A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects
A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects
This is a multicenter, randomized, placebo-controlled, dose-escalation study. Enrollment is
 planned to occur at approximately 14 global sites. Approximately 24 subjects with CF.
This is a multicenter, randomized double-blind, placebo-controlled dose-escalation and
 parallel-arm, dose-ranging study. Enrollment is planned to occur at approximately 14 global
 sites. Approximately 24 subjects with CF who are homozygous for the F508del-CFTR mutation
 will be enrolled in two cohorts.
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.