ARO-ANG3 Injection for Homozygous Familial Hypercholesterolemia
(Gateway Trial)

Recruiting in Palo Alto (17 mi)

+7 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Arrowhead Pharmaceuticals

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial involves giving ARO-ANG3 injections to people with severe genetic high cholesterol to see if it safely lowers their cholesterol by blocking a protein involved in its production.

Eligibility Criteria

This trial is for individuals with a severe form of high cholesterol called homozygous familial hypercholesterolemia (HoFH). Participants must be on stable cholesterol-lowering therapy, not have heart disease symptoms or uncontrolled diabetes, and agree to use effective contraception. They should also follow a low-fat diet and have LDL-C levels over 100 mg/dL.

Inclusion Criteria

Willing to provide written informed consent and to comply with study requirements

I am on a stable dose of the highest safe cholesterol medication.

Participants of childbearing potential (males & females) must agree to use highly-effective contraception during the study and for at least 24 weeks from the last dose of study medication

+6 more

Exclusion Criteria

Your fasting triglyceride levels are higher than 300 mg/dL when tested before the study.

I have a hormone-related condition affecting my cholesterol or fat levels.

I was diagnosed with diabetes in the last 3 months or my diabetes is not well-controlled (HbA1c > 9%).

+6 more

Participant Groups

The study tests ARO-ANG3 injections in people with HoFH. It involves two initial doses followed by safety and effectiveness checks over 36 weeks. Those who finish can join a further 24-month extension for up to eight more doses.

2Treatment groups

Experimental Treatment

Group I: ARO-ANG3 Dose 2Experimental Treatment1 Intervention

ARO-ANG3 Dose Level 2 SC

Group II: ARO-ANG3 Dose 1Experimental Treatment1 Intervention

ARO-ANG3 Dose Level 1 subcutaneous (SC)