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 | 12.1 |
E.1.2 | Level | PT |
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 long-term safety and efficacy of inhaled Nacystelyn®, in the presence or absence of rhDNase, in patients with CF as reflected in a change per treatment group in the time to first pulmonary exacerbation (PE) during the study period. |
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E.2.2 | Secondary objectives of the trial |
To determine the effects of 52 weeks of inhaled Nacystelyn®, in the presence or absence of rhDNase, on pulmonary function, the number of patients experiencing a PE, the total number of PEs experienced, the number of patients treated by intravenous antibiotics for PE, the number of PEs treated by intravenous antibiotics, the total duration of antibiotic use for PE treatment, the total duration of hospitalisations for PE, and quality of life (QoL). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients are eligible to be included in the study if they: 1. Have a confirmed diagnosis of CF based on the following: sweat chloride >40 mEq/L and/or genotype with two identifiable mutations consistent with CF and one or more clinical features consistent with CF; 2. Are aged ≥ 12 years; 3. Have FEV1 ≥ 40.0% of predicted; 4. Have experienced at least one treated PE in the previous 12 months prior to Screening; 5. Are on stable rhDNase treatment (2.5 mg via nebuliser once daily) for 3 months before Screening (only for the patients taking rhDNase during the study); 6. Have >80% and <120% compliance during the Run-In period; 7. Have signed a written informed consent form (or assent provided by the parent/guardian) and are willing and able to comply with all study procedures.
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E.4 | Principal exclusion criteria |
Patients will be ineligible if one or more of the following statements are applicable: 1. Patient has experienced a treated PE, resolved or unresolved, within 1 month of being screened; 2. Patient has been hospitalised or has had changes in antimicrobial, anti inflammatory, corticosteroids or long-acting bronchodilator medications within 2 weeks of randomisation; 3. Patient has been taking any mucolytic drug except rhDNase within 2 weeks of randomisation; 4. Patient has a history of hemoptysis (> 30 cc) within 3 months of randomisation; 5. Patient has participated in another clinical study within 1 month of randomisation; 6. Patient is a smoker of >10 cigarettes/day (or equivalent) or having a smoking history of >10 pack years; 7. Patient has a history of drug, alcohol, or medication abuse within the past 12 months; 8. Patient is pregnant or breast-feeding; 9. Female patient is of child-bearing potential and they or their partner are not using an acceptable form of contraception (defined as the use of an intra-uterine device [IUD], a barrier method with spermicide, condoms, subdermal implants or oral contraceptives); 10. Patient has a history of malignancy within the past 5 years; 11. Patient has a history of lung transplantation, is planning to undergo a lung transplantation during the study period or is currently on a transplantation list; 12. Patient known to have, or at risk of contracting, human immunodeficiency virus (HIV), Hepatitis B, Hepatitis C or tuberculosis; 13. Patients with a know hypersensitivity or intolerance of nacystelyn, acetylcysteine, lysine or lactose; 14. Patient has a condition/abnormality of baseline clinical laboratory results that, in the opinion of the Principal Investigator, would compromise the safety of the patient.
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E.5 End points |
E.5.1 | Primary end point(s) |
Time to first PE within the study duration on an ITT population. |
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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 | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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 | No |
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 | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 78 |
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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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 | 2 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 0 |