A Phase III, Multicentre, Randomised, Placebo-Controlled, Double Blind Study of the Incidence of Recurring Pulmonary Exacerbations in Cystic Fibrosis Patients using Two Different Doses of Inhaled Nacystelyn®

Phase 1

• Conditions: Cystic fibrosis; MedDRA version: 12.1Level: PTClassification code 10011762Term: Cystic fibrosis

• Registration Number: EUCTR2010-018454-13-FR
• Lead Sponsor: aboratoires SMB S.A.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2010-05-28
• Last Update Posted: 18 August 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 552

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.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified