Peripheral targeting of inhaled rhDNase in stable CF patients.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 50
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •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.
排除标准
- •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.