NCT05217667TerminatedPhase 2
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH)
Source: ClinicalTrials.gov · Last updated 12 Jun 2026
Reason given: Business decision, unrelated to safety concerns.
Brief summary (registry, in English)
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.
- Status (registry)
- Terminated
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 18 (actual)
- First submitted
- 20 Jan 2022
- Start date
- 22 Apr 2022
- Primary completion date
- 2 May 2023
- Lead sponsor
- Arrowhead Pharmaceuticals
- Diseases
- Familial hypercholesterolaemia
- NCT number
- NCT05217667
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Use of systemic corticosteroids (some exceptions apply)
Other illness
- 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
Lab values
- 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%)
Other
- 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)
Who the study is looking for
- 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
Age in the registry: 16 years to - · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| - | Camperdown | Australia | Unknown status |
| - | Nedlands | Australia | Unknown status |
| - | Chicoutimi | Canada | Unknown status |
| - | Québec | Canada | Unknown status |
| - | Cincinnati | United States | Unknown status |
| - | Mount Sinai | United States | Unknown status |
| - | Johannesburg | South Africa | Unknown status |
Related publications
- Zodasiran, an RNAi therapeutic targeting ANGPTL3, for treating patients with homozygous familial hypercholesterolaemia (GATEWAY): an open-label, randomised, phase 2 trial.Lancet Diabetes Endocrinol · 2026 · PMID 41422812
This page is information, not medical advice. Discuss any trial with your doctor.