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

Phase 1 Completed 112 enrolled Arrowhead Pharmaceuticals

RandomizedSequentialQuadruple-blindTreatment

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).

Timeline

Start: 2019-03-08
Primary completion: 2021-02-11
Completion: 2021-02-11

Publications

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ARO-APOC3	Small interfering RNA (siRNA)	10 mg	Subcutaneous
Subject	ARO-APOC3	Small interfering RNA (siRNA)	25 mg	Subcutaneous
Subject	ARO-APOC3	Small interfering RNA (siRNA)	50 mg	Subcutaneous
Subject	ARO-APOC3	Small interfering RNA (siRNA)	100 mg	Subcutaneous

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

Summary

Timeline

Publications

Drugs

Indications