Study of Inhaled ARO-RAGE in Allergen-induced Mild Asthma
Inflammatory Pulmonary Diseases
active
ID: NCT07241546
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.
Inclusion Criteria
Clinically stable, mild atopic asthma (FEV1 ≥70% predicted)
Established allergy confirmed by positive skin prick test at screening
Willing and able to perform lung function tests and other study-related procedures
Participants of childbearing potential must consent to use a method of highly-effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last investigational product administration, whichever is later
Trial Details
Start date:
December 2025
Eligibility criteria:
18 to 65 Years Old, All Sexes, No Healthy Volunteers
active
Study of Inhaled ARO-RAGE in Allergen-induced Mild Asthma
Inflammatory Pulmonary Diseases
ID: NCT07241546
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.
A Double-blind, Randomized, Placebo-controlled, Multicenter Study Assessing Olpasiran Use to Prevent First Major Cardiovascular Events in Participants With Elevated Lipoprotein(a) (OCEAN(a)-PreEvent)
Cardiovascular Disease
recruiting
ID: NCT07136012
The primary objective is to evaluate the effect of olpasiran, compared to placebo, on the risk for coronary heart disease death (CHD death), myocardial infarction, or urgent coronary revascularization in participants at risk for a first major cardiovascular event with elevated lipoprotein(a) (Lp[a]).
Inclusion Criteria
Age ≥50 years
Lp(a)≥ 200 nmol/L during screening
Multiple atherosclerotic cardiovascular disease risk factors, and/or evidence of atherosclerosis
Trial Details
Start date:
August 22, 2025
End date:
October 20, 2031
Locations:
Australia, Canada, United States
Eligibility criteria:
50 Years to 105 Years, All Sexes, No Healthy Volunteers
recruiting
A Double-blind, Randomized, Placebo-controlled, Multicenter Study Assessing Olpasiran Use to Prevent First Major Cardiovascular Events in Participants With Elevated Lipoprotein(a) (OCEAN(a)-PreEvent)
Cardiovascular Disease
ID: NCT07136012
The primary objective is to evaluate the effect of olpasiran, compared to placebo, on the risk for coronary heart disease death (CHD death), myocardial infarction, or urgent coronary revascularization in participants at risk for a first major cardiovascular event with elevated lipoprotein(a) (Lp[a]).
Study of ARO-DIMERPA in Adult Participants With Mixed Hyperlipidemia
Mixed Hyperlipidemia
recruiting
ID: NCT07223658
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.
Inclusion Criteria
Willing to follow diet counseling as per Investigator judgment based on local standard of care
Specific fasting TG, LDL-C, and non-high density lipoprotein cholesterol levels at Screening
Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later; participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later
Trial Details
Start date:
December 22, 2025
Eligibility criteria:
18 Years or Older, All Sexes, No Healthy Volunteers
recruiting
Study of ARO-DIMERPA in Adult Participants With Mixed Hyperlipidemia
Mixed Hyperlipidemia
ID: NCT07223658
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.
Study of ARO-ALK7 in Adult Volunteers With Obesity With and Without Type 2 Diabetes Mellitus
Obesity
recruiting
ID: NCT06937203
This study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple doses of ARO-ALK7 in adult volunteers with obesity without Type 2 Diabetes Mellitus (T2DM) (Part 1) and the safety, tolerability, and PD of multiple doses of ARO-ALK7 in adult volunteers with obesity with and without (T2DM) receiving tirzepatide (Part 2).
Inclusion Criteria
Obesity, defined as BMI between 30-50 kg/m2, inclusive, with weight at Screening not to exceed 159 kg (350 lbs)
At least one self-reported, unsuccessful attempt at weight loss with lifestyle modification
No abnormal finding of clinical relevance at Screening that could adversely impact participant safety during the study or adversely impact study results
Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study for at least 90 days following the end of the study or last dose of study drug, whichever is later
Trial Details
Locations:
Australia, New Zealand
Eligibility criteria:
18 to 65 Years Old, All Sexes, No Healthy Volunteers
recruiting
Study of ARO-ALK7 in Adult Volunteers With Obesity With and Without Type 2 Diabetes Mellitus
Obesity
ID: NCT06937203
This study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple doses of ARO-ALK7 in adult volunteers with obesity without Type 2 Diabetes Mellitus (T2DM) (Part 1) and the safety, tolerability, and PD of multiple doses of ARO-ALK7 in adult volunteers with obesity with and without (T2DM) receiving tirzepatide (Part 2).
Study of ARO-MAPT-SC in Healthy Subjects and Subjects With Early Alzheimer’s Disease
Alzheimer’s Disease
recruiting
ID: NCT07221344
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.
Inclusion Criteria (All Participants)
Body mass index between 18.0 and 35.0 kg/m^2 at Screening
Not pregnant or breast-feeding
Able and willing to provide written informed consent prior to the performance of any study specific procedures
Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later; participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later
Inclusion Criteria (Alzheimer's Disease)
Adults aged 50 to 75 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis
On stable doses of AD-related medications for at least 8 weeks prior to Screening Visit and throughout the Screening period until Day 1
Trial Details
Start date:
November 18, 2025
Eligibility criteria:
18 to 75 Years Old, All Sexes, Accepts Healthy Volunteers
recruiting
Study of ARO-MAPT-SC in Healthy Subjects and Subjects With Early Alzheimer’s Disease
Alzheimer’s Disease
ID: NCT07221344
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.
Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Homozygous Familial Hypercholesterolemia
recruiting
ID: NCT07037771
This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.
Inclusion Criteria
Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol >500 mg/dL OR treated LDL-C concentration of ≥ AND both parents with documented total cholesterol >250 mg/dL OR cutaneous or tendinous xanthoma before 10 years of age)
LDL-C ≥70 mg/dL (1.8 mmol/L)
Hemoglobin A1c (HbA1c) ≤9.5%
Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome
Alanine aminotransferase or aspartate aminotransferase <3×ULN
On standard of care, maximally tolerated lipid-lowering therapy
Trial Details
Start date:
June 17, 2025
End date:
August 20, 2027
Eligibility criteria:
12 Years or Older, All Sexes, No Healthy Volunteers
recruiting
Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Homozygous Familial Hypercholesterolemia
ID: NCT07037771
This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.
A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2
Spinocerebellar Ataxia 2
recruiting
ID: NCT06672445
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.
Inclusion Criteria
Non-pregnant, non-lactating
Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on source verifiable medical records or genetic testing at Screening
Scale of Assessment and Rating of Ataxia (SARA) score ≤14
Coagulation parameters within normal ranges at Screening
Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later.
Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later
Trial Details
Eligibility criteria:
18 to 65 Years Old, All Sexes, No Healthy Volunteers
recruiting
A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2
Spinocerebellar Ataxia 2
ID: NCT06672445
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-INHBE in Adult Volunteers With Obesity With and Without Diabetes Mellitus
Obesity
recruiting
ID: NCT06700538
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 (Part 1), and the safety, tolerability and PD of repeat doses of ARO-INHBE in adult participants with obesity with and without type 2 diabetes mellitus receiving tirzepatide (Part 2). Part 2 will commence after evaluation of safety data from Part 1 multiple-dose cohorts through Day 43. The dose for Part 2 will be based upon evaluation of data from Part 1.
Inclusion Criteria
Obesity, defined as Body Mass Index (BMI) between 30 to 50 kg/m2 at Screening
At least one self-reported, unsuccessful attempt at weight loss with lifestyle modification
Willing, able and motivated to comply with all study assessments and adhere to the protocol schedule, including adherence to a stable diet and exercise routine for the