E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis (CF) is the most common life–limiting autosomal recessive disease among people of European heritage. The condition is a result of a mutation in the cystic fibrosis transmembrane conductance regulator (cftr) gene on chromosome seven, which encodes a chloride channel. In the lung defective channel activity leads to thick, viscous secretion and impaired mucociliary clearance. This causes trapping of mucus, colonization with bacteria and fungi, and a persistent inflammatory response. |
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E.1.1.1 | Medical condition in easily understood language |
In CF, the mucus is thicker and thinner than normally. It is difficult to cough up and it affects the breathing. Some bacteria benefit particularly well in the mucus and causes respiratory infections. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10074549 |
E.1.2 | Term | Cystic fibrosis respiratory infection suppression |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 study the pharmacokinetic of posaconazole in exhaled breath. |
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E.2.2 | Secondary objectives of the trial |
To assess how the concentration of posaconazol in exhaled breath relates to that in serum and saliva.
To assess if there is a correlation between concentration of posaconazol in exhaled breath and pulmonary changes.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Confirmed diagnosis of cystic fibrosis
2. ≥ 18 and < 65 years of age
3. Males and females
4. Clinically stable disease (but different pulmonary status)
5. Willing to participate in the study
6. Willing to use contraceptives
7. Signed informed consent
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E.4 | Principal exclusion criteria |
1. Previous adverse event on posaconazole
2. Ongoing treatment with posaconazol.
3. Advanced liver disease
4. Prolonged QTc-interval
5. Pregnancy, breastfeeding
6. Ongoing upper or lower respiratory infection requiring antibiotic treatment
7. Ongoing treatment with compounds contraindicated according to FASS
8. Ongoing or previous participation in a clinical pharmaceutical study within 28-days before screening.
9. Investigator assesses that it is not an appropriate study for the patient
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E.5 End points |
E.5.1 | Primary end point(s) |
Concentration of posaconazol in exhaled breath at different time points. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
0.5, 1, 1.5, 2, 3, 4, 8, 11, 24, 32, and 48 h after dosing. |
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E.5.2 | Secondary end point(s) |
Concentration of of posaconazol in serum and saliva.
Pulmonary changes measured by pulmonary function (FEV1) and pulmonary structural changes measured by existing HRCT.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
0.5, 1, 1.5, 2, 3, 4, 8, 11, 24, 32, and 48 h after dosing. |
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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 | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
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 | No |
E.8.7 | Trial has a data monitoring committee | No |
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 | |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | |