContactEinat Dekel, DVM
Backup ContactYasmin Omer Azulay, MPH
Outcome results
None listed