NCT04375514

Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetic Effects of ARO-ENaC in Normal Healthy Volunteers and Safety, Tolerability and Efficacy in Patients With Cystic Fibrosis

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single doses of ARO-ENaC in healthy adult volunteers; and to evaluate the safety, tolerability, PK and efficacy of multiple doses of ARO-ENaC in patients with pulmonary cystic fibrosis.

Conditions

• Cystic Fibrosis, Pulmonary

Interventions

Type	Name	Description
DRUG	ARO-ENaC	single or multiple doses of ARO-ENaC by inhalation of nebulized solution
DRUG	Placebo	calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Timeline

• Start date: 2020-07-30
• Primary completion: 2021-05-09
• Completion: 2022-05-13
• First posted: 2020-05-05
• Last updated: 2025-12-17
• Results posted: 2025-12-17

Locations

7 sites across 2 countries: Australia, New Zealand

Regulatory

• FDA-regulated drug study