Cystic Fibrosis, rhDNase (Pulmozyme), inhalation, airways.
Conditions
Interventions
25 patients will receive four weeks of treatment with inhaled rhDNase targeted to the peripheral airways and 25 patients will receive four weeks of treatment with inhaled rhDNase targeted to the centr
Sponsors
Investigator initiated study.
Initiator: Harm Tiddens, M.D. PhD.
ErasmusMC - Sophia Children's Hospital
Eligibility
Inclusion criteria
Inclusion criteria: 1. Age between 6 and 18 years old; 2. Diagnosis of CF confirmed by sweat-test and/or DNA analysis and/or electro physiology testing (nasal potential difference measurement); 3. Routine treatment with rhDNase once daily, started at least one month before enrolment in the study; 4. Stable condition, in this study defined as: no i.v antibiotics (hospital or at home) in the previous month and constant medication regime during the previous 2 weeks (for example: no additional oral antibiotics course, no newly started inhaled or systemic corticosteroids etc). 5. Ability to perform lung function tests (assessed by trained lung function technician); 6. Lung function: FVC > 40% predicted; 7. Signed written informed consent.
Exclusion criteria
Exclusion criteria: 1. Inability to follow instructions of the investigator; 2. Inability to inhale rhDNase; 3. Clinical condition not stable, as assessed by the patient’s paediatrician; 4. Concomitant medical conditions that effect inhaled treatment (e.g. cleft palate, severe malacia); 5. Current respiratory tract infection; 6. Pulmonary complications that might put the patient at risk to participate in the study; 7. Neuromuscular disease; 8. Poor compliance with treatment as assessed by the patient’s paediatrician; 9. Active ABPA (allergic bronchopulmonary aspergillosis) defined as an oral course of prednisone for ABPA within the last three months.
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints will include: 1. Lung clearance index (LCI) measurements as assessed by multiple breath washout; 2. Other values obtained in the flow volume curve: MMEF25-75, FEV1, FVC. 3. Other study parameters, such as use of antibiotics and number of exacerbations (if applicable). | — |
Primary
| Measure | Time frame |
|---|---|
| Primary endpoint will be the change in FEF75 as a result of treatment. FEF75 is the most suitable endpoint since it is sensitive to peripheral airways obstruction. | — |
Contacts
Public ContactMarije Bakker
Division of Pediatric Respiratory Medicine Room Sb-2666 Erasmus MC - Sophia Children's Hospital Dr. Molenwaterplein 60
Outcome results
None listed