Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)

• ClinicalTrials.gov Identifier: NCT03783377
• Recruitment Status: Completed
• First Posted: December 21, 2018
• Last Update Posted: March 3, 2021
• Sponsor: Arrowhead Pharmaceuticals
• Information provided by (Responsible Party): Arrowhead Pharmaceuticals

Tracking Information

• First Submitted Date: December 19, 2018
• First Posted Date: December 21, 2018
• Last Update Posted Date: March 3, 2021
• Actual Study Start Date: March 8, 2019
• Actual Primary Completion Date: February 11, 2021 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: December 19, 2018): Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment [ Time Frame: Up to Day 113 (+/- 3 days) ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: December 19, 2018)

• Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
• PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
• PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
• PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
• PK of ARO-APOC3 in NHVs: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf) [ Time Frame: Single dose phase: Up to 48 hours post-dose ]
• Reduction in Fasting Serum APOC3 from Pre-Dose Baseline [ Time Frame: Up to Day 113 (+/- 3 days) ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)
• Official Title: A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
• Brief Summary: The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: Randomized; Intervention Model: Sequential Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Hypertriglyceridemia
• Familial Chylomicronemia

Intervention

• Drug: ARO-APOC3
  single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections
• Drug: sterile normal saline (0.9% NaCl)
  calculated volume to match active treatment

Study Arms

• Experimental: ARO-APOC3
  Intervention: Drug: ARO-APOC3
• Placebo Comparator: Placebo
  Intervention: Drug: sterile normal saline (0.9% NaCl)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: March 2, 2021): 112
• Original Estimated Enrollment (submitted: December 19, 2018): 63
• Actual Study Completion Date: February 11, 2021
• Actual Primary Completion Date: February 11, 2021 (Final data collection date for primary outcome measure)

Eligibility 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
• Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS

Exclusion Criteria:

• Clinically significant health concerns
• Regular use of alcohol within one month prior to Screening
• Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study
• Recent use of illicit drugs
• Use of more than two tobacco/nicotine containing or cannabis products per month within 6 months prior to drug administration (applicable only to Normal Healthy Volunteers)

Note: additional inclusion/exclusion criteria may apply, per protocol

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 70 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Canada, New Zealand

Administrative Information

• NCT Number: NCT03783377
• Other Study ID Numbers: AROAPOC31001
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• 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

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Arrowhead Pharmaceuticals
• Study Sponsor: Arrowhead Pharmaceuticals
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Arrowhead Pharmaceuticals
• Verification Date: March 2021