A Study to Assess various doses of an ENaC inhibitor medication (mucociliary clearance) in Healthy Volunteers and People with Cystic Fibrosis


Therapeutic category
Mucociliary Clearance
Trial status
closed Participating Centres
Phase I/II

Full title

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
Trial type
ENaC inhibitor (mucociliary clearance)
Last edited date
CF sponsor
Ionis Pharmaceuticals
CF sponsor type

Who can take part?

Top inclusion criteria
  • Confirmed diagnosis of CF
  • Weight >40Kg
  • FEV1 >/= 50% of predicted
Top exclusion criteria
  • Respiratory infection within 4 weeks of Study Day 1

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