Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Sponsored by Arrowhead Pharmaceuticals
About this trial
Last updated 8 months ago
Study ID
AROANG3-2003
Status
Active not recruiting
Type
Interventional
Phase
Phase 2
Placebo
No
Accepting
16+ Years
All Sexes
Trial Timing
Ended 7 months ago
What is this trial about?
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.
What are the participation requirements?
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 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