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

Plain English Summary

Not provided at time of registration

Study website

Type

Scientific

Contact name

Dr Lianne van der Giessen

ORCID ID

Contact details

Erasmus Medical Center
Sophia Children's Hospital
Department Paediatric Physiotherapy
SK 0327
Dr Molewaterplein 60
Rotterdam
3015 GJ
Netherlands
+31 (0)10 463 6764
L.vandergiessen@erasmusmc.nl

EudraCT/CTIS number

IRAS number

ClinicalTrials.gov number

Protocol/serial number

NTR282

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

Acronym

Study hypothesis

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

Study design

Randomised double blind placebo controlled trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Study setting(s)

Not specified

Study type

Treatment

Patient information sheet

Condition

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

Pharmaceutical study type(s)

Phase

Not Applicable

Drug/device/biological/vaccine name(s)

rhDNase

Primary outcome measure

Pulmonary function test: MEF25

Secondary outcome measures

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

Overall study start date

01/09/2004

Overall study end date

01/02/2006

Reason abandoned (if study stopped)

Participant 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

Participant type(s)

Patient

Age group

Other

Sex

Both

Target number of participants

25

Participant 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

Recruitment start date

01/09/2004

Recruitment end date

01/02/2006

Countries of recruitment

Netherlands

Study participating centre

Erasmus Medical Center
Rotterdam
3015 GJ
Netherlands

Organisation

Roche Nederland BV (Netherlands)

Sponsor details

Postbus 44
Woerden
3440 AA
Netherlands

Sponsor type

Industry

Website

http://www.roche.nl

ROR

https://ror.org/01gcg9888

Funder type

Industry

Funder name

Roche Nederland BV (Netherlands)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Individual participant data (IPD) sharing plan

IPD sharing plan summary

Not provided at time of registration

Study outputs