A double-blind, randomised, double dummy, cross-over, study to assess the difference in efficacy between nebulisation of rhDNase in the morning versus nebulisation before going to sleep

ISRCTN	ISRCTN74815264
DOI	https://doi.org/10.1186/ISRCTN74815264
Protocol serial number	NTR282
Sponsor	Roche Nederland BV (Netherlands)
Funder	Roche Nederland BV (Netherlands)

Submission date: 20/12/2005
Registration date: 20/12/2005
Last edited: 13/09/2017

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Nutritional, Metabolic, Endocrine

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr Lianne van der Giessen
Scientific

Erasmus Medical Center
Sophia Children's Hospital
Department Paediatric Physiotherapy
SK 0327, Dr Molewaterplein 60
Rotterdam
3015 GJ
Netherlands

Phone	+31 (0)10 463 6764
Email	L.vandergiessen@erasmusmc.nl

Study information

Primary study design	Interventional
Study design	Randomised double blind placebo controlled trial
Secondary study design	Randomised controlled trial
Scientific title	A double-blind, randomised, double dummy, cross-over, study to assess the difference in efficacy between nebulisation of rhDNase in the morning versus nebulisation before going to sleep
Study objectives	Inhalation of rhDNase before sleep increases the expiratory flow at 25% of the actual forced vital capacity (MEF25) compared to inhalation of rhDNase in the morning.
Ethics approval(s)	Received from the local medical ethics committee
Health condition(s) or problem(s) studied	Cystic fibrosis
Intervention	All subjects nebulised daily both rhDNase (2.5 mg of rhDNase in 2.5 ml buffered solution: 8.77 mg/ml sodium chloride and 0.15 mg/ml calcium chloride) and a placebo (2.5 ml of a buffered solution: 8.77 mg/ml sodium chloride and 0.15 mg/ml calcium chloride) once daily for a period of four weeks. Placebo was similar to rhDNase in both colour and taste. Subjects were randomised to two groups: 1. Group I used rhDNase before going to sleep and the placebo in the morning. Airway clearance techniques are performed 30 minutes after the nebulisation. In the following two rhDNase and placebo were taken in reversed order. 2. Group II used placebo before going to sleep and rhDNase in the morning. Airway clearance techniques are performed 30 minutes after the nebulisation. In the following two weeks placebo and rhDNase were taken in reversed order. Patients were asked to carry out their daily routine ACT and not to change their routine technique.
Intervention type	Drug
Phase	Not Applicable
Drug / device / biological / vaccine name(s)	rhDNase
Primary outcome measure(s)	Pulmonary function test: MEF25
Key secondary outcome measure(s)	1. Pulmonary function tests: FVC, forced expiratory volume in one second (FEV1), Rint 2. Frequency and duration of coughing measured with audio recording 3. Oxygenation at night recording transcutaneous oxygen saturation; percentage with saturation below 95% 4. Severity of cough with a verbal category descriptive (VCD) score 5. Sputum characteristics: amount, viscosity with a visual analogue scale (VAS) score 6. Quality of sleep and appetite with a VAS score 7. Presence of morning sickness
Completion date	01/02/2006

Eligibility

Participant type(s)	Patient
Age group	Other
Sex	All
Target sample size at registration	25
Key inclusion criteria	1. Proven cystic fibrosis (CF), as evidenced by an abnormal sweat test or an abnormal rectum potential difference measurement or by the presence of two CF mutations and at least one clinical feature of CF 2. Treated at the Erasmus Medical Centre - Sophia 3. Five years and older 4. Able to perform reproducible manoeuvres for spirometry 5. Maintenance treatment with rhDNase for at least one month 6. Clinically stable for at least one month (no intravenous antibiotics and/or hospitalisations within one month before enrolment) 7. Willing to participate in and comply with study procedures, and willingness of the parent or guardian and subjects greater than 12 years to provide written informed consent
Key exclusion criteria	1. Forced vital capacity (FVC) less than 40% 2. Using rhDNase more than once daily 3. Mentally retarded 4. History of non-adherence to treatment advice known to the physician
Date of first enrolment	01/09/2004
Date of final enrolment	01/02/2006

Locations

Countries of recruitment

Netherlands

Study participating centre

Erasmus Medical Center

Rotterdam
3015 GJ
Netherlands

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/10/2007		Yes	No

Editorial Notes

13/09/2017: internal review