Clinical trials

This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.

This study is designed to evaluate the safety and efficacy of ARO-INHBE in adults with suspected metabolic dysfunction-associated steatohepatitis (MASH).

The purpose of this study is to evaluate the impact of inhaled ARO-RAGE on the late asthmatic response (LAR) following an inhaled allergen challenge in participants with mild atopic asthma.

This is a Phase 1/2a double-blind dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-INHBE in adult participants with obesity (in Part 1), the safety, tolerability, PK, and PD of multiple doses of ARO-INHBE either as monotherapy, or in combination with tirzepatide, in adult participants with obesity with and without type 2 diabetes mellitus (in Part 2 and Part 3).

This study will evaluate the efficacy and safety of plozasiran in approximately 288 adult participants with severe hypertriglyceridemia (SHTG) and history of at least two prior acute pancreatitis (AP) events not attributed to other etiologies, with at least one occurring within the last 12 months prior to screening. Eligible participants will be randomly assigned in a double-blind manner to either receive plozasiran 25 mg by subcutaneous (SC) injection every three months (Q3M) or matching placebo. Enrolled participants will be counseled to remain on the specified low-fat diet and background medications throughout the study. Following completion of the double-blind treatment period, or if the participant has a positively adjudicated AP event (whichever occurs first), participants will transition to the 12-month Open-Label Extension (OLE) treatment period receiving plozasiran 25 mg by SC injection Q3M.

Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia.

This study is designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-033 compared to placebo in adult normal healthy volunteers (NHVs) and the safety, tolerability, PK, PD, and efficacy of multiple doses of ARO-033 in participants with age-related macular degeneration (AMD).

Study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and effects on low-density lipoprotein cholesterol (LDL-C) and triglycerides (TGs) of single-dose ARO-DIMERPA and multiple doses of ARO-DIMERPA in adult participants with mixed hyperlipidemia.

This is a Phase 1/2a double-blind dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of ARO-ALK7 in adult participants with obesity without Type 2 Diabetes Mellitus (T2DM) (Part 1), and the safety, tolerability, and PD of multiple doses of ARO-ALK7 in adult participants with obesity with and without T2DM, either as monotherapy or in combination with tirzepatide (Part 2).

This is a treatment program for the use of plozasiran in adults (AROAPOC3-EAP-002) and adolescents (AROAPOC3-EAP-003) with familial chylomicronemia syndrome (FCS) as well as in adults (AROAPOC3-EAP-004) with high risk severe hypertriglyceridemia (SHTG). The program will enroll eligible patients ≥ 15 years of age, with fasting triglycerides (TGs) ≥ 880 mg/dL (≥ 10 mmol/L) that is not adequately controlled with standard lipid-lowering therapy, and with a diagnosis of FCS. Patients will receive 25 mg of plozasiran by subcutaneous (sc) injection on Day 1 and every 3 months for a total of 5 injections. The duration of the program is 15 months. The program will also enroll eligible patients ≥18 years of age, with fasting TGs \> 880 mg/dL (\> 9.94 mmol/L), or fasting TGs \> 500 mg/dL plus a history of acute pancreatitis, that are not adequately controlled with standard lipid-lowering therapy, and with a diagnosis of high risk SHTG. SHTG patients will receive 25 mg of plozasiran by sc injection on Day 1 and every 3 months for a total of 7 injections. The duration of the program is 21 months.