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


Therapeutic category
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

Mucociliary Clearance

Trial status

Closed to recruitment

Participating Centres
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

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


the name of the treatment or therapy being researched

ENaC inhibitor (mucociliary clearance)

Last edited date

04 June 2020

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

CF centres running this trial


Celerion Research Institute


22-24 Lisburn Rd Belfast BT9 6AD

Recruitment starts

July 2019

Recruitment ends

January 2020


Elborn, Stuart

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King's College Hospital NHS Foundation Trust


Denmark Hill London Greater London SE5 9RS

Recruitment starts

February 2020

Recruitment ends

May 2020


Waller , Michael

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Medicines Evaluation Unit


The Langley Building Southmoor Road Wythenshawe Manchester M23 9QZ

Recruitment ends

January 2020



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