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

  • End date
    Oct 28, 2023
  • participants needed
  • sponsor
    Arrowhead Pharmaceuticals
Updated on 28 July 2022


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.

Condition Homozygous Familial Hypercholesterolemia
Treatment ARO-ANG 3 Injection
Clinical Study IdentifierNCT05217667
SponsorArrowhead Pharmaceuticals
Last Modified on28 July 2022


Yes No Not Sure

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 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
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