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

Phase 1

Completed

• Conditions: Hypertriglyceridemia; Familial Chylomicronemia
• Interventions: Drug: ARO-APOC3; Drug: sterile normal saline (0.9% NaCl)

• Registration Number: NCT03783377
• Lead Sponsor: Arrowhead Pharmaceuticals

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-12-19
• Study First Submitted QC: 2018-12-19
• Study First Posted: 2018-12-21
• Study Start: 2019-03-08
• Primary Completion: 2021-02-11
• Study Completion: 2021-02-11
• Status Verified: 2021-03
• Last Update Submitted: 2021-03-02
• Last Update Posted: 2021-03-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 112

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
ARO-APOC3	ARO-APOC3	-
Placebo	sterile normal saline (0.9% NaCl)	-

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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: 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
Reduction in Fasting Serum APOC3 from Pre-Dose Baseline	Up to Day 113 (+/- 3 days)
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: Terminal Elimination Half-Life (t1/2)	Single dose phase: Up to 48 hours post-dose

Trial Locations

Locations (10)

Linear Clinical Research; 🇦🇺 Perth, Western Australia, Australia

Royal Prince Alfred Hospital; 🇦🇺 Camperdown, New South Wales, Australia

Royal Adelaide Hospital; 🇦🇺 Adelaide, South Australia, Australia

Ecogene-21; 🇨🇦 Chicoutimi, Quebec, Canada

University of the Sunshine Coast Clinical Trial Centre; 🇦🇺 Sippy Downs, Queensland, Australia

Robarts Research Institute; 🇨🇦 London, Ontario, Canada

Institute de Recherches Cliniques de Montreal; 🇨🇦 Montreal, Quebec, Canada

Auckland Clinical Studies Limited; 🇳🇿 Grafton, Auckland, New Zealand

Lipid & Diabetes Research Group; 🇳🇿 Christchurch, New Zealand

Middlemore Hospital; 🇳🇿 Papatoetoe, Auckland, New Zealand