Peripheral targeting of inhaled rhDNase in stable CF patients.

• Register: Netherlands Trial Register
• Last refreshed on: 28 April 2013
• Main ID: NTR912
• Date of registration: 19/02/2007
• Primary sponsor: Erasmus Medical Center, Sophia Children's Hospital
• Public title: Peripheral targeting of inhaled rhDNase in stable CF patients.
• Scientific title: Peripheral targeting of inhaled rhDNase in stable CF patients. -
• Date of first enrolment: 1/5/2007
• Target sample size: 50
• Recruitment status: complete
• URL: http://www.trialregister.nl/trialreg/admin/rctview.asp?TC=912
• Study type: intervention
• Study design: Randomised: Yes; Masking: Double; Control: Active; Group: Parallel; Type: [default]

Countries of recruitment

The Netherlands

Contacts

• Name: Marije Bakker

• Name: H.A.W.M. Tiddens

Key inclusion & exclusion 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.


Age minimum:
Age maximum:
Gender:

Health Condition(s) or Problem(s) studied

Cystic fibrosis in children, Recombinant human deoxyribonuclease (rhDNase) (Pulmozyme), Inhalation, Airways

Intervention(s)

25 patients will receive four weeks of treatment with inhaled rhDNase targeted to the peripheral airways and 25 patients will receive four weeks of treatment with inhaled rhDNase targeted to the central airways. The central airways regimen is aimed to simulate equal deposition pattern as compared to conventional maintenance therapy. The peripheral airway regimen deposits a greater percentage of the medication in the peripheral airways.

Primary Outcome(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.

Secondary Outcome(s)

Secondary endpoints will include:
1. Lung clearance index (LCI) measurements as assessed by multiple breath washout;
2. Other values obtained in the flow volume curve: MMEF25-75, FEV1, FVC.
3. Other study parameters, such as use of antibiotics and number of exacerbations (if applicable).

Secondary ID(s)

2412325-3

ISRCTN64225851

Source(s) of Monetary Support

Roche Nederland BV, Erasmus Medical Center, Sophia Children's Hospital