Peripheral targeting of inhaled rhDNase in stable CF patients.
- Conditions
- Cystic Fibrosis (CF)10038716
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 25
The criteria of inclusion will be the following:
- Age between 6 and 18 years old;
- Diagnosis of CF confirmed by sweat-test and/or DNA analysis and/or electro physiology testing (nasal potential difference measurement);
- Routine treatment with rhDNase once daily, started at least one month before enrolment in the study;
- Stable condition, in this study defined as: condition of patient judged to be stable by the treating physician AND 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 antibiotics course, no newly started inhaled or systemic corticosteroids etc).
- Ability to perform lung function tests (assessed by trained lung function technician);
- Lung function: FVC > 40% predicted;
- Signed written informed consent.
The following exclusion criteria will be used:
- Inability to follow instructions of the investigator;
- Inability to inhale rhDNase;
- Concomitant medical conditions that effect inhaled treatment (e.g. cleft palate, severe malacia);
- Current respiratory tract infection;
- Pulmonary complications that might put the patient at risk to participate in the study;
- Neuromuscular disease;
- Poor compliance with treatment as assessed by the patient*s paediatrician;
- Active ABPA (allergic bronchopulmonary aspergillosis) defined as an oral course of prednisone for ABPA within the last three months.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Primary endpoint will be the change in FEF75 compared to baseline after one<br /><br>month of treatment. FEF75 is the most suitable endpoint since it is sensitive<br /><br>to peripheral airways obstruction. </p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary endpoints will include:<br /><br>Lung clearance index (LCI) measurements as assessed by multiple breath washout;<br /><br>Other values obtained in the flow volume curve: MMEF25-75, FEV1, FVC.<br /><br>Other study parameters, such as use of antibiotics and number of exacerbations<br /><br>(if applicable)</p><br>