TerminatedPhase 2

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

Stopped: Business decision, unrelated to safety concerns.

United States, Australia, Canada18 participantsStarted 2022-04-22

Plain-language 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.

Who can participate

Age range

16 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Fasting LDL-C \>100 mg/dL at Screening * Weight of ≥ 40 kg and body mass index ≥ 18.5 and ≤ 40 kg/m2 * Diagnosis of HoFH based on a supportive genetic test or clinical diagnosis * On stable maximally tolerated lipid lowering therapy * Willing to abide by stable low-fat, low-cholesterol, heart-healthy diet for at least 4 weeks prior to Day 1 * 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. * Women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding * Women of childbearing potential on hormonal contraceptives must be stable on the medications for \> 2 menstrual cycles prior to Day 1 * Willing to provide written informed consent and to comply with study requirements Exclusion Criteria: * Current use or use within 365 days from Day 1 of any hepatocyte targeted small interfering RNA oligonucleotides (siRNA) or antisense oligonucleoside molecule * Use of evinacumab (some exceptions apply) * Fasting TG \> 300 mg/dL at Screening * Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins * Newly diagnosed (within 3 months prior to informed consent) or poorly controlled diabetes (Hemoglobin A1c \> 9%) * Use of systemic corticosteroids (some exceptions apply) * Symptoms of myocardial ischemia or severe left ventricular dysfu…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percent Change From Baseline in Fasting LDL-C at Week 24

Timeframe: Baseline, Week 24

Trial details

NCT IDNCT05217667

SponsorArrowhead Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-05-02

Results submitted2026-04-29

View on ClinicalTrials.gov More Homozygous Familial Hypercholesterolemia trials