NCT03647228

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

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

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 98 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 50 Years
• Healthy volunteers: Accepted

Summary

This Phase 1/2a study is a double-blinded (subject and Investigator), randomized, placebo-controlled, dose-escalation study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple nebulized doses of IONIS-ENaCRx.

Detailed description

This study will be conducted in 3 parts: a single ascending dose (SAD) leading to a multiple ascending dose (MAD) in healthy volunteers, followed by a MAD in patients with cystic fibrosis. The study will enroll up to 88 participants. The study will consist of 4 single-dose randomized cohorts. Participants enrolled will receive a single inhaled dose of the Study Drug (IONIS-ENaCRx or placebo) on Day 1.

Conditions

• Healthy Subjects
• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	IONIS-ENaCRx	Ascending single and multiple doses of IONIS-ENaCRx inhaled or nebulized.
DRUG	Placebo	Placebo comparator calculated volume to match active comparator inhaled or nebulized.

Timeline

• Start date: 2018-12-13
• Primary completion: 2020-10-13
• Completion: 2020-10-13
• First posted: 2018-08-27
• Last updated: 2021-02-04

Locations

8 sites across 2 countries: Germany, United Kingdom

Regulatory

• FDA-regulated drug study