Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12618001831279
Ethics application status
Approved
Date submitted
9/02/2018
Date registered
9/11/2018
Date last updated
13/07/2025
Date data sharing statement initially provided
9/11/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
Finding the Optimal Regimen for Mycobacteroides abscessus Treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease: a multi-centre, randomised, multi-arm, adaptive platform trial.
Scientific title
Finding the Optimal Regimen for Mycobacterium abscessus Treatment (FORMaT)
Secondary ID [1] 293945 0
ClinicalTrials.gov Identifier NCT04310930
Secondary ID [2] 314893 0
The UK's Clinical Study Registry, ISRCTN67303903
Universal Trial Number (UTN)
U1111-1209-0672
Trial acronym
FORMaT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Mycobacterium abscessus pulmonary disease 306443 0
Mycobacterium abscessus 315340 0
Condition category
Condition code
Respiratory 305529 305529 0 0
Other respiratory disorders / diseases
Human Genetics and Inherited Disorders 308844 308844 0 0
Cystic fibrosis
Infection 308845 308845 0 0
Other infectious diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This study is a standing platform trial to evaluate the efficacy of antibiotic therapy combinations in clearing Mycobacterium abscessus pulmonary disease (MABS-PD). The study will provide a pipeline for new therapies to be evaluated for treating MABS-PD in all age groups.

There are two different phases of treatment; intensive therapy followed by consolidation therapy. Intervention cohort participants will be randomised to 1 of 3 treatment arms for 6 weeks of intensive therapy. After the first 6 weeks of intensive therapy participants who are culture positive for MABS are randomised to either prolonged intensive therapy (additional 6 weeks of intensive therapy) then consolidation therapy, or to commence consolidation therapy. If, at week 6 participants are MABS culture-negative they will commence consolidation therapy. Participants commencing consolidation therapy are randomised to one of two treatment arms for 50 weeks. There are 12 possible paths through the trial and they are detailed below:
1. Intensive Arm A for 6 weeks, Consolidation arm b for 46 weeks,
2. Intensive Arm A for 12 weeks, Consolidation arm b for 46 weeks,
3. Intensive Arm A for 6 weeks, “Consolidation: arm a” for 46 weeks,
4. Intensive Arm A for 12 weeks, “Consolidation: arm a” for 46 weeks,
5. Intensive Arm B for 6 weeks, Consolidation arm b for 46 weeks,
6. Intensive Arm B for 12 weeks, Consolidation arm b for 46 weeks,
7. Intensive Arm B for 6 weeks, “Consolidation: arm a” for 46 weeks,
8. Intensive Arm B for 12 weeks, “Consolidation: arm a” for 46 weeks,
9. Intensive Arm C for 6 weeks, Consolidation arm b for 46 weeks,
10. Intensive Arm C for 12 weeks, Consolidation arm b for 46 weeks,
11. Intensive Arm C for 6 weeks, “Consolidation: arm a” for 46 weeks,
12. Intensive Arm C for 12 weeks, “Consolidation: arm a” for 46 weeks,
Where Intensive Arm A is IV amikacin, and; IV tigecycline, and; IV imipenem/cilastatin or IV cefoxitin, and; Oral azithromycin or oral clarithromycin, and; Oral clofazimine.
Intensive Arm B is Inhaled amikacin, and; IV tigecycline, and; IV imipenem/cilastatin or IV cefoxitin, and; Oral azithromycin or oral clarithromycin, and; Oral clofazimine.
Intensive Arm C is IV amikacin, and; IV tigecycline, and; IV imipenem/cilastatin or IV cefoxitin, and; Oral azithromycin or oral clarithromycin.
Consolidation arm a is Oral clofazimine, and; Oral azithromycin or oral clarithromycin, and; In combination with one to three of the following oral antibiotics: Oral linezolid, Oral trimethoprim / sulfamethoxazole, Oral bedaquiline, Oral rifabutin, Oral doxycycline, Oral moxifloxacin.
Consolidation arm b is Inhaled amikacin, and; Oral clofazimine, and; Oral azithromycin or oral clarithromycin, and; In combination with one to three of the following oral antibiotics: Oral linezolid, Oral trimethoprim / sulfamethoxazole, Oral bedaquiline, Oral rifabutin, Oral doxycycline, Oral moxifloxacin.
Dosing recommendations for each drug is outlined in the Drug dosing Regimen Tables contained in the relevant sections of the FORMaT Intervention Program Appendix A1 and Appendix A2 modules.

This standing platform trial design enables assessment of short intensive therapy, prolonged intensive therapy, and consolidation therapy components individually as well as the overall combination of intensive therapy and consolidation therapy. After 100 patients have completed short intensive therapy an interim analysis will be conducted, and Bayesian adaptive randomisation (BAR) will be implemented. New interventions may be added or stopped in the future due to lack of benefit at interim analyses.

Those participants that have a positive respiratory specimen but do not meet all three ATS criteria or those who are MABS-PD positive but are not commencing drug therapy can be recruited into the observation cohort where they will undergo all the same monitoring and outcomes collection but will not receive treatment. If they meet ATS criteria at a later stage during the trial they may be randomised into the active treatment phases (intensive and consolidation) of the trial.

The FORMaT trial has several substudies that are conducted alongside the main trial. These substudies are registered on a separate trial registration.
Intervention code [1] 300217 0
Treatment: Drugs
Comparator / control treatment
The active comparator group are those intervention program participants who are treated according to current therapy guidelines (Intensive arm A). Active comparator treatment for the intensive phase is (based on current guidelines): IV amikacin + IV tigecycline + IV imipenem or IV cefoxitin (guided by local susceptibility testing and patient specific hypersensitivity) + oral azithromycin or oral clarithromycin AND clofazimine..
See attachment 1 for the drug dosing regimens for the intensive treatment phase for both paediatrics and adults
Control group
Active

