Peripheral targeting of inhaled recombinant human deoxyribonuclease in stable cystic fibrosis patients

Completed

Conditions: Cystic fibrosis
Nutritional, Metabolic, Endocrine
Metabolic disorders

Registration Number: ISRCTN64225851

Lead Sponsor: Erasmus Medical Centre (The Netherlands)

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 50

Inclusion Criteria

1. Age between six and 18 years old
2. Diagnosis of CF confirmed by sweat-test and/or deoxyribonucleic acid (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 intravenous (i.v.) antibiotics (hospital or at home) in the previous month and constant medication regime during the previous two 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: forced vital capacity (FVC) greater than 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 allergic bronchopulmonary aspergillosis (ABPA) 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
Primary endpoint will be the change in forced expiratory flow (FEF75) as a result of treatment. FEF75 is the most suitable endpoint since it is sensitive to peripheral airways obstruction.

Secondary Outcome Measures

Name	Time	Method
Secondary endpoints will include: <br>1. Lung Clearance Index (LCI) measurements as assessed by multiple breath washout <br>2. Other values obtained in the flow volume curve:<br>2.1. Maximum mean expiratory flow (MMEF25-75)<br>2.2. Forced expiratory volume in one second (FEV1)<br>2.3. Forced Vital Capacity (FVC)<br>3. Other study parameters, such as use of antibiotics and number of exacerbations (if applicable)