NCT03783377

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).

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
112

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Mar 2019

Typical duration for phase_1

Geographic Reach
3 countries

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 19, 2018

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 21, 2018

Completed
3 months until next milestone

Study Start

First participant enrolled

March 8, 2019

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 11, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 11, 2021

Completed
Last Updated

December 24, 2025

Status Verified

December 1, 2025

Enrollment Period

1.9 years

First QC Date

December 19, 2018

Last Update Submit

December 18, 2025

Conditions

HypertriglyceridemiaFamilial Chylomicronemia

Outcome Measures

Primary Outcomes (1)

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

    Up to Day 113 (+/- 3 days)

Secondary Outcomes (6)

  • Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax)

    Single dose phase: Up to 48 hours post-dose

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

    Single dose phase: Up to 48 hours post-dose

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

    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)

    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)

    Single dose phase: Up to 48 hours post-dose

  • +1 more secondary outcomes

Study Arms (2)

ARO-APOC3

EXPERIMENTAL
Drug: ARO-APOC3

Placebo

PLACEBO COMPARATOR
Drug: sterile normal saline (0.9% NaCl)

Interventions

ARO-APOC3DRUG

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

ARO-APOC3
sterile normal saline (0.9% NaCl)DRUG

calculated volume to match active treatment

Placebo

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Research Site 2

Camperdown, New South Wales, 2050, Australia

Location

Research Site 5

Sippy Downs, Queensland, Australia

Location

Research Site 3

Adelaide, South Australia, 5000, Australia

Location

Research Site 4

Perth, Washington, 6009, Australia

Location

Research Site 7

London, Ontario, N6A 5B7, Canada

Location

Research Site 6

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Research Site 8

Montreal, Quebec, H2W 1R7, Canada

Location

Research Site 9

Grafton, Auckland, 1010, New Zealand

Location

Research Site 10

Papatoetoe, Auckland, 2025, New Zealand

Location

Research Site 11

Christchurch, 8011, New Zealand

Location

Related Publications (1)

  • 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.

    PMID: 38320498DERIVED

MeSH Terms

Conditions

HypertriglyceridemiaHyperlipoproteinemia Type I

Condition Hierarchy (Ancestors)

HyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemias

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 19, 2018

First Posted

December 21, 2018

Study Start

March 8, 2019

Primary Completion

February 11, 2021

Study Completion

February 11, 2021

Last Updated

December 24, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

AU(4)CA(3)NZ(3)