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.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the effect of IDPM compared to control on FEV1 in patients with CF. |
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E.2.2 | Secondary objectives of the trial |
To determine the effect of IDPM compared to control on FEV1 in patients with CF on existing RhDNase treatment. (key objective) To assess whether IDPM treatment: • Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective) • Improves quality of life (key objective) • Reduces days on IV antibiotics, rescue oral or inhaled antibiotics • Reduces days in hospital due to pulmonary exacerbations • Improves other measures of lung function • Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination) • Reduces hospital and community care costs. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects may be included in the study if all of the following criteria are met. The subject must: 1. Have given written informed consent to participate in this study in accordance with local regulations 2. Have a confirmed diagnosis of cystic fibrosis 3. Be aged >= 6 years 4. Have FEV1 >=30 % and <90% predicted 5. Be able to perform all the techniques necessary to measure lung function. |
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E.4 | Principal exclusion criteria |
Subjects are excluded from participating in this study if one or more of the following criteria are met. The subject must NOT: 1. Be investigators, site personnel directly affiliated with this study, and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted. 2. Be considered “terminally ill” or listed for lung transplantation 3. Have had a lung transplant 4. Be using nebulised hypertonic saline concurrently or in the 2 weeks prior to visit 1 5. Have had a significant episode of haemoptysis (>60 mL) in the three months prior to enrolment 6. Have had a myocardial infarction in the three months prior to enrolment 7. Have had a cerebral vascular accident in the three months prior to enrolment 8. Have had major ocular surgery in the three months prior to enrolment 9. Have had major abdominal, chest or brain surgery in the three months prior to enrolment 10. Have a known cerebral, aortic or abdominal aneurysm 11. Be breast feeding or pregnant, or plan to become pregnant while in the study 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only) 13. Be participating in another investigative drug study, parallel to, or within 4 weeks of study entry 14. Have a known allergy to mannitol 15. Be using beta blockers 16. Have uncontrolled hypertension – systolic BP > 190 and or diastolic BP > 100 17. Have a condition or be in a situation which in the Investigator’s opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient’s participation in the study 18. Be ‘Aridol-MTT test positive’. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change in absolute FEV1 (forced expiratory volume in 1 second). |
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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 | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | Yes |
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
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 | Yes |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
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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 | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 27 |
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 | Yes |
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 trial will end at the last visit of the last subject undergoing the trial, including the 26-week extension. For statistical purposes, the study will be terminated once the required numbers of subjects to meet statistical analysis requirements have completed the protocol successfully. |
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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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |