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Completed

NCT Number: NCT03783377

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

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).

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Key information

Conditions

Hypertriglyceridemia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Familial Chylomicronemia Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type I Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site 2, Camperdown, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ARO-APOC3

Drug

single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections

Sterile Normal Saline (0.9% NaCl)

Drug

calculated volume to match active treatment

Primary outcomes

  1. Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment

    Time frame: Up to Day 113 (+/- 3 days)

Secondary outcomes

  1. 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

  2. PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax)

    Time frame: Single dose phase: Up to 48 hours post-dose

  3. PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2)

    Time frame: Single dose phase: Up to 48 hours post-dose

  4. 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

  5. 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

  6. Reduction in Fasting Serum APOC3 from Pre-Dose Baseline

    Time frame: Up to Day 113 (+/- 3 days)

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2019
Primary completion
2021
Study completion
2021
First posted
Dec 21, 2018
Registry last updated
Dec 24, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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