A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis Read More

Inclusion Criteria:

• Male or female subjects with a confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis or two CF-causing mutations,documented in the subject's medical record or confirmed at screening.
• Age 18 and above on the date of informed consent.
• Weight ≥40 kg.
• Homozygous for the F508del-CFTR mutation. Genotyping to be confirmed at screening.
• Ability to perform a valid, reproducible spirometry test with demonstration of a forced expiratory volume in 1 sec (FEV1) >40% of predicted normal for age, sex and height.
• Screening laboratory tests with no clinically significant abnormalities that would interfere with the study assessments (as judged by the Investigator).
• Subjects who are sexually active must agree to follow the study's contraception requirements.

Exclusion Criteria:

• An acute upper or lower respiratory tract infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks prior to Day 1.
• Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening.
• Impaired renal function or known portal hypertension.
• History of prolonged QT and/or QTcF (Fridericia's correction) interval (>450 msec) or QTcF >450 msec at Screening.
• History of solid organ or hematological transplantation.
• History of alcohol abuse or drug addiction (including cannabis, cocaine and opiates) during the past year, (as judged by the Investigator).
• Use of ivacaftor or lumacaftor, within 4 weeks of Day 1
• Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to Day 1.
• Ongoing immunosuppressive therapy (including systemic corticosteroids).
• Hemoglobin <10 g/dL.
• Abnormal liver function, at screening.
• Abnormal renal function at screening.
• Ongoing participation in another clinical study or prior participation without appropriate washout (minimum of 10 half- lives or 30 days, whichever is longer) prior to Screening visit.