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A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients 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

Open 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 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


Trial type


the name of the treatment or therapy being researched

ENaC inhibitor (mucociliary clearance)

Last edited date

10 June 2019

CF sponsor

Ionis Pharmaceuticals

CF sponsor type


Who can take part?



Top inclusion criteria
  • Willing to refrain from strenuous exercise/activity for at least 72 hours prior to study visits.
  • Body mass index (BMI) < 35 kg/m2 with a minimum weight of 45 kg.
  • Normal diffusing capacity in the lung (≥ 80% predicted) at Screening
Top exclusion criteria
  • Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1.
  • Respiratory infection within 4 weeks of Study Day 1.
  • Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion, including but not limited to Urine protein/creatinine (P/C) ratio ≥ 0.2 mg/mg,Positive test (including trace) for blood on urinalysis, Alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, alkaline phosphatase (ALP), serum creatinine, blood urea nitrogen (BUN), fasting blood glucose, potassium > upper limit of normal (ULN) and Platelet count

CF centres running this trial


Celerion Research Institute


22-24 Lisburn Rd Belfast BT9 6AD

Recruitment starts

July 2019

Recruitment ends

October 2019


Elborn, Stuart

Get in touch


Medicines Evaluation Unit


The Langley Building Southmoor Road Wythenshawe Manchester M23 9QZ



Get in touch