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

Hypertriglyceridemia Clinical Trial

Official title:

A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03783377
• Other study ID #: AROAPOC31001
• Status: Completed
• Phase: Phase 1
• Start date: March 8, 2019
• Est. completion date: February 11, 2021

Study information

• Verified date: March 2021
• Source: Arrowhead Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 112
• Est. completion date: February 11, 2021
• Est. primary completion date: February 11, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Familial Chylomicronemia
• Hyperlipoproteinemia Type I
• Hypertriglyceridemia

Intervention

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

Locations

Country	Name	City	State
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Royal Prince Alfred Hospital	Camperdown	New South Wales
Australia	Linear Clinical Research	Perth	Western Australia
Australia	University of the Sunshine Coast Clinical Trial Centre	Sippy Downs	Queensland
Canada	Ecogene-21	Chicoutimi	Quebec
Canada	Robarts Research Institute	London	Ontario
Canada	Institute de Recherches Cliniques de Montreal	Montreal	Quebec
New Zealand	Lipid & Diabetes Research Group	Christchurch
New Zealand	Auckland Clinical Studies Limited	Grafton	Auckland
New Zealand	Middlemore Hospital	Papatoetoe	Auckland

Sponsors (1)

Lead Sponsor	Collaborator
Arrowhead Pharmaceuticals

Countries where clinical trial is conducted

Australia,  Canada,  New Zealand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment		Up to Day 113 (+/- 3 days)
Secondary	Pharmacokinetics (PK) of ARO-APOC3 in Normal Healthy Volunteers (NHVs): Maximum Observed Plasma Concentration (Cmax)		Single dose phase: Up to 48 hours post-dose
Secondary	PK of ARO-APOC3 in NHVs: Time to Maximum Plasma Concentration (Tmax)		Single dose phase: Up to 48 hours post-dose
Secondary	PK of ARO-APOC3 in NHVs: Terminal Elimination Half-Life (t1/2)		Single dose phase: Up to 48 hours post-dose
Secondary	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
Secondary	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
Secondary	Reduction in Fasting Serum APOC3 from Pre-Dose Baseline		Up to Day 113 (+/- 3 days)