E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic fibrosis |
Cystic fibrosis |
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E.1.1.1 | Medical condition in easily understood language |
Cystic fibrosis |
Taaislijmziekte |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To investigate the pharmacokinetics of once daily inhalation of the recommended daily dose of tobramycin with the Akita® nebulizer, specifically targeted to the peripheral small airways, in patients with CF. |
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E.2.2 | Secondary objectives of the trial |
To compare data from primary objective to 1) pharmacokinetics of once daily tobramycin inhalation with the conventional and registered PARI-LC® Plus nebulizer and to 2) pharmacokinetic data from the literature about standard twice daily tobramycin inhalation with the PARI-LC® Plus. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
In order to be eligible to participate in this study, a subject must meet all of the following criteria:
• Age ≥ 18 years;
• FEV1 predicted ≥ 30%;
• Clinical diagnosis of CF and a positive sweat test or two CF-related mutations;
• Chronic PA colonization requiring maintenance therapy with inhaled tobramycin;
• Ability to breathe through a mouthpiece and to use the inhaler;
• Ability to perform lung function tests;
• Written informed consent.
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E.4 | Principal exclusion criteria |
A potential subject who meets any of the following criteria will be excluded from participation in this study:
• Severe acute exacerbation of pulmonary infection (needing intravenous treatment);
• Known impaired kidney function (estimated creatinine clearance < 60 ml/min);
• Known aminoglycoside hypersensitivity;
• Therapy (e.g. furosemide) or disease which may complicate evaluation of the study protocol, as judged by the investigator;
• Participation in another drug-investigating clinical study at the start or within 1 month prior to the start;
• Inability to follow instructions of the investigator.
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E.5 End points |
E.5.1 | Primary end point(s) |
Systemic bioavailability of inhaled tobramycin, defined as serum tobramycin AUC0-24hr. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
• Serum tobramycin peak and trough levels;
• Adverse events.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
Tobramycin nebulisation with PariLCPlus nebuliser compared to Akita nebuliser |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |