Cystic Fibrosis
Conditions
Interventions
Product Name: Moli1901
Product Code: Moli1901
Pharmaceutical Form: Nebuliser solution
CAS Number: 1391-36-2
Current Sponsor code: Moli1901
Other descriptive name: Duramycin, 2622U90
Concentration unit
Sponsors
AOP Orphan Pharmaceuticals AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Male or female subjects of 12 years of age or older -Body mass index equal or above -2 SDS (Standard Deviation Score-According to CDC standards) -Have a confirmed diagnosis of cystic fibrosis -Have Screening FEV1 between 55% and 85%. -Have oxygen saturation level measured by pulse oximetry (SpO2) > 90 % on room air -Willing and able to give informed consent -Willing and able to undergo procedures required by this -Stable FEV1 60% or greater of predicted, measured during the run in period within the range of ±5 % between highest and lowest value. -No pulmonary exacerbation during the screening and run in period Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -Have a pulmonary disease such as pneumonia, tuberculosis, or lung cancer -Have had an acute upper respiratory tract infection within 2 weeks of screening -Have had an acute lower respiratory tract infection (requiring antibiotics or hospitalization) within 4 weeks of screening -Have had an exacerbation within 4 weeks of screening -Have had any changes from routine maintenance therapy within 4 weeks of screening -Have any scheduled changes to inhaled antibiotics regimen during the course of the study -Are on, or are planned to be on, a tobramycin “on-off” treatment regimen -Have completed tobramycin “on-off” treatment within less than 6 weeks prior to patient enrollment -Receive parenteral antibiotics via “on-off” treatment regimen -Have completed therapy with parenteral antibiotics in “on-off” treatment regimen within less than 6 weeks prior to patient enrollment -Are on, or are planned to be on a dornase alfa "on-off" tretment regimen -Have completed dornase alfa "on-off" treatment within less than 6 weeks prior to patient enrollment -Have any clinically significant liver, renal, cardiac, neurological, or hematologic disease -Have Burkholderia cepacia or allergic bronchopulmonary aspergillosis. -Have poorly controlled diabetes mellitus -Have smoked more than 3 cigarettes per day within the past 12 months. -Have a history of alcohol (> 40g/day) or drug abuse -Have participated in an investigational drug study within 4 weeks of screening -Have participated in another pharmacological treatment study in which transepithelial chloride transport or ion composition of the epithelial lining fluid might have been influenced within 4 weeks of screening -Show bronchial hyperresponsiveness as known from previous visits e.g. the subject needs additional puffs of salbutamol (or other beta-2-agonists) more than twice daily. This also applies to subjects who have shown bronchoconstriction to previously administered inhalative therapies -Have shown any evidence for unstable lung function, e.g have had more than 10% FEV1 variation (difference between highest and lowest measured value) in the last 3 months before treatment starts -Are women of child bearing potential and refuse to use effective contraception or are pregnant or lactating -Are unwilling to perform appropriate safe contraception for at least 6 months after Study Drug administration
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess efficacy and safety of Moli1901 used in CF patients;Secondary Objective: ·Improvement in exercise capacity (as measured by Exercise tolerance test) ·Change in oxygen saturation (as measured with pulse oximetry) ·Change in Microbiologic Markers ·Changes of sputum rheology (in subgroups of patients, where feasible);Primary end point(s): Primary efficacy criterion: The relative change in the percentage of the predicted FEV1 (Forced Expiratory Volume in first second) value Safety endpoints: Complete physical examination Lung auscultation Vital signs ECG Spirometry (FEV1, FVC, and FEF25-75) Clinical laboratory tests (chemistry and hematology) Urinalysis Adverse events Concomitant medications Change in microbiologic markers | — |
Countries
Czech Republic, Hungary, Slovakia
Outcome results
None listed