‘Peripheral targeting of inhaled rhDNase in stable CF patients.’

Conditions: Cystic Fibrosis
MedDRA version: 9.1Level: LLTClassification code 10011762Term: Cystic fibrosis
MedDRA version: 9.1Level: LLTClassification code 10011763Term: Cystic fibrosis lung

Registration Number: EUCTR2007-000935-25-NL

Lead Sponsor: ErasmusMC - Sophia Children's Hospital

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 50

Inclusion Criteria

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: 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).
- Ability to perform lung function tests (assessed by trained lung function technician);
- Lung function: FVC ³ 40% predicted;
- Signed written informed consent.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years)
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The following exclusion criteria will be used:
- Inability to follow instructions of the investigator;
- Inability to inhale rhDNase;
- Clinical condition not stable, as assessed by the patient’s paediatrician;
- 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 clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate the effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on FEF75 (a lung function parameter for peripheral airflow limitation) in children with CF who are on maintenance treatment with rhDNase.;Secondary Objective: To investigate the effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on lung clearance index (LCI), and on spirometry parameters (MMEF25-75, FEV1 and FVC) in children with CF who are on maintenance treatment with rhDNase. ;Primary end point(s): 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. <br><br>Secondary endpoints will include: <br>- Lung clearance index (LCI) measurements as assessed by multiple breath washout; <br>- Other values obtained in the flow volume curve: MMEF25-75, FEV1, FVC.<br>

Secondary Outcome Measures