CompletedNCT03783377Updated Dec 24, 2025

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

A Phase 1 interventional study of ARO-APOC3 and sterile normal saline (0.9% NaCl) in Hypertriglyceridemia and Familial Chylomicronemia, sponsored by Arrowhead Pharmaceuticals. Completed at 10 sites in 3 countries. Open to participants aged 18 Years to 70 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-12-24.

Sponsored by Arrowhead Pharmaceuticals · Phase 1, Interventional, and Treatment

Phase: Phase 1
Study type: Interventional
Enrollment: 112
Allocation: Randomized
Ages: 18 Years to 70 Years
Sex: All

Study 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).

Conditions studied

Hypertriglyceridemia
Familial Chylomicronemia

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Who can participate

Ages eligible: 18 Years to 70 Years
Sexes eligible: All
Accepts healthy volunteers: Yes

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

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

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Sequential assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 112 participants (actual)

Study arms

Experimental
ARO-APOC3
Drug: ARO-APOC3
Placebo comparator
Placebo
Drug: sterile normal saline (0.9% NaCl)

Interventions

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

What researchers measure

Primary outcomes

Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment
Time frame: Up to Day 113 (+/- 3 days)

Secondary outcomes

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)

Study locations

10 sites

Research Site 2
Camperdown, New South Wales 2050, Australia
Research Site 5
Sippy Downs, Queensland, Australia
Research Site 3
Adelaide, South Australia 5000, Australia
Research Site 4
Perth, Washington 6009, Australia
Research Site 7
London, Ontario N6A 5B7, Canada
Research Site 6
Chicoutimi, Quebec G7H 7K9, Canada
Research Site 8
Montreal, Quebec H2W 1R7, Canada
Research Site 9
Grafton, Auckland 1010, New Zealand
Research Site 10
Papatoetoe, Auckland 2025, New Zealand
Research Site 11
Christchurch, 8011, New Zealand

References and documents

Publications

Gaudet D, Clifton P, Sullivan D, Baker J, Schwabe C, Thackwray S, Scott R, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Watts GF, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA Interference Therapy Targeting Apolipoprotein C-III in Hypertriglyceridemia. NEJM Evid. 2023 Dec;2(12):EVIDoa2200325. doi: 10.1056/EVIDoa2200325. Epub 2023 Nov 17. PubMed 38320498 ↗

Individual participant data

Plan to share: No

Registry details

Key details

Study ID: NCT03783377
Lead sponsor: Arrowhead Pharmaceuticals
Responsible party: Sponsor
First posted: Dec 21, 2018
Start date: Mar 8, 2019
Primary completion: Feb 11, 2021
Completion: Feb 11, 2021
Last update: Dec 24, 2025

Oversight

Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.

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