Peripheral targeting of inhaled rhDNase in stable CF patients.
Overview
- Phase
- Not Applicable
- Intervention
- Not specified
- Conditions
- Cystic Fibrosis, rhDNase (Pulmozyme), inhalation, airways.
- Sponsor
- Investigator initiated study.Initiator: Harm Tiddens, M.D. PhD.ErasmusMC - Sophia Children's Hospital
- Enrollment
- 50
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
Earlier publications have shown that: - patients develop chronic airway infection and inflammation which start early in life [1. Dakin, C.J., et al., Inflammation, infection, and pulmonary function in infants and young children with cystic fibrosis. Am J Respir Crit Care Med, 2002. 165(7): p. 904-10. 2. Armstrong, D.S., et al., Lower airway inflammation in infants and young children with cystic fibrosis. Am J Respir Crit Care Med, 1997. 156(4 Pt 1): p. 1197-204. 3. Nixon, G.M., et al., Early airway infection, inflammation, and lung function in cystic fibrosis. Arch Dis Child, 2002. 87(4): p. 306-11]. And that: - Predominantly the peripheral airways are damaged and filled with sputum in CF [Tiddens, H.A., et al., Cartilaginous airway wall dimensions and airway resistance in cystic fibrosis lungs. Eur Respir J, 2000. 15(4): p. 735-42]. Therefore we hypothesize that rhDNase targeted to the peripheral airways can improve lung function in children with CF.
Investigators
Eligibility 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
- •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.
Outcomes
Primary Outcomes
Not specified