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 phase 1/2a study to assess various doses of an ENaC inhibitor medication called ION-827359 in Healthy Volunteers and People With Cystic Fibrosis
A study medication called ION-827359 is being developed to treat lung disease symptoms in people with cystic fibrosis. The study medication is intended to help treat the thick mucus found in the airways of patients with cystic fibrosis.
This new inhaled (nebulized) medication will be investigated in both people with, and without cystic fibrosis. The study will compare the study medication with a placebo (a solution that looks identical to the study medication but contains no active ingredients), which means some participants will receive the study medication, whilst others will receive the placebo. Participants won’t know whether they are receiving the study medication or the placebo.
Participation in the study lasts for approximately 21 weeks (just over 5 months), and will require numerous visits to complete study assessments (blood tests, questionnaires, lung function etc.) to the recruitment centre.
Trial Reference Number
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the name of the treatment or therapy being researched
ENaC inhibitor (mucociliary clearance)
Last edited date
11 February 2020
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