Cystic fibrosis MedDRA version: 12.1 Level: PT Classification code 10011762 Term: Cystic fibrosis
Conditions
Interventions
Product Name: Nacystelyn®
Product Code: NAL
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: L-Lysine-N-acetylcysteinate
Current Sponsor code: NAL
Other descriptive name: Nacy
Sponsors
Laboratoires SMB S.A.
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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).;Primary end point(s): Time to first PE within the study duration on an ITT population. | — |
Countries
Czech Republic, France, Germany, Hungary, Italy, Spain, United Kingdom
Outcome results
None listed