Chronic pulmonary infections due to Pseudomonas aeruginosa in patients with cystic fibrosis MedDRA version: 20.0 Level: PT Classification code 10011763 Term: Cystic fibrosis lung System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
EnBiotix, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male and female subjects, ages 12 years and older: a. Step A: ages 18 years and older b. Step B: ages 12 years and older 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 day 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. FEV1 at screening of =30% of predicted values 6. Arterial oxygen saturation (SpO2) = 90% on room air at screening Are the trial subjects under 18? yes Number of subjects for this age range: 18 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Hx of daily continuous O2 or requirement for > 2 liters/min at night 2. Hx of any investigational drug/device use within 28 days of screening or within 6 half-lives of investigational drug (whichever is longer) 3. Hx of lung or liver transplantation 4. Hx 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Tolerability and Safety of Open Label inhaled colistimethate sodium, comparing once daily (QD) with twice daily (BID) Administration for 28 days, measured by safety and respiratory tolerability events.;Secondary Objective: Additional assessments of pulmonary function and clinical events as measured by • Pulmonary function (ppFEV1) • Pulmonary exacerbations (PEx), severity of PEx, hospitalizations • Additional antibacterials taken;Primary end point(s): Number of observed safety events during the 28-day treatment period in each treatment arm;Timepoint(s) of evaluation of this end point: 28 days | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Number of observed safety events during the 42-day follow-up period in each treatment arm 2. Number of observed safety events during the 169 days total follow-up period in each treatment arm 3. Absolute change from baseline (Day 1) to Day 14 and Day 28 in ppFEV1 in each treatment arm 4. Change in mean absolute ppFEV1 from baseline (after SABA) to post dose values during serial spirometry at Day 1 and Day 28 in each treatment arm 5. Number of newly diagnosed total and severe PEx from baseline (Day 1) to Day 28, to Day 42 and to 169 in each treatment arm 6. Additional antibacterials taken 7. Number of hospitalizations from baseline (Day 1) to Day 28, to Day 42 and to Day 169 in each treatment arm ;Timepoint(s) of evaluation of this end point: 1. 42 days 2. 169 days 3. Day 14 and Day 28 4. Day 1 and Day 28 5. Day 28, Day 42 and Day 169 6. Day 28 7. Day 28, Day 42 and Day 169 | — |
Countries
Australia, Bulgaria, Greece, Hungary, Poland, Slovakia, United Kingdom
Contacts
Public ContactRegulatory Affairs
Accelsiors CRO and Consultancy Services Ltd
Outcome results
None listed