Outcomes
Primary outcome [1] 304705 0
The primary outcome of the Intervention Program is microbiological clearance of MABS with good tolerance of the interventions. Definition of MABS clearance at final outcome: Negative MABS cultures from four consecutive sputum samples with one of those sputum specimens collected four weeks after the completion of consolidation therapy, or a MABS negative Bronchoalveolar Lavage (BAL) collected four weeks after completion of consolidation. Definition of tolerance: Tolerance is based on the Common Terminology Criteria for Adverse Events (CTCAE version 5.0). Only adverse events that are attributed as either “possibly-“, “probably-“, or “definitely-“ related to study drug will be assessed in the determination of tolerance. “Good” tolerance is defined as no adverse events occurring or only adverse events coded as CTCAE grades 1 and 2. “Poor” tolerance is defined as any adverse events attributed as possibly-, probably-, or definitely-related to study drug coded as CTCAE grades 3, 4, or 5. Nested Study A1.1 Type of Short Intensive Therapy - MABS clearance from respiratory sample(s) with tolerance. To compare the microbiological clearance of MABS from respiratory samples collected at 4 weeks with good tolerability assessed at the end of short intensive therapy between the use of Inhaled Amikacin (Arm B) and the use of Intravenous Amikacin (Arm A) given during intensive phase. Definition of MABS clearance is 3 MABS negative sputum samples or ONE MABS negative Bronchoalveolar Lavage (BAL) at end of Short Intensive Therapy. “Good” tolerance is defined as no adverse events occurring or only adverse events that are attributed as either “possibly-“, “probably-“, or “definitely-“ related to study drug coded as CTCAE grades 1 and 2. Nested Study A1.2 - Duration of intensive therapy for patients completing short intensive treatment with ongoing positive MABS cultures collected at 4 weeks and randomised to either a further 6 weeks intensive therapy or immediate consolidation. To compare the microbiological clearance from samples collected at 10 weeks with good tolerability between those who are allocated to prolonged intensive therapy and those allocated to immediate consolidation following short intensive therapy. MABS clearance, assessed at the end of prolonged intensive therapy (for those allocated to prolonged intensive) or at 12 weeks (for those allocated to immediate consolidation) will be defined as negative MABS cultures from all 3 sputum samples or from one BAL sample collected at 10 weeks. “Good” tolerance is defined as no adverse events occurring or only adverse events that are attributed as either “possibly-“, “probably-“, or “definitely-“ related to study drug coded as CTCAE grades 1 and 2. Nested Study 1.3 Consolidation Therapy – The use of oral therapy only or oral therapy and inhaled amikacin for consolidation therapy. Description: To compare the microbiological clearance with good tolerability of MABS between those allocated to consolidation therapy with oral treatment and those allocated to consolidation therapy with oral treatment and additional Inhaled Amikacin at Final Outcome. Definition of MABS clearance at final outcome: Negative MABS cultures from four consecutive sputum samples with one of those sputum specimens collected four weeks after the completion of consolidation therapy, or a MABS negative Bronchoalveolar Lavage (BAL) collected four weeks after completion of consolidation. “Good” tolerance is defined as no adverse events occurring or only adverse events that are attributed as either “possibly-“, “probably-“, or “definitely-“ related to study drug coded as CTCAE grades 1 and 2.
Timepoint [1] 304705 0
Time frame for the Intervention Program: Screening (Day 0) to End of treatment plus four weeks off-treatment (Final Outcome Visit (Week 56 for those allocated to Immediate Consolidation or Week 62 for those allocated to Prolonged Intensive). Time frame for Nested study A1.1: Screening (Day 0) to the End of Short Intensive Therapy (Week 6). Time Frame for Nested Study A1.2: Screening (Day 0) to EITHER the End of Prolonged Intensive Therapy (for those allocated to Prolonged Intensive) OR Week 12 Visit (for those allocated to immediate consolidation therapy). Time frame for Nested Study A1.3: Start of Consolidation Therapy (Date of Randomisation to Consolidation Therapy) to End of Treatment plus 4 weeks off treatment (Final Outcome Visit - Week 56 for those randomised to Immediate Consolidation or Week 62 for those in Prolonged
Secondary outcome [1] 342866 0
Probability of MABS clearance at Final Outcome irrespective of toxicity according to participant’s treatment pathway. Definition of MABS clearance at final outcome: Negative MABS cultures from four consecutive sputum samples with one of those sputum specimens collected four weeks after the completion of consolidation therapy, or a MABS negative Bronchoalveolar Lavage (BAL) collected four weeks after completion of consolidation.
Timepoint [1] 342866 0
Screening (Day 0); at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [2] 353072 0
Safety of treatment combinations based on the reporting of adverse events that are attributed as either “possibly-“, “probably-“, or “definitely-“ related to study drugs.
Timepoint [2] 353072 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [3] 353081 0
Safety of treatments based on changes to microbiological resistance of the MABS bacteria during the study treatment period.
Timepoint [3] 353081 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [4] 353220 0
Change in FEV1 z-score at Final Outcome compared with Screening in participants who do and do not clear MABS at Final Outcome.
Timepoint [4] 353220 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [5] 377245 0
Phenotype of the structural abnormalities of chest CTs and changes in chest CT scores between Screening and Final Outcome between participants who clear or do not clear MABS at Final Outcome.
Timepoint [5] 377245 0
Screening (Day 0); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [6] 428915 0
Predictive value of structural abnormalities on Screening CT scans for sputum conversion and for progression of structural changes in relation to treatment.
Timepoint [6] 428915 0
Screening (Day 0); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [7] 428916 0
Change in 6-minute walk distance (6MWD) for adult participants from Screening to Final Outcome according to treatment pathway and in participants who do and do not clear MABS at Final Outcome.
Timepoint [7] 428916 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [8] 428917 0
Change in HRQoL (measured using the CFQ-R, EQ-5D, SGRQ, SF-36, PedsQL and CHU-9D) for participants from Screening to Final Outcome according to treatment pathway and in participants who do and do not clear MABS at Final Outcome. CFQ-R is the Cystic Fibrosis Questionnaire Revised and will be completed by all CF participants, EQ-5D is the EuroQol 5 Dimensions and will be completed by all participants; SGRQ is the St George's Respiratory Questionnaire and will be completed by all non-CF participants who are 18 years and older; the SF-36 is the Short-Form 36 questionnaire and will be completed by all participants 16 years and older; the PedsQL is the Pediatric Quality of Life Inventory questionnaire and will be completed by participants 2 years old to 16 years old; CHU-9D is the Child Health Utility 9D and will be completed by all children aged 7 to 17 years of age.
Timepoint [8] 428917 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [9] 428918 0
Cost effectiveness of the treatment combinations across intensive and consolidation phases of the trial. Cost effectiveness of the treatments will be assessed using the unvalidated "Costs Questionnaire" and where applicable and available, the linked administrative healthcare utilisation data e.g. in Australian participants.
Timepoint [9] 428918 0
Screening (Day 0); At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).
Secondary outcome [10] 428919 0
Causes for early withdrawal from MABS-PD treatment due to reasons other than poor tolerance as defined in the primary objectives.
Timepoint [10] 428919 0
At End of Short Intensive Therapy (Week 6); At End of Prolonged Intensive Therapy OR at Week 12 visit; at End of Treatment plus 4 weeks off treatment (Final Outcome Visit - either Week 56 or Week 62).

