Cystic Fibrosis, Pulmonary Clinical Trial
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
Verified date | October 2022 |
Source | Arrowhead Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Arrowhead Pharmaceuticals |
Australia, New Zealand,
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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