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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT04375514
Other study ID # AROENaC1001
Secondary ID
Status Terminated
Phase Phase 1
First received
Last updated
Start date August 10, 2020
Est. completion date September 3, 2021

Study information

Verified date October 2022
Source Arrowhead Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Terminated
Enrollment 43
Est. completion date September 3, 2021
Est. primary completion date September 3, 2021
Accepts healthy volunteers Accepts Healthy Volunteers
Gender All
Age group 18 Years to 55 Years
Eligibility 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

Study Design


Intervention

Drug:
ARO-ENaC
single or multiple doses of ARO-ENaC by inhalation of nebulized solution
Placebo
calculated volume to match active treatment by inhalation of nebulized solution

Locations

Country Name City State
Australia Research Site Chermside Queensland
Australia Research Site Hamilton
Australia Research Site Nedlands Western Australia
Australia Research Site South Brisbane Queensland
New Zealand Research Site Christchurch
New Zealand Research Site Dunedin
New Zealand Research Site Grafton Auckland

Sponsors (1)

Lead Sponsor Collaborator
Arrowhead Pharmaceuticals

Countries where clinical trial is conducted

Australia,  New Zealand, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment single dose phase: Up to 29 (+/- 2) days; multiple dose phase: Up to 113 (+/- 5 days) post-dose for patients with CF
Secondary Change from Baseline in Serum Electrolytes: Potassium, Sodium, Bicarbonate and Chloride (all in mmol/L) Baseline, single dose phase: Up to 29 (+/- 2) days; multiple dose phase: Up to 113 (+/- 5 days) post-dose for patients with CF
Secondary Change from Baseline in Forced Expiratory Volume (FEV1) in Normal Healthy Volunteers Baseline, Up through Day 29 after a single dose
Secondary PK of ARO-ENaC: Maximum observed Plasma Concentration (Cmax) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
Secondary PK of ARO-ENaC: Time to Maximum Plasma Concentration (Tmax) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
Secondary PK of ARO-ENaC: Terminal Elilmination Half-Life (t1/2) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
Secondary PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
Secondary PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
Secondary PK of ARO-ENaC: Area Under the Plasma Concentration Versus Time Curve From Zero to the Last Quantifiable Plasma Concentration (AUClast) single dose phase: Up through Day 5; multiple dose phase: Up through Day 30 (+/- 2 days)
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