E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10,000 children. In cystic fibrosis chloride transport across the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli1901 corrects the abnormal transport of chloride ans water in the lungs thereby reducing the formation of mucus plugs and improving clearance. |
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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 |
The study objective is to determine safety and tolerability of a multiple-dose regimens of aerosolized Moli1901 in adolescents (12 years of age or older) and adults with cystic fibrosis and mild to moderate lung disease. |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
16 years of age or older (Phase I of Study) resp. 12 years of age or older (Phase II of Study). Have a confirmed diagnosis of cystic fibrosis [positive sweat chloride value > 60 mEq/l (by quantitative pilocarpine iontophoresis) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype. Have Screening FEV1 greater than 60% of predicted. Have oxygen saturation level measured by pulse oximetry (Sp02) > 90 % on room air.
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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. Are on a tobramycin “on-off” treatment Receive parenteral antibiotics via “on-off” treatment. Have any clinically significant liver, renal, cardiac, neurologic, or hematologic disease. Have Burkholderia cepacia or allergic bronchopulmonary aspergillosis. Have smoked more than 3 cigarettes per day within the past 12 months. Have a history of alcohol (> 40g/d) or drug abuse. Have participated in an investigational drug study within 4 weeks of screening. Show bronchial hyperresponsiveness. Do not have stable lung function, i.e. have more than 10% FEV1 variation 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.
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E.5 End points |
E.5.1 | Primary end point(s) |
•Safety and tolerability will be assessed by evaluating general physical condition, adverse events, changes in pulmonary function test results, pulse oximetry, vital signs, physical examinations, ECGs (electrocardiograms), and clinical laboratory tests. Pulmonary function evaluation will focus on pre-treatment and post-treatment pulmonary function test results as demonstrated by FEV1 (Forced Expiratory Volume in One Second), FVC (Forced Vital Capacity), and FEF25-75 (Forced Expiratory Flow 25–75% of FVC). •Exercise tolerance will be assessed based on evidence of either improvement or worsening. •Quality of life will be assessed using a questionnaire to be filled out by the study subjects at Study Start and end of treatment phase and at Study End
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Information not present in EudraCT |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Information not present in EudraCT |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Information not present in EudraCT |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
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 | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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
| Information not present in EudraCT |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 0 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 12 |