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®

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2010-018454-13-CZ
• Date of registration: 21/06/2010
• Prospective Registration: Yes
• Primary sponsor: Laboratoires SMB S.A.
• Public title: 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®
• Scientific title: 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®
• Date of first enrolment: 27/08/2010
• Target sample size: 552
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-018454-13
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Czech Republic, France, Germany, Hungary, Italy, Spain, United Kingdom

Key inclusion & exclusion 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 <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.



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic fibrosis
MedDRA version: 12.1 Level: PT Classification code 10011762 Term: Cystic fibrosis

Intervention(s)

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: Nacystelyn®
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Inhalation powder, hard capsule
Route of administration of the placebo: Inhalation use

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: Nacystelyn®
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Inhalation powder, hard capsule
Route of administration of the placebo: Inhalation use

Primary Outcome(s)

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).

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.

Primary end point(s): Time to first PE within the study duration on an ITT population.

Secondary ID(s)

SMB-NAL-III-09-1

2010-018454-13-DE