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

NCT ID: NCT03783377

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

112 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-03-08

Study Completion Date

2021-02-11

Brief Summary

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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

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Conditions

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Hypertriglyceridemia Familial Chylomicronemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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ARO-APOC3

Group Type EXPERIMENTAL

ARO-APOC3

Intervention Type DRUG

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

Placebo

Group Type PLACEBO_COMPARATOR

sterile normal saline (0.9% NaCl)

Intervention Type DRUG

calculated volume to match active treatment

Interventions

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ARO-APOC3

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

Intervention Type DRUG

sterile normal saline (0.9% NaCl)

calculated volume to match active treatment

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site 2

Camperdown, New South Wales, Australia

Site Status

Research Site 5

Sippy Downs, Queensland, Australia

Site Status

Research Site 3

Adelaide, South Australia, Australia

Site Status

Research Site 4

Perth, Washington, Australia

Site Status

Research Site 7

London, Ontario, Canada

Site Status

Research Site 6

Chicoutimi, Quebec, Canada

Site Status

Research Site 8

Montreal, Quebec, Canada

Site Status

Research Site 9

Grafton, Auckland, New Zealand

Site Status

Research Site 10

Papatoetoe, Auckland, New Zealand

Site Status

Research Site 11

Christchurch, , New Zealand

Site Status

Countries

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Australia Canada New Zealand

References

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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.

Reference Type DERIVED
PMID: 38320498 (View on PubMed)

Other Identifiers

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AROAPOC31001

Identifier Type: -

Identifier Source: org_study_id