Status:
ACTIVE_NOT_RECRUITING
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Lead Sponsor:
Arrowhead Pharmaceuticals
Conditions:
Homozygous Familial Hypercholesterolemia
Eligibility:
All Genders
16+ years
Phase:
PHASE2
Brief 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 par...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 22 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 1 2025
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT05217667
Start Date
April 22 2022
End Date
November 1 2025
Last Update
October 10 2025
Active Locations (7)
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1
Research Site 4
Mount Sinai, New York, United States, 10029
2
Research Site 5
Cincinnati, Ohio, United States, 45227
3
Research Site 8
Camperdown, New South Wales, Australia, 2050
4
Research Site 3
Nedlands, Western Australia, Australia, 6009