Efficacy of peripherally targeted inhaled rhDNase for persistent obstructive asthma in childhood

ISRCTN ISRCTN71537084
DOI https://doi.org/10.1186/ISRCTN71537084
EudraCT/CTIS number 2006-002337-20
Secondary identifying numbers 2412325-2, NL612, NTR671
Submission date
09/06/2006
Registration date
09/06/2006
Last edited
08/01/2021
Recruitment status
No longer recruiting
Overall study status
Completed
Condition category
Respiratory
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Dr H.A.W.M. Tiddens
Scientific

Erasmus Medical Center
Sophia Children’s Hospital Rotterdam
Department of Pediatric Pulmonology
Dr. Molewaterplein 60
Rotterdam
3015 GJ
Netherlands

Phone +31 (0)10 4636690
Email h.tiddens@erasmusmc.nl

Study information

Study designRandomised controlled trial
Primary study designInterventional
Secondary study designRandomised controlled trial
Study setting(s)Not specified
Study typeTreatment
Scientific titleEfficacy of peripherally targeted inhaled rhDNase for persistent obstructive asthma in childhood
Study acronymIDOL
Study objectivesRecombinant human deoxyribonuclease (rhDNase) improves lung function in children with persistent asthma who have persistent obstructive pulmonary function.

Treatment of mucus impaction is an interesting alternative approach to treating peripheral airflow limitation in asthmatic patients. In severe asthma, dramatic improvement has been described in a few patients after inhalation of the mucolytic rhDNase. In addition, in pathology studies, extensive mucus plugging has been described in asthmatic patients. Based on these findings we think that additional treatment benefit can be obtained when mucus plugging is targeted as part of asthma treatment. Especially for those asthmatic children with persistent peripheral airways obstruction, rhDNase is a well known and safe drug that could be used for this treatment. Therefore we hypothesise that rhDNase has additional effect on lung function in children with persistent asthma who have persistent obstructive pulmonary function.
Ethics approval(s)Not provided at time of registration
Health condition(s) or problem(s) studiedObstructive pulmonary function, asthma
InterventionNebulization with rhDNase or placebo once daily (each participant is treated for two weeks with rhDNase and for 2 weeks with placebo)
Intervention typeDrug
Pharmaceutical study type(s)
PhaseNot Specified
Drug / device / biological / vaccine name(s)Recombinant human deoxyribonuclease (rhDNase)
Primary outcome measurePrimary endpoint will be the change in FEF75 as a result of treatment. FEF75 is the most suitable endpoint since it is sensitive to peripheral airways obstruction.
Secondary outcome measuresSecondary endpoints will include:
1. Lung clearance index (LCI) measurements as assessed by multiple breath washout
2. Cumulative symptom diary scores evaluating asthma symptoms in the second week of intervention (e.g. shortness of breath, cough, exercise intolerance, bronchodilator use etc.);
3. Fraction of exhaled nitric oxide (FENO)
4. Other values obtained in the flow volume curve: FEV1, FVC, peak expiratory flow (PEF)
Overall study start date01/09/2006
Completion date01/01/2008

Eligibility

Participant type(s)Patient
Age groupChild
Lower age limit6 Years
Upper age limit18 Years
SexBoth
Target number of participants60
Total final enrolment64
Key inclusion criteria1. Aged 6 - 18 years
2. Asthma diagnosed according to Global Initiative For Asthma (GINA) guidelines
3. Attending the outpatient clinic for at least one year
4.Treatment with at least 400 mg/day inhaled budesonide or equivalent (dose constant for at least 6 months) and bronchodilators as needed or daily
5. Clinically stable asthma while using a constant dose of Inhaled Corticosteroid (ICS) for at least three months
6. Ability to perform lung function tests (assessed by trained lung function technician)
7. Persistent peripheral airways obstruction as assessed by pulmonary function testing, defined as: dissociation between forced vital capacity (FVC) and FEF75 values: FEF75 at least 20% (absolute % predicted) lower than FVC (FEF = Forced Expiratory Flow rate)
8. FVC within normal limits (for this study defined as FVC >80% predicted)
Key exclusion criteria1. Asthma exacerbation with hospital admission in last three months
2. Intensive care unit (ICU) admission for asthma within the last year
3. Current respiratory tract infection
4. Inability to follow instructions of the investigator
5. Inability to inhale rhDNase
6. Concomitant medical conditions that can affect inhaled treatment (e.g. cleft palate, severe malacia)
7. Neuromuscular disease
8. Smoking
Date of first enrolment01/09/2006
Date of final enrolment01/01/2008

Locations

Countries of recruitment

  • Netherlands

Study participating centre

Erasmus Medical Center
Rotterdam
3015 GJ
Netherlands

Sponsor information

Erasmus Medical Center (The Netherlands)
University/education

P.O. Box 2040
Rotterdam
3000 CA
Netherlands

ROR logo "ROR" https://ror.org/018906e22

Funders

Funder type

Industry

Roche Nederland BV

No information available

Results and Publications

Intention to publish date
Individual participant data (IPD) Intention to shareNo
IPD sharing plan summaryNot provided at time of registration
Publication and dissemination planNot provided at time of registration
IPD sharing plan

Study outputs

Output type Details Date created Date added Peer reviewed? Patient-facing?
Results article results 01/08/2013 08/01/2021 Yes No

Editorial Notes

08/01/2021: The following changes have been made:
1. Publication reference added.
2. The final enrolment number has been added from the reference.
3. The NTR numbers have been added.
4. The EudraCT number has been added.