Eligibility
Key inclusion criteria
Eligibility criteria
Entry into the study can occur at two different levels:
1. Participants of any age meeting the ATS criteria1 for MABS-PD and not receiving treatment are eligible to enrol in the Intervention program, and;
2. Participants of any age from their first MABS isolate who do not require treatment or who have elected not to receive MABS therapy can enrol in the Observational cohort. Participants enrolled into the Observational cohort who proceed to meet the ATS criteria for MABS-PD can transition to the Intervention Program at any time.

Intervention program participants are randomised to receive MABS-PD therapy combinations.

Intervention program inclusion criteria
Participants with positive MABS cultures, including those with mixed NTM infections (slow growers + MABS) or with recurrence of MABS, are eligible for the Intervention program if the following inclusion criteria are met.
1. ATS diagnostic criteria for MABS-PD are fulfilled (clinical, radiographic and microbiological).
a. Clinical: Pulmonary symptoms and exclusion of other causes.
b. Radiographic: Nodular or cavitary opacities on chest radiograph or a chest high-resolution computed tomography (HRCT) scan showing multifocal bronchiectasis with multiple small nodules.
c. Microbiological: MABS positive culture results from at least two separate expectorated sputum samples.
or
Positive culture results from one bronchial wash or bronchoalveolar lavage (BAL).
or
One lung biopsy with histology consistent with NTM and the biopsy or sputum culture positive for MABS.
Screening samples must be collected within the timeframes stated in the relevant appendix.
2. Participants of any age and sex.
3. Not received treatment for MABS-PD in the preceding 12-months, or as specified in the relevant appendix (this includes drugs prescribed for the treatment of other mycobacteria and/or other indications that may have activity against MABS).
4. Informed consent signed by participant or parent/legal guardian (if participant is aged
<18-years).
5. Ability to comply with study visits, therapies and study procedures as judged by the site investigator.

Observational cohort inclusion criteria (Appendix B)
Participants meeting the following criteria are eligible to be recruited into the Observational Cohort:
1. Participants of any age with at least one positive respiratory culture for MABS.
2. Informed consent signed by participant or parent/legal guardian if participant is aged <18-years.
3. Ability to comply with study visits and study procedures as judged by the site investigator.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Intervention program exclusion criteria
1. Receiving current treatment for MABS (including drugs prescribed for other mycobacteria and/or other indications that may have activity against MABS, except for azithromycin as part of routine treatment for CF or chronic infection-related pulmonary disease).
2. QTc interval >500 milliseconds.
3. Pregnancy or breast feeding.
4. Known hypersensitivity to any of the therapies for which there are no alternative option(s).

Observational cohort exclusion criteria
Receiving current treatment for MABS (including drugs prescribed for other mycobacteria and/or other indications that may have activity against MABS, except for azithromycin as part of routine treatment for CF or chronic infection-related pulmonary disease).

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)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Minimisation is a dynamic randomisation approach used in clinical trials to balance allocation to treatment arms with respect to a number of important stratification factors.

There will be three stages of randomisation in the intervention program of this study, dictating the treatment the participant will receive:
Randomisation 1: The first randomisation will be at the start of the intensive phase, with all participants randomised between the different intensive therapy arms for a period of 6 weeks.
Randomisation 2: The second randomisation will ONLY be for participants who are still MABS positive at week six. Randomisation will occur at the end of week 6 and will allocate participants to either;
1- Continue intensive therapy which will be followed by consolidation
2- Immediately commence consolidation therapy.

Randomisation 3: This randomisation will allocate participants to the consolidation therapy arms either at week 6 or at week 12 depending on the most recent MABS clearance result, i.e. from week six or (if applicable) from week 12 following prolonged intensive therapy.
Week 6: This randomisation occurs at 6 weeks for patients who have cleared MABS at 6 weeks and for patients who remained MABS positive at 6 weeks and were randomised in randomisation 2 to start consolidation.
Week 12: Patients who are randomised to prolonged intensive therapy will be randomised between the consolidation arms at the end of week 12.

