Familial Chylomicronemia, Hypertriglyceridemia
Conditions
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).
Interventions
DRUGARO-APOC3
single or multiple doses of ARO-APOC3 by subcutaneous (sc) injections
DRUGsterile normal saline (0.9% NaCl)
calculated volume to match active treatment
Sponsors
Arrowhead Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
Yes
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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants with Adverse Events (AEs) Possibly or Probably Related to Treatment | Up to Day 113 (+/- 3 days) |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| 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: 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) |
| 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 |
Countries
Australia, Canada, New Zealand
Outcome results
None listed