E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess efficacy and safety of Moli1901 used in CF patients |
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E.2.2 | Secondary objectives of the trial |
·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) |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal 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 |
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E.4 | Principal 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 |
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E.5 End points |
E.5.1 | 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 |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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End of the trial will be when the last patient has completed the full study period (12 weeks double blind period+12 weeks optional open label treatment period + 4 weeks follow up period without treatment) and has had the last study-visit (last visit of the follow up period) |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 6 |