Intervention cohort participants will be randomsied according to the stratification criteria below:
1- Macrolide resistance*: *Any of these measurement methods are acceptable for defining macrolide resistance (in order of preference);
a. Inducible at 14 days or constitutive at 3 days
b. Erm(41) status: Functional or dysfunctional
c. MABS subspecies: M. abscessus & M. bolletii combined or M. massiliense
2. Age: <12 years, 12-30 years and >30 years of age
3. Sex
4. Location: Australia and New Zealand as one stratum, United Kingdom and Ireland as one stratum, Denmark, France and the Netherlands as another stratum and Canada as one stratum
5. Cystic Fibrosis Status
6. Mixed NTM infections at enrolment
7. MABS positive culture (at initial randomisation to intensive therapy and for randomisation 2 all participants will have a positive culture, so this factor will not be required. However, it will be required for randomisation 3)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
For participants enrolled in the intervention cohort a respiratory sample assessing MABS will be collected at the completion of 4 weeks of intensive therapy. Intervention cohort subjects will continue intensive therapy until results are available (10-14 days). Subjects with positive cultures for MABS at this point, will be randomised 1:1 to either continuing a further 6 weeks of intensive treatment before starting consolidation or starting consolidation immediately.
When randomised to the consolidation phase subjects will be randomised 1:1 to oral azithromycin, clofazimine and doxycycline or moxifloxacin + additional IA for 46 wks to complete a total 52 or 58 wks therapy.
Phase
Phase 2
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
2/12/2019
Actual
2/03/2020
Date of last participant enrolment
Anticipated
31/12/2028
Actual
Date of last data collection
Anticipated
31/12/2028
Actual
Sample size
Target
300
Accrual to date
101
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA,VIC
Recruitment hospital [1] 9967 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 9968 0
Greenslopes Private Hospital - Greenslopes
Recruitment hospital [3] 9969 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [4] 9971 0
Mater Adult Hospital - South Brisbane
Recruitment hospital [5] 9972 0
Gold Coast University Hospital - Southport
Recruitment hospital [6] 9973 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [7] 9974 0
John Hunter Hospital - New Lambton
Recruitment hospital [8] 9977 0
Sydney Children's Hospital - Randwick
Recruitment hospital [9] 9978 0
The Alfred - Prahran
Recruitment hospital [10] 9983 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment hospital [11] 9984 0
The Royal Adelaide Hospital - Adelaide
Recruitment hospital [12] 12231 0
Perth Children's Hospital - Nedlands
Recruitment hospital [13] 15331 0
Sunshine Coast University Hospital - Birtinya
Recruitment hospital [14] 25659 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [15] 25660 0
Royal Perth Hospital - Perth
Recruitment hospital [16] 25661 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [17] 25726 0
Royal Melbourne Hospital - City campus - Parkville
Recruitment hospital [18] 28204 0
Austin Health - Austin Hospital - Heidelberg
Recruitment postcode(s) [1] 18807 0
4032 - Chermside
Recruitment postcode(s) [2] 18808 0
4120 - Greenslopes
Recruitment postcode(s) [3] 18809 0
4102 - Woolloongabba
Recruitment postcode(s) [4] 18811 0
4215 - Southport
Recruitment postcode(s) [5] 18812 0
2050 - Camperdown
Recruitment postcode(s) [6] 18813 0
2305 - New Lambton
Recruitment postcode(s) [7] 18815 0
2031 - Randwick
Recruitment postcode(s) [8] 18816 0
3004 - Prahran
Recruitment postcode(s) [9] 18821 0
6009 - Nedlands
Recruitment postcode(s) [10] 18822 0
5000 - Adelaide
Recruitment postcode(s) [11] 24416 0
6009 - Broadway Nedlands
Recruitment postcode(s) [12] 28640 0
4575 - Birtinya
Recruitment postcode(s) [13] 41549 0
6000 - Perth
Recruitment postcode(s) [14] 41550 0
3050 - Parkville
Recruitment postcode(s) [15] 41680 0
3050 - Parkville
Recruitment postcode(s) [16] 44416 0
3084 - Heidelberg
Recruitment outside Australia
Country [1] 9563 0
United Kingdom
State/province [1] 9563 0
Nottingham
Country [2] 9564 0
United Kingdom
State/province [2] 9564 0
Birmingham
Country [3] 9567 0
Denmark
State/province [3] 9567 0
Copenhagen
Country [4] 25844 0
Singapore
State/province [4] 25844 0
Singapore
Country [5] 25845 0
Israel
State/province [5] 25845 0
Tel Aviv
Country [6] 25847 0
Taiwan, Province Of China
State/province [6] 25847 0
Taipei
Country [7] 25849 0
France
State/province [7] 25849 0
Country [8] 25888 0
Singapore
State/province [8] 25888 0
Singapore
Country [9] 25889 0
Israel
State/province [9] 25889 0
Beer-Sheva
Country [10] 25891 0
Taiwan, Province Of China
State/province [10] 25891 0
Taichung
Country [11] 25969 0
Israel
State/province [11] 25969 0
Haifa
Country [12] 27213 0
Israel
State/province [12] 27213 0
Jerusalem
Country [13] 27214 0
United Kingdom
State/province [13] 27214 0
Glasgow
Country [14] 27215 0
United Kingdom
State/province [14] 27215 0
Bristol
Country [15] 27216 0
United Kingdom
State/province [15] 27216 0
Liverpool
Country [16] 27217 0
United Kingdom
State/province [16] 27217 0
Edinburgh
Country [17] 27218 0
United Kingdom
State/province [17] 27218 0
Southampton
Country [18] 27219 0
United Kingdom
State/province [18] 27219 0
Manchester
Country [19] 27220 0
United Kingdom
State/province [19] 27220 0
Wales
Country [20] 27221 0
Israel
State/province [20] 27221 0
Petah-Tikva
Country [21] 27222 0
Israel
State/province [21] 27222 0
Ramat Gan
Country [22] 27223 0
Belgium
State/province [22] 27223 0
Leuven

