Peripheral targeting of inhaled recombinant human deoxyribonuclease in stable cystic fibrosis patients
| ISRCTN | ISRCTN64225851 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN64225851 |
| Secondary identifying numbers | 2412325-3; NTR912 |
- Submission date
- 07/03/2007
- Registration date
- 07/03/2007
- Last edited
- 12/08/2008
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nutritional, Metabolic, Endocrine
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Marije Bakker
Scientific
Scientific
Division of Pediatric Respiratory Medicine, Room Sb-2666
Erasmus Medical Centre
Sophia Children's Hospital
Dr. Molenwaterplein 60
Rotterdam
3015 GJ
Netherlands
| Phone | +31 (0)10 463 6683 |
|---|---|
| e.bakker@erasmusmc.nl |
Study information
| Study design | Randomised, active controlled, parallel group, double blinded, multicentre trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Scientific title | |
| Study objectives | 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(s) | Ethics approval received from the Medical Ethical Committee of Erasmus MC Rotterdam on the 26th April 2007. |
| Health condition(s) or problem(s) studied | 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 |
| Pharmaceutical study type(s) | |
| Phase | Not Specified |
| Drug / device / biological / vaccine name(s) | Recombinant human deoxyribonuclease (rhDNase) |
| Primary outcome measure | 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 study start date | 01/05/2007 |
| Completion date | 01/05/2008 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 6 Years |
| Upper age limit | 18 Years |
| Sex | Not Specified |
| Target number of participants | 50 |
| Key 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 |
| Key exclusion criteria | 1. Inability to follow instructions of the investigator 2. Inability to inhale rhDNase 3. Clinical condition not stable, as assessed by the patients 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 patients paediatrician 9. Active allergic bronchopulmonary aspergillosis (ABPA) defined as an oral course of prednisone for ABPA within the last three months |
| Date of first enrolment | 01/05/2007 |
| Date of final enrolment | 01/05/2008 |
Locations
Countries of recruitment
- Italy
- Netherlands
Study participating centre
Division of Pediatric Respiratory Medicine, Room Sb-2666
Rotterdam
3015 GJ
Netherlands
3015 GJ
Netherlands
Sponsor information
Erasmus Medical Centre (The Netherlands)
Hospital/treatment centre
Hospital/treatment centre
Sophia Children's Hospital
Dr. Molewaterplein 60
Rotterdam
3015 GJ
Netherlands
| Website | http://www.erasmusmc.nl/ |
|---|---|
"ROR" |
https://ror.org/018906e22 |
Funders
Funder type
Industry
Roche Nederland B.V. (The Netherlands)
No information available
Erasmus Medical Centre (The Netherlands)
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
