Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)

Arrowhead Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Severe Hypertriglyceridemia

Treatments: Drug: ARO-APOC3; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04720534

AROAPOC3-2001

Details and patient eligibility

About

The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.

Enrollment: 229 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Based on medical history, evidence of TG ≥ 500 mg/dL and ≤ 4000 mg/dL at Screening
• Fasting TG ≥ 500 mg/dL at Screening
• Willing to follow diet counseling per Investigator judgment based on local standard of care
• Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
• Willing to provide written informed consent and to comply with study requirements

Exclusion criteria

• Active pancreatitis within 12 weeks prior to first dose
• Any planned bariatric surgery or similar procedures to induce weight loss from consent to end of study
• Acute coronary syndrome event within 24 weeks of first dose
• Major surgery within 12 weeks of first dose
• Planned coronary intervention (e.g., stent placement or heart bypass) or any non-cardiac major surgical procedure throughout the study
• Uncontrolled hypertension
• Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B (HBV), seropositive for Hepatitis C (HCV)
• Uncontrolled hypothyroidism or hyperthyroidism
• Hemorrhagic stroke within 24 weeks of first dose
• Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply)

Note: additional inclusion/exclusion criteria may apply per protocol

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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229 participants in 4 patient groups, including a placebo group

ARO-APOC3 10 mg, Day 1 and Week 12

Experimental group

Description:

2 doses of ARO-APOC3 by subcutaneous (sc) injection

Treatment:

Drug: ARO-APOC3

ARO-APOC3 25 mg, Day 1 and Week 12

Experimental group

Description:

2 doses of ARO-APOC3 by sc injection

Treatment:

Drug: ARO-APOC3

ARO-APOC3 50 mg, Day 1 and Week 12

Experimental group

Description:

2 doses of ARO-APOC3 by sc injection

Treatment:

Drug: ARO-APOC3

Placebo, Day 1 and Week 12

Placebo Comparator group

Description:

calculated volume to match active treatment by sc injection

Treatment:

Drug: Placebo