The Sponsor is developing KB407, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) to the airways of people with cystic fibrosis via nebulization. This study is designed to evaluate safety and tolerability of KB407 in people with cystic fibrosis. This study will enroll 4 participants into each of the first two cohorts and will enroll five subjects into the last cohort. Cohort 1 will receive a single dose of KB407 and be followed for 60 days. Subjects in Cohort 1 may rollover into Cohort 2 at the Day 28 Visit. A Data Safety Monitoring Board (DSMB) will meet to determine study progress from Cohort 2 into Cohort 3. In Cohort 2, subjects will be dosed bi-weekly at Day 0 and Day 14. In Cohort 3 subjects will be dosed weekly at Day 0, Day 7, Day 14 and Day 21. All subjects will be followed for a year after the last dose of KB407.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Nebulized solution of KB407, a replication-incompetent HSV-1 expressing full length human CFTR
Hunter Medical Research Institute
Newcastle, New South Wales, Australia
To evaluate the safety and tolerability of KB407 in subjects with Cystic Fibrosis through adverse events as assessed by NCI-CTCAE v5.0
Number of participants with treatment related adverse events as assessed by NCI-CTCAE v5.0
Time frame: Baseline to End of the treatment assessed up to an average of 60 days
To measure the difference in lung function over the course of the study, by change from baseline in forced expiratory volume (FEV1).
Assessment of forced expiratory volume, in one second (FEV1,) will be assessed by pulmonary function test (PFT) as compared to baseline.
Time frame: Baseline to End of the treatment up to an average of 60 days
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