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

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ClinicalTrials.gov Identifier: NCT04375514
Recruitment Status : Recruiting
First Posted : May 5, 2020
Last Update Posted : February 24, 2021
Sponsor:
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals

Brief 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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis, Pulmonary Drug: ARO-ENaC Drug: Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 92 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
Estimated Primary Completion Date : December 15, 2021
Estimated Study Completion Date : March 15, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

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




Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. Change from Baseline in Forced Expiratory Volume (FEV1) in Normal Healthy Volunteers [ Time Frame: Baseline, Up through Day 29 after a single dose ]
  3. 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) ]
  4. 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) ]
  5. 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) ]
  6. 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) ]
  7. 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) ]
  8. 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) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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


Information from the National Library of Medicine

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


Contacts
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Contact: Medical Monitor 626-304-3400 AROENAC@arrowheadpharma.com

Locations
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Australia, Queensland
Research Site Recruiting
Chermside, Queensland, Australia, 4032
Research Site Recruiting
South Brisbane, Queensland, Australia, 4101
Australia, Western Australia
Research Site Recruiting
Nedlands, Western Australia, Australia, 6009
Australia
Research Site Recruiting
Hamilton, Australia, 3204
New Zealand
Research Site Recruiting
Grafton, Auckland, New Zealand, 1010
Research Site Recruiting
Christchurch, New Zealand, 8140
Research Site Recruiting
Dunedin, New Zealand, 9054
Sponsors and Collaborators
Arrowhead Pharmaceuticals
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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, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Additional relevant MeSH terms:
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Cystic Fibrosis
Pulmonary Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases