Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis
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ClinicalTrials.gov Identifier: NCT04375514 |
Recruitment Status :
Terminated
(Business decision)
First Posted : May 5, 2020
Last Update Posted : February 24, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cystic Fibrosis, Pulmonary | Drug: ARO-ENaC Drug: Placebo | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 43 participants |
Allocation: | Randomized |
Intervention Model: | Sequential Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Actual Study Start Date : | August 10, 2020 |
Actual Primary Completion Date : | September 3, 2021 |
Actual Study Completion Date : | September 3, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: ARO-ENaC
ARO-ENaC Inhalation
|
Drug: ARO-ENaC
single or multiple doses of ARO-ENaC by inhalation of nebulized solution |
Placebo Comparator: Placebo
Sterile normal saline (0.9% NaCl)
|
Drug: Placebo
calculated volume to match active treatment by inhalation of nebulized solution |
- Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: single dose phase: Up to 29 (+/- 2) days; multiple dose phase: Up to 113 (+/- 5 days) post-dose for patients with CF ]
- Change from Baseline in Serum Electrolytes: Potassium, Sodium, Bicarbonate and Chloride (all in mmol/L) [ Time Frame: Baseline, single dose phase: Up to 29 (+/- 2) days; multiple dose phase: Up to 113 (+/- 5 days) post-dose for patients with CF ]
- Change from Baseline in Forced Expiratory Volume (FEV1) in Normal Healthy Volunteers [ Time Frame: Baseline, Up through Day 29 after a single dose ]
- PK of ARO-ENaC: Maximum observed Plasma Concentration (Cmax) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]
- PK of ARO-ENaC: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]
- PK of ARO-ENaC: Terminal Elilmination Half-Life (t1/2) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]
- PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]
- PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]
- PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to the Last Quantifiable Plasma Concentration (AUClast) [ Time Frame: single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days) ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 55 Years (Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
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)
Note: additional inclusion/exclusion criteria may apply per protocol

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04375514
Australia, Queensland | |
Research Site | |
Chermside, Queensland, Australia, 4032 | |
Research Site | |
South Brisbane, Queensland, Australia, 4101 | |
Australia, Western Australia | |
Research Site | |
Nedlands, Western Australia, Australia, 6009 | |
Australia | |
Research Site | |
Hamilton, Australia, 3204 | |
New Zealand | |
Research Site | |
Grafton, Auckland, New Zealand, 1010 | |
Research Site | |
Christchurch, New Zealand, 8140 | |
Research Site | |
Dunedin, New Zealand, 9054 |
Responsible Party: | Arrowhead Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT04375514 |
Other Study ID Numbers: |
AROENaC1001 |
First Posted: | May 5, 2020 Key Record Dates |
Last Update Posted: | February 24, 2022 |
Last Verified: | January 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
Cystic Fibrosis Pulmonary Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases |
Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |