Condition category
Nutritional, Metabolic, Endocrine
Date applied
07/03/2007
Date assigned
07/03/2007
Last edited
12/08/2008
Prospective/Retrospective
Prospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Dr Marije Bakker

ORCID ID

Contact details

Division of Pediatric Respiratory Medicine
Room Sb-2666
Erasmus Medical Centre
Sophia Children's Hospital
Dr. Molenwaterplein 60
Rotterdam
3015 GJ
Netherlands
+31 (0)10 463 6683
e.bakker@erasmusmc.nl

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

2412325-3; NTR912

Study information

Scientific title

Acronym

Study hypothesis

Recombinant human deoxyribonuclease (rhDNase) targeted to the peripheral airways improves lung function in children with cystic fibrosis (CF) and a stable clinical condition.

Ethics approval

Ethics approval received from the Medical Ethical Committee of Erasmus MC Rotterdam on the 26th April 2007.

Study design

Randomised, active controlled, parallel group, double blinded, multicentre trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Condition

Cystic fibrosis

Intervention

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.

Intervention type

Drug

Phase

Not Specified

Drug names

Recombinant human deoxyribonuclease (rhDNase)

Primary outcome measures

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

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:
2.1. Maximum mean expiratory flow (MMEF25-75)
2.2. Forced expiratory volume in one second (FEV1)
2.3. Forced Vital Capacity (FVC)
3. Other study parameters, such as use of antibiotics and number of exacerbations (if applicable)

Overall trial start date

01/05/2007

Overall trial end date

01/05/2008

Reason abandoned

Eligibility

Participant 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

Participant type

Patient

Age group

Child

Gender

Not Specified

Target number of participants

50

Participant 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

Recruitment start date

01/05/2007

Recruitment end date

01/05/2008

Locations

Countries of recruitment

Italy, Netherlands

Trial participating centre

Division of Pediatric Respiratory Medicine, Room Sb-2666
Rotterdam
3015 GJ
Netherlands

Sponsor information

Organisation

Erasmus Medical Centre (The Netherlands)

Sponsor details

Sophia Children's Hospital
Dr. Molewaterplein 60
Rotterdam
3015 GJ
Netherlands

Sponsor type

Hospital/treatment centre

Website

http://www.erasmusmc.nl/

Funders

Funder type

Industry

Funder name

Roche Nederland B.V. (The Netherlands)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Funder name

Erasmus Medical Centre (The Netherlands)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes