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

• Status: Recruiting
• Trial end date: 2022-03-15
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Arrowhead Pharmaceuticals

Criteria

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