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

• Register: ISRCTN
• Last refreshed on: 2 October 2017
• Main ID: ISRCTN74815264
• Date of registration: 20/12/2005
• Prospective Registration: No
• Primary sponsor: Roche Nederland BV (Netherlands)
• Public 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
• 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
• Date of first enrolment: 01/09/2004
• Target sample size: 25
• Recruitment status: Completed
• URL: http://isrctn.com/ISRCTN74815264
• Study type: Interventional
• Study design: Randomised double blind placebo controlled trial (Treatment)
• Phase: Not Applicable

Countries of recruitment

Netherlands

Contacts

• Name: Lianne van der Giessen

Key inclusion & exclusion criteria

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
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

Age minimum:
Age maximum:
Gender: Both

Health Condition(s) or Problem(s) studied

Cystic fibrosis
Nutritional, Metabolic, Endocrine
Cystic fibrosis

Intervention(s)

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.

Primary Outcome(s)

Pulmonary function test: MEF25

Secondary Outcome(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

Secondary ID(s)

NTR282

Source(s) of Monetary Support

Roche Nederland BV (Netherlands)