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Trial registered on ANZCTR


Registration number
ACTRN12624001381572p
Ethics application status
Submitted, not yet approved
Date submitted
31/10/2024
Date registered
21/11/2024
Date last updated
21/11/2024
Date data sharing statement initially provided
21/11/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Feasibility study of 2-weeks of erdosteine used during exacerbations in children with bronchiectasis
Scientific title
Evaluating the effect of 2-weeks erdosteine, a novel mucolytic, as add-on treatment during respiratory exacerbations in children with bronchiectasis - a feasibility study
Secondary ID [1] 313269 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record
This is a follow-up study of a study in children and adults evaluating the effect of erdosteine on respiratory exacerbation rate of children and adults with bronchiectasis taken twice daily for a 12 month period - a multicentre double-blind, randomised controlled trial.
ACTRN12621000315819

Health condition
Health condition(s) or problem(s) studied:
Bronchiectasis 335596 0
Condition category
Condition code
Respiratory 332163 332163 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Erdosteine 300mg capsules: Oral twice daily doses for 2 weeks [one capsule (300mg) per dose for children >20kg; half capsule (150mg) per dose for children 15-20kg]

Adherence will be monitored by return of bottles or capsule packaging.

In a subset of participants, we will also evaluate if inflammatory and/or microbial biomarkers will identify pathways and/or predict those at greater risk of recurrent respiratory exacerbations. These outcomes will not be reported in the trial. Participants for this component will be based on whether they consent to these additional procedures (sputum and blood). Where possible, bloods and sputum will be taken at baseline and at commencement of an exacerbation.
Intervention code [1] 329848 0
Treatment: Drugs
Comparator / control treatment
Placebo = matching placebo microcellulose capsule.
Oral twice daily doses for 2 weeks [one capsule per dose for children >20kg; half capsule per dose for children 15-20kg]
Control group
Placebo

Outcomes
Primary outcome [1] 339754 0
Duration of symptoms of respiratory exacerbation
Timepoint [1] 339754 0
End of respiratory exacerbation (total number of days through continuous reporting)
Secondary outcome [1] 441141 0
Quality of Life (QoL)
Timepoint [1] 441141 0
Change in QoLs scores at weeks 1, 2 and 4 from commencement of intervention
Secondary outcome [2] 441142 0
Spirometry values (FEV1 % predicted)
Timepoint [2] 441142 0
Change in values at 4 weeks from commencement of intervention
Secondary outcome [3] 441143 0
Time to next acute respiratory exacerbation
Timepoint [3] 441143 0
From end of exacerbation until next exacerbation, to a maximum of 12 months post-exacerbation
Secondary outcome [4] 441144 0
Adverse events (e.g. nausea, vomiting etc during 2 week intervention/treatment period)
Timepoint [4] 441144 0
From start of intervention for 2 week period
Secondary outcome [5] 441145 0
Biomarkers (gene expression markers of blood)
Timepoint [5] 441145 0
Baseline (stable state) compared to start of exacerbation
Secondary outcome [6] 441734 0
Spirometry (FVC % predicted)
Timepoint [6] 441734 0
Change in values at 4 weeks from commencement of intervention
Secondary outcome [7] 441735 0
Airway microbia (airway specimens e.g. sputum, bronchoscopic lavage, tracheal aspirate)
Timepoint [7] 441735 0
Baseline (stable state) compared to start of exacerbation

Eligibility
Key inclusion criteria
1. Participants aged 2 to 18 years who have chest computed tomography (CT) proven bronchiectasis
2. Participants have had at least 2 acute respiratory exacerbations in the last 18 months requiring antibiotics OR one hospitalisation for treatment of a respiratory exacerbation
3. Able to be contacted for the 12 month follow up period.
Minimum age
2 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Cystic fibrosis
2. Contraindication to erdosteine use (e.g. liver dysfunction, hypersensitivity, renal failure, deficiency of the cystathionine-synthetase enzyme, phenylketonuria, active peptic ulcer)
3. Pregnant, pregnancy planned (in next 12 months)
4. Participation in another concurrent randomised controlled trial
5. Taking tacrolimus, sirolimus, everolimus and cyclosporin
6. Past (treatment within the last 6 months) or current infection with non-tuberculous mycobacteria.
7. Diagnosed with tuberculosis infection and still considered contagious

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3 / Phase 4
Type of endpoint/s
Statistical methods / analysis
Recruitment for this feasibility study will be as many as possible in 6 month recruitment window to a maximum of 30 participants.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
6/01/2025
Actual
Date of last participant enrolment
Anticipated
17/07/2025
Actual
Date of last data collection
Anticipated
14/08/2026
Actual
Sample size
Target
30
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 27286 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 43373 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 317719 0
Government body
Name [1] 317719 0
Medical Research Future Fund/National Health and Medical Research Council
Country [1] 317719 0
Australia
Funding source category [2] 317824 0
Charities/Societies/Foundations
Name [2] 317824 0
Queensland Children's Hospital Foundation
Country [2] 317824 0
Australia
Funding source category [3] 317825 0
Hospital
Name [3] 317825 0
Queensland Children's Hospital
Country [3] 317825 0
Australia
Primary sponsor type
University
Name
Queensland University of Technology
Address
Country
Australia
Secondary sponsor category [1] 320037 0
None
Name [1] 320037 0
Address [1] 320037 0
Country [1] 320037 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 316410 0
Children’s Health Queensland Hospital and Health Service Human Research Ethics Committee
Ethics committee address [1] 316410 0
Ethics committee country [1] 316410 0
Australia
Date submitted for ethics approval [1] 316410 0
23/10/2024
Approval date [1] 316410 0
Ethics approval number [1] 316410 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 137802 0
A/Prof Julie Marchant
Address 137802 0
Queensland Children's Hospital, Stanley St, South Brisbane, Qld, 4101
Country 137802 0
Australia
Phone 137802 0
+61 7 3069 7350
Fax 137802 0
Email 137802 0
[email protected]
Contact person for public queries
Name 137803 0
Julie Marchant
Address 137803 0
Queensland Children's Hospital, Stanley St, South Brisbane, Qld, 4101
Country 137803 0
Australia
Phone 137803 0
+61 7 3068 1111
Fax 137803 0
Email 137803 0
[email protected]
Contact person for scientific queries
Name 137804 0
Julie Marchant
Address 137804 0
Queensland Children's Hospital, Stanley St, South Brisbane, Qld, 4101
Country 137804 0
Australia
Phone 137804 0
+61 7 3068 1111
Fax 137804 0
Email 137804 0
[email protected]

Data sharing statement
Will there be any conditions when requesting access to individual participant data?
No
Children's data and thus not shared in accordance to ethical requirements in our institution

Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Children's data and thus not shared in accordance to ethical requirements in our institution


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.