Funding & Sponsors
Funding source category [1] 298573 0
Government body
Name [1] 298573 0
The Commonwealth of Australian represented by Department of Health
Country [1] 298573 0
Australia
Funding source category [2] 298629 0
Other
Name [2] 298629 0
Anonymous donor
Country [2] 298629 0
Australia
Funding source category [3] 304376 0
Charities/Societies/Foundations
Name [3] 304376 0
Cystic Fibrosis Foundation
Country [3] 304376 0
United States of America
Funding source category [4] 304377 0
University
Name [4] 304377 0
The Unviersity of Queensland
Country [4] 304377 0
Australia
Funding source category [5] 304378 0
Charities/Societies/Foundations
Name [5] 304378 0
Children's Hospital Foundation
Country [5] 304378 0
Australia
Primary sponsor type
University
Name
The University of Queensland
Address
Office of Sponsored Research
The University of Queensland
Cumbrae Stewart Building 72
Research Road, QLD 4072
Country
Australia
Secondary sponsor category [1] 300554 0
None
Name [1] 300554 0
Address [1] 300554 0
Country [1] 300554 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 299539 0
Children’s Health Queensland Human Research Ethics Committee
Ethics committee address [1] 299539 0
Ethics committee country [1] 299539 0
Australia
Date submitted for ethics approval [1] 299539 0
18/09/2019
Approval date [1] 299539 0
10/10/2019
Ethics approval number [1] 299539 0
Ethics committee name [2] 299574 0
Metro North Health Human Research Ethics Committee
Ethics committee address [2] 299574 0
Ethics committee country [2] 299574 0
Australia
Date submitted for ethics approval [2] 299574 0
22/08/2019
Approval date [2] 299574 0
26/08/2019
Ethics approval number [2] 299574 0
Ethics committee name [3] 318022 0
Region Hovedstaden Ethics Committee
Ethics committee address [3] 318022 0
Ethics committee country [3] 318022 0
Denmark
Date submitted for ethics approval [3] 318022 0
Approval date [3] 318022 0
02/01/2023
Ethics approval number [3] 318022 0
H-21048431
Ethics committee name [4] 318023 0
National Health Group Domain Specific Review Board
Ethics committee address [4] 318023 0
Ethics committee country [4] 318023 0
Singapore
Date submitted for ethics approval [4] 318023 0
Approval date [4] 318023 0
09/11/2023
Ethics approval number [4] 318023 0
2023/00432
Ethics committee name [5] 318024 0
London – Brighton & Sussex Research Ethics Committee
Ethics committee address [5] 318024 0
Ethics committee country [5] 318024 0
United Kingdom
Date submitted for ethics approval [5] 318024 0
Approval date [5] 318024 0
14/01/2025
Ethics approval number [5] 318024 0
24/LO/0838
Ethics committee name [6] 318025 0
Research Ethics Committee E – National Taiwan University Hospital
Ethics committee address [6] 318025 0
Ethics committee country [6] 318025 0
Taiwan, Province Of China
Date submitted for ethics approval [6] 318025 0
Approval date [6] 318025 0
27/03/2025
Ethics approval number [6] 318025 0
202502093MINE
Ethics committee name [7] 318026 0
Research Ethics Committee F – Kaohsiung Medical University Hospital
Ethics committee address [7] 318026 0
Ethics committee country [7] 318026 0
Taiwan, Province Of China
Date submitted for ethics approval [7] 318026 0
Approval date [7] 318026 0
11/04/2025
Ethics approval number [7] 318026 0
KMUHIRB-F(I)-20250111
Ethics committee name [8] 318027 0
Hadassah Ethics Committee
Ethics committee address [8] 318027 0
Ethics committee country [8] 318027 0
Israel
Date submitted for ethics approval [8] 318027 0
Approval date [8] 318027 0
21/01/2025
Ethics approval number [8] 318027 0
0262-24-HMO
Ethics committee name [9] 318028 0
Sheba Ethics Committee
Ethics committee address [9] 318028 0
Ethics committee country [9] 318028 0
Israel
Date submitted for ethics approval [9] 318028 0
Approval date [9] 318028 0
02/03/2025
Ethics approval number [9] 318028 0
1372-24-SMC
Ethics committee name [10] 318029 0
Rambam Ethics Committee
Ethics committee address [10] 318029 0
Ethics committee country [10] 318029 0
Israel
Date submitted for ethics approval [10] 318029 0
Approval date [10] 318029 0
12/05/2025
Ethics approval number [10] 318029 0
0307-24-RMB

