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

NCT ID: NCT04375514

Last Updated: 2025-12-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-30

Study Completion Date

2022-05-13

Brief Summary

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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.

Detailed Description

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Conditions

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Cystic Fibrosis, Pulmonary

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Normal Healthy Volunteer Cohort: ARO-ENaC 20 mg, Days 1-3

Normal healthy volunteers receive double-blind ARO-ENaC 20 mg on Days 1, 2, 3

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: Placebo for ARO-ENaC 20 mg, Days 1-3

Normal healthy volunteers receive double-blind placebo for ARO-ENaC 20 mg on Days 1, 2, 3

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: ARO-ENaC 40 mg, Days 1-3

Normal healthy volunteers receive double-blind ARO-ENaC 40 mg on Days 1, 2, 3

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: Placebo for ARO-ENaC 40 mg, Days 1-3

Normal healthy volunteers receive double-blind placebo for ARO-ENaC 40 mg on Days 1, 2, 3

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: ARO-ENaC 65 mg, Days 1-3

Normal healthy volunteers receive double-blind ARO-ENaC 65 mg on Days 1, 2, 3

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: Placebo for ARO-ENaC 65 mg, Days 1-3

Normal healthy volunteers receive double-blind placebo for ARO-ENaC 65 mg on Days 1, 2, 3

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: ARO-ENaC 180 mg, Days 1-3

Normal healthy volunteers receive double-blind ARO-ENaC 180 mg on Days 1, 2, 3

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: Placebo for ARO-ENaC 180 mg, Days 1-3

Normal healthy volunteers receive double-blind placebo for ARO-ENaC 180 mg on Days 1, 2, 3

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: ARO-ENaC 180 mg, Days 1-3 With Bronchoscopy

Normal healthy volunteers receive double-blind ARO-ENaC 180 mg on Days 1, 2, 3 with the purpose of collecting bronchoscopic samples

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Normal Healthy Volunteer Cohort: Placebo for ARO-ENaC 180 mg, Days 1-3 With Bronchoscopy

Normal healthy volunteers receive double-blind placebo for ARO-ENaC 180 mg on Days 1, 2, 3 to collect bronchoscopic samples

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Cystic Fibrosis Cohort: ARO-ENaC 40 mg, Days 1-3, 22-24

Participants with cystic fibrosis receive double-blind ARO-ENaC 40 mg dosed on Days 1, 2, and 3, and 40 mg dosed on Days 22, 23, and 24

Group Type EXPERIMENTAL

ARO-ENaC

Intervention Type DRUG

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Cystic Fibrosis Cohort: Placebo

Participants with cystic fibrosis receive double-blind placebo for ARO-ENaC 40 mg dosed on Days 1, 2, and 3, and Days 22, 23, and 24.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Interventions

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ARO-ENaC

single or multiple doses of ARO-ENaC by inhalation of nebulized solution

Intervention Type DRUG

Placebo

calculated volume of normal saline to match active treatment by inhalation of nebulized solution

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
* Willing to provide written informed consent and to comply with study requirements
* Normal electrocardiogram (ECG) at Screening
* Non-smoking
* Normal pulmonary function tests at Screening (NHVs only)
* No abnormal finding of clinical relevance at Screening other than CF for CF patients
* Confirmed diagnosis of CF based on source verifiable medical record (CF patients only)
* All other treatments for CF have been stable for at least 2 months and patient is willing to continue this treatment regimen without change for duration of study (CF patients only)

Exclusion Criteria

* Acute lower respiratory infection within 30 days of Screening (NHVs only)
* History of asthma (specifically, those subjects at risk of bronchial hyperactivity), anaphylaxis or airway hyper-reactivity
* Clinically significant history of hyperkalemia or presence of hyperkalemia at Screening
* Clinically significant health concerns (other than CF in CF patients)
* Human Immunodeficiency virus (HIV) infection, seropositive for Hepatitis B Virus (HBV), seropositive for Hepatitis C Virus (HCV)
* Uncontrolled hypertension
* Excessive use of alcohol within one month prior to Screening
* Use of illicit drugs within 1 year prior to Screening
* Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
* CF exacerbation within 30 days of Dosing (CF patients)
* History of solid organ transplant (CF patients)
* Diagnosis of hepatic cirrhosis (CF patients)
Minimum Eligible Age

18 Years

Maximum Eligible Age

55 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site 3

Chermside, Queensland, Australia

Site Status

Research Site 4

South Brisbane, Queensland, Australia

Site Status

Research Site 1

Nedlands, Washington, Australia

Site Status

Research Site 2

Hamilton, , Australia

Site Status

Research Site 6

Grafton, Auckland, New Zealand

Site Status

Research Site 8

Christchurch, , New Zealand

Site Status

Research Site 7

Dunedin, , New Zealand

Site Status

Countries

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Australia New Zealand

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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AROENaC1001

Identifier Type: -

Identifier Source: org_study_id