ARO-ANG3 Injection for Homozygous Familial Hypercholesterolemia

(Gateway Trial)

Recruiting at 7 trial locations

Overseen By Robert Rosenson, MD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Arrowhead Pharmaceuticals

Must be taking: Lipid lowering therapy

Must not be taking: siRNA, Antisense oligonucleoside, Evinacumab, Corticosteroids

Disqualifiers: Uncontrolled diabetes, Endocrine disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 36 weeks

18 Participants Needed

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

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%).

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 2 open-label doses of ARO-ANG3 and are evaluated for safety and efficacy parameters

36 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt to continue in an additional 24-month extension period receiving up to 8 doses of ARO-ANG3

24 months

Treatment Details

Interventions

• ARO-ANG3 Injection

Trial Overview 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.

Participant Groups

Group I: ARO-ANG3 Dose 2 Experimental Treatment 1 Intervention

ARO-ANG3 Dose Level 2 SC

Group II: ARO-ANG3 Dose 1 Experimental Treatment 1 Intervention

ARO-ANG3 Dose Level 1 subcutaneous (SC)

Who Is Running the Clinical Trial?

Arrowhead Pharmaceuticals

Lead Sponsor

Trials

45

Recruited

6,200+