NCT03747224

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
93

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Jan 2019

Typical duration for phase_1

Geographic Reach
2 countries

6 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

November 16, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

November 20, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

January 7, 2019

Completed
2.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 17, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 17, 2021

Completed
Last Updated

December 24, 2025

Status Verified

December 1, 2025

Enrollment Period

2.4 years

First QC Date

November 16, 2018

Last Update Submit

December 18, 2025

Conditions

DyslipidemiasFamilial HypercholesterolemiaHypertriglyceridemia

Outcome Measures

Primary Outcomes (1)

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

    Up to 113 (+/- 3 days) post-dose

Secondary Outcomes (6)

  • Pharmacokinetics (PK) of ARO-ANG3: Maximum Observed Plasma Concentration (Cmax)

    Single dose phase: Up to 48 hours post-dose

  • PK of ARO-ANG3: Time to Maximum Plasma Concentration (Tmax)

    Single dose phase: Up to 48 hours post-dose

  • PK of ARO-ANG3: Terminal Elimination Half-Life (t1/2)

    Single dose phase: Up to 48 hours post-dose

  • PK of ARO-ANG3: 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-ANG3: 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-ANG3

EXPERIMENTAL
Drug: ARO-ANG3

Placebo

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

Interventions

ARO-ANG3DRUG

single or multiple doses of ARO-ANG3 by subcutaneous (sc) injections

ARO-ANG3
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 child bearing 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
  • On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
  • Normal electrocardiogram (ECG) at Screening

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 (6)

Research Site 2

Camperdown, New South Wales, 2050, Australia

Location

Research Site 3

Adelaide, South Australia, 5000, Australia

Location

Research Site 1

Nedlands, 6009, Australia

Location

Research Site 4

Grafton, Auckland, 1010, New Zealand

Location

Research Site 5

Papatoetoe, Aukland, 2025, New Zealand

Location

Research Site 6

Christchurch, 8011, New Zealand

Location

Related Publications (2)

  • Watts GF, Schwabe C, Scott R, Gladding PA, Sullivan D, Baker J, Clifton P, Hamilton J, Given B, Melquist S, Zhou R, Chang T, San Martin J, Gaudet D, Goldberg IJ, Knowles JW, Hegele RA, Ballantyne CM. RNA interference targeting ANGPTL3 for triglyceride and cholesterol lowering: phase 1 basket trial cohorts. Nat Med. 2023 Sep;29(9):2216-2223. doi: 10.1038/s41591-023-02494-2. Epub 2023 Aug 25.

    PMID: 37626170DERIVED

  • Ying Q, Chan DC, Watts GF. Angiopoietin-like protein 3 inhibitors and contemporary unmet needs in lipid management. Curr Opin Lipidol. 2021 Jun 1;32(3):210-212. doi: 10.1097/MOL.0000000000000747. No abstract available.

    PMID: 33900278DERIVED

MeSH Terms

Conditions

DyslipidemiasHyperlipoproteinemia Type IIHypertriglyceridemia

Condition Hierarchy (Ancestors)

Lipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemias

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

November 16, 2018

First Posted

November 20, 2018

Study Start

January 7, 2019

Primary Completion

May 17, 2021

Study Completion

May 17, 2021

Last Updated

December 24, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

AU(3)NZ(3)