An Evaluation of the Safety and Tolerability of Multiple Dose Regimens of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis and Stable Lung Disease

Conditions: Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10,000 children. In cystic fibrosis chloride transport across the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli1901 corrects the abnormal transport of chloride ans water in the lungs thereby reducing the formation of mucus plugs and improving clearance.

Registration Number: EUCTR2005-004344-30-HU

Lead Sponsor: AOP Orphan Pharmaceuticals AG

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 18

Inclusion Criteria

16 years of age or older (Phase I of Study) resp. 12 years of age or older (Phase II of Study).
Have a confirmed diagnosis of cystic fibrosis [positive sweat chloride value > 60 mEq/l (by quantitative pilocarpine iontophoresis) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype.
Have Screening FEV1 greater than 60% of predicted.
Have oxygen saturation level measured by pulse oximetry (Sp02) > 90 % on room air.

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) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Have a pulmonary disease such as pneumonia, tuberculosis, or lung cancer.
Have had an acute upper respiratory tract infection within 2 weeks of screening.
Have had an acute lower respiratory tract infection (requiring antibiotics or hospitalization) within 4 weeks of screening.
Have had an exacerbation within 4 weeks of screening.
Are on a tobramycin on-off” treatment
Receive parenteral antibiotics via on-off” treatment.
Have any clinically significant liver, renal, cardiac, neurologic, or hematologic disease.
Have Burkholderia cepacia or allergic bronchopulmonary aspergillosis.
Have smoked more than 3 cigarettes per day within the past 12 months.
Have a history of alcohol (> 40g/d) or drug abuse.
Have participated in an investigational drug study within 4 weeks of screening.
Show bronchial hyperresponsiveness.
Do not have stable lung function, i.e. have more than 10% FEV1 variation in the last 3 months before treatment starts.
Are women of child bearing potential and refuse to use effective contraception or are pregnant or lactating.