Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Stopped early · Phase 2
Conditions studied: Homozygous Familial Hypercholesterolemia
In brief
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.
Key facts
- Study ID
- NCT05217667
- Run by
- Arrowhead Pharmaceuticals
- People needed
- 18
- Starts
- 2022-04-22
- Expected to finish
- 2025-11-13
- Last updated by the study team
- 2026-06-12
Who can join
Age: 16 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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 dysfunction
- History of metastatic malignancy within 3 years of Day 1 (some exceptions apply)
- Planned cardiac procedure/surgery such as coronary artery bypass graft (CABG) surgery, percutaneous coronary intervention (PCI), carotid surgery or stenting, or carotid revascularization
- Note: additional inclusion/exclusion criteria may apply per protocol
Where it is running
- Research Site 4 — Mount Sinai, New York, United States
- Research Site 5 — Cincinnati, Ohio, United States
- Research Site 8 — Camperdown, New South Wales, Australia
- Research Site 3 — Nedlands, Western Australia, Australia
- Research Site 2 — Chicoutimi, Quebec, Canada
- Research Site 1 — Québec, Quebec, Canada
- Research Site 7 — Johannesburg, South Africa
Full record on ClinicalTrials.gov
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