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

NCT05217667 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-05-22

Study results available
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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

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

Sponsors & Collaborators

  • Arrowhead Pharmaceuticals

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
16 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-04-22
Primary Completion
2023-05-02
Completion
2025-11-13
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • South Africa

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05217667 on ClinicalTrials.gov