None listed
Conditions
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.
Interventions
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)
Sponsors
Spexis Australia Pty Ltd
Study design
Allocation
Randomised controlled trial
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
12 Years to No maximum
Healthy volunteers
No
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.
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
Outcome results
None listed