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Trial registered on ANZCTR


Registration number
ACTRN12624000601538
Ethics application status
Approved
Date submitted
2/04/2024
Date registered
9/05/2024
Date last updated
9/05/2024
Date data sharing statement initially provided
9/05/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Tolerability and Safety of Inhaled Colistimethate Sodium (CMS) Administered Once Daily Compared to Twice Daily Dosing in Adult and Adolescent Subjects with Cystic Fibrosis and Chronic Pseudomonas Aeruginosa Lung Infection (COPILOT)
Scientific title
Tolerability and Safety of Inhaled Colistimethate Sodium (CMS) Administered Once Daily Compared to Twice Daily Dosing in Adult and Adolescent Subjects with Cystic Fibrosis and Chronic Pseudomonas Aeruginosa Lung Infection (COPILOT)
Secondary ID [1] 311936 0
IND- 139943
Secondary ID [2] 311937 0
EudraCT number: 2022-000476-18
Universal Trial Number (UTN)
Trial acronym
Linked study record
EUCTR2022-000476-18-HU
This record only describes the Australian study

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
 333195 0
Pseudomonas aeruginosa infection 333528 0
Condition category
Condition code
Respiratory 329883 329883 0 0
Other respiratory disorders / diseases
Infection 330209 330209 0 0
Other infectious diseases
Human Genetics and Inherited Disorders 330210 330210 0 0
Cystic fibrosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Colistimethate Sodium (CMS) 4 MIU: vials of CMS 4 MIU (320 mg CMS) are reconstituted with 8 mL 0,9% saline solution, once daily, administered by inhalation via a nebuliser for 28 days
The protocol will recruit in 2 steps, which are:
Step A: enrollment of adults (ages 18 years and older)
Step B: enrollment of children, adolescents, and adults (ages 12 years and older)
Intervention code [1] 328181 0
Treatment: Drugs
Comparator / control treatment
Colistimethate Sodium (CMS) 2 MIU: vials of CMS 2 MIU (160 mg CMS) are reconstituted with 4 mL 0,9% saline solution, twice daily, administered by inhalation via a nebuliser for 28 days
Control group
Dose comparison

Outcomes
Primary outcome [1] 337651 0
Number of observed safety events during the 28-day treatment period in each treatment arm
Timepoint [1] 337651 0
Assessment will occur at day 28 for all safety events that have occurred from Randomisation to day 28
Secondary outcome [1] 432686 0
Number of observed safety events during the 42-day follow-up period in each treatment arm
Timepoint [1] 432686 0
Assessment will occur at day 42 for all safety events that have occurred from day 29 to day 42

