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

• Status: Not yet recruiting
• Trial end date: 2023-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Arrowhead Pharmaceuticals

Criteria

Inclusion Criteria:

• Fasting LDL-C >100 mg/dL at Screening

• Weight of ≥ 30 kg and body mass index ≥ 16 and ≤ 40 kg/m2

• Diagnosis of HoFH based on a supportive genetic test

• 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 dysfunction

• History of 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