the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use
A Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION-827359, an Antisense Oligonucleotide Inhibitor of ENaC, Administered to Healthy Volunteers and Patients With Cystic Fibrosis
This study will be conducted in 3 parts: a single ascending dose leading to a multiple ascending dose (MAD) in healthy volunteers, followed by a MAD in participants with cystic fibrosis. The study will enroll approximately 88 subjects.
The study will consist of 4 single-dose randomized cohorts. Subjects enrolled will receive a single inhaled dose of the Study Drug (IONIS-ENaCRx or placebo) on Day 1.
Trial Reference Number
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the name of the treatment or therapy being researched
ENaC inhibitor (mucociliary clearance)
Last edited date
10 June 2019
CF sponsor type