Eligibility
Key inclusion criteria

1. Male and female subjects, ages 12 years and older:
a. Step A: ages 18 years and older (adults)
b. Step B: ages 12 years and older (children, adolescents, and adults)
2. Previous diagnosis of cystic fibrosis as confirmed by:
a. documented sweat chloride greater than or equal to 60 mEq/L by quantitative pilocarpine iontophoresis test prior to initiation of therapy with CFTR modulators (e.g., elexacaftor, tezacaftor, ivacaftor), if applicable; or
b. two well-characterized genetic mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene; and
c. symptoms characteristic of cystic fibrosis
3. Persistent airways infection with P. aeruginosa as evidenced by:
a. positive P. aeruginosa in a sputum/deep-throat cough swab culture (or bronchoalveolar lavage [BAL]) within 6 months prior to Screening; or
b. history of inhaled antibiotic treatment for suppression of P. aeruginosa
4. Stable treatment regimen of CF therapeutics for at least 2 months prior to randomization defined as:
a. regimen of inhaled antibacterials for either at least 2 months of continuous therapy or at least a total of 56 days of consecutive on-treatment periods in case of cyclical therapy (e.g., 28 days on treatment, followed by 28 days off treatment, followed by another 28 days on treatment) prior to randomization AND
b. no changes in either any current treatment regimen or initiation of treatment with hypertonic saline, dornase alfa, CFTR modulators or other CF specific therapeutics.
5. Ability to perform reproducible pulmonary function tests.
6. FEV1 at Screening of greater than or equal to 30% of predicted values
7. Arterial oxygen saturation (SpO2) greater than or equal to 90% on room air at Screening
8. Clinically stable in the opinion of the Investigator
9. Female subjects of childbearing potential must have a negative pregnancy test at the Screening Visit and must use a highly acceptable method of contraception after the first dose of trial drug and for 28 days after the last dose of trial drug, as defined in the protocol. To be considered “not of childbearing potential”:
a. female subjects must be postmenopausal for at least 1 year as confirmed by an elevated follicle-stimulating hormone (FSH) level (greater than or equal to 30 mIU/mL) at Screening and 1 year of amenorrhea, or have been irreversibly surgically sterilized by hysterectomy, oophorectomy, or bilateral tubal ligation for at least 3 months prior to the first dose of trial drug; or
b. female subjects must be before their first menstrual cycle (i.e., menarche)
10. Male subjects whose female partners are of childbearing potential (definition as above) must agree to use an acceptable method of birth control for the duration of trial treatment and for 28 days after the last dose of trial drug.
11. Signed written informed consent.
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Current need for daily continuous O2 or requirement for > 2 liters/min at night
2. History of any investigational drug/device use within 28 days of screening or within 6 half-lives of investigational drug (whichever is longer)
3. History of lung or liver transplantation
4. History or current diagnosis of myasthenia gravis or porphyria
5. Abnormal renal or hepatic function measured in serum chemistry at Screening (AST or ALT > 3x upper limit of normal (ULN); Creatinine > 2x ULN)
6. Positive pregnancy test at Screening
7. Are pregnant, plan to become pregnant during this trial, are nursing mothers or are unwilling to use an acceptable method of contraception for the duration of the trial.
8. Have any serious or active medical or psychiatric illness, which in the opinion of the Investigator, would interfere with subjects’ treatment, assessment, or compliance with the protocol.
9. Have a history or suspicion of unreliability, poor cooperation, or non-compliance with medical treatment.
10. Have previously been randomized and treated in this trial.
11. Have any other condition that, in the opinion of the Investigator, would prohibit the subject from participating in the trial

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)
central randomisation by computer
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Simple randomisation using a randomisation table created by computer software
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 3
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
31/05/2024
Actual
Date of last participant enrolment
Anticipated
31/08/2024
Actual
Date of last data collection
Anticipated
30/11/2024
Actual
Sample size
Target
38
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA,VIC

Funding & Sponsors
Funding source category [1] 316046 0
Commercial sector/Industry
Name [1] 316046 0
Enbiotix Inc
Country [1] 316046 0
United States of America
Funding source category [2] 316047 0
Commercial sector/Industry
Name [2] 316047 0
Spexis Australia Pty Ltd
Country [2] 316047 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Enbiotix Inc
Address
Country
United States of America
Secondary sponsor category [1] 318208 0
Commercial sector/Industry
Name [1] 318208 0
Spexis Australia Pty Ltd
Address [1] 318208 0
Country [1] 318208 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 314868 0
Children’s Health Queensland Hospital and Health Service Human Research Ethics Committee
Ethics committee address [1] 314868 0
http://www.childrens.health.qld.gov.au/research/human-research-ethics-committee
Ethics committee country [1] 314868 0
Australia
Date submitted for ethics approval [1] 314868 0
Approval date [1] 314868 0
20/02/2024
Ethics approval number [1] 314868 0

Summary
Brief summary
To assess the Tolerability and Safety of inhaled CMS and the profile while, comparing once daily (QD) with twice daily (BID) Administration for 28 days, The study will be measured by safety and respiratory tolerability events.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 132990 0
Prof Claire Wainwright
Address 132990 0
Queensland Chlidren's Hospital, 501 Stanley St, South Brisbane QLD 4101
Country 132990 0
Australia
Phone 132990 0
+617 30681111
Fax 132990 0
Email 132990 0
[email protected]
Contact person for public queries
Name 132991 0
Rachelle Kirk-Burnnand
Address 132991 0
Spexis Australia, C/- RSM Australia Level 21, 55 Collins St, Melbourne VIC 3000
Country 132991 0
Australia
Phone 132991 0
+61439615368
Fax 132991 0
Email 132991 0
[email protected]
Contact person for scientific queries
Name 132992 0
Rachelle Kirk-Burnnand
Address 132992 0
Spexis Australia, C/- RSM Australia Level 21, 55 Collins St, Melbourne VIC 3000
Country 132992 0
Australia
Phone 132992 0
+61439615368
Fax 132992 0
Email 132992 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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.