E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011763 |
E.1.2 | Term | Cystic fibrosis lung |
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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 effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on FEF75 (a lung function parameter for peripheral airflow limitation) in children with CF who are on maintenance treatment with rhDNase. |
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E.2.2 | Secondary objectives of the trial |
To investigate the effect of treatment with nebulized rhDNase targeted to the peripheral airways compared to rhDNase targeted to the central airways on lung clearance index (LCI), and on spirometry parameters (MMEF25-75, FEV1 and FVC) in children with CF who are on maintenance treatment with rhDNase. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
The criteria of inclusion will be the following: - Age between 6 and 18 years old; - Diagnosis of CF confirmed by sweat-test and/or DNA analysis and/or electro physiology testing (nasal potential difference measurement); - Routine treatment with rhDNase once daily, started at least one month before enrolment in the study; - Stable condition, in this study defined as: no i.v antibiotics (hospital or at home) in the previous month and constant medication regime during the previous 2 weeks (for example: no additional oral antibiotics course, no newly started inhaled or systemic corticosteroids etc). - Ability to perform lung function tests (assessed by trained lung function technician); - Lung function: FVC ³ 40% predicted; - Signed written informed consent.
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E.4 | Principal exclusion criteria |
The following exclusion criteria will be used: - Inability to follow instructions of the investigator; - Inability to inhale rhDNase; - Clinical condition not stable, as assessed by the patient’s paediatrician; - Concomitant medical conditions that effect inhaled treatment (e.g. cleft palate, severe malacia); - Current respiratory tract infection; - Pulmonary complications that might put the patient at risk to participate in the study; - Neuromuscular disease; - Poor compliance with treatment as assessed by the patient’s paediatrician; - Active ABPA (allergic bronchopulmonary aspergillosis) defined as an oral course of prednisone for ABPA within the last three months.
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint will be the change in FEF75 as a result of treatment. FEF75 is the most suitable endpoint since it is sensitive to peripheral airways obstruction.
Secondary endpoints will include: - Lung clearance index (LCI) measurements as assessed by multiple breath washout; - Other values obtained in the flow volume curve: MMEF25-75, FEV1, FVC.
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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 | No |
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 | 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 | 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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Control: central (=standard) deposition of rhDNase. Investigationl: peripheral deposition of rhDNase |
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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 | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 | 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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The investigator will notify the accredited METC of the end of the study within a period of 8 weeks (90 days). The end of the study is defined as the last patient’s last visit. In case the study is ended prematurely, the investigator will notify the accredited METC within 15 days including the reasons for the premature termination.
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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 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |