NCT05217667

Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)

Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 18 (actual)
• Sponsor: Arrowhead Pharmaceuticals · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	ARO-ANG 3 Injection	Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period

Timeline

• Start date: 2022-04-22
• Primary completion: 2023-05-02
• Completion: 2025-11-01
• First posted: 2022-02-01
• Last updated: 2025-10-10

Locations

7 sites across 4 countries: United States, Australia, Canada, South Africa

Regulatory

• FDA-regulated drug study