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

Contacts
Principal investigator
Name 80778 0
Prof Claire Wainwright
Address 80778 0
Centre for Children's Health Research Level 7, 62 Graham Street Lady Cilento Children's Hospital Precinct South Brisbane QLD 4101
Country 80778 0
Australia
Phone 80778 0
+617 30681111
Fax 80778 0
+617 30697159
Email 80778 0
[email protected]
Contact person for public queries
Name 80779 0
Claire Wainwright
Address 80779 0
Centre for Children's Health Research Level 7, 62 Graham Street Lady Cilento Children's Hospital Precinct South Brisbane QLD 4101
Country 80779 0
Australia
Phone 80779 0
+617 30681111
Fax 80779 0
+617 30697159
Email 80779 0
[email protected]
Contact person for scientific queries
Name 80780 0
Claire Wainwright
Address 80780 0
Centre for Children's Health Research Level 7, 62 Graham Street Lady Cilento Children's Hospital Precinct South Brisbane QLD 4101
Country 80780 0
Australia
Phone 80780 0
+617 30681111
Fax 80780 0
+617 30697159
Email 80780 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
Yes
Will there be any conditions when requesting access to individual participant data?
Persons/groups eligible to request access:
• Data will be made available to recognised academic institutes and clinical teams upon written request with a proposal for data usage to Chief Investigator, Professor Claire Wainwright

Conditions for requesting access:
• -

What individual participant data might be shared?
• All de-identified raw data measured during the trial will be made available.

What types of analyses could be done with individual participant data?
• Any purpose.

When can requests for individual participant data be made (start and end dates)?
From:
Immediately following publication with no end date.

To:
-

Where can requests to access individual participant data be made, or data be obtained directly?
• Upon the approval for data sharing by Professor Claire Wainwright the requester will be required to sign a data agreement.

Are there extra considerations when requesting access to individual participant data?
No


What supporting documents are/will be available?



Results publications and other study-related documents

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

Documents added automatically
SourceTitleYear of PublicationDOI
EmbasePulmonary Mycobacterium abscessus complex in children with cystic fibrosis: A practical management guideline.2019https://dx.doi.org/10.1111/jpc.14427
N.B. These documents automatically identified may not have been verified by the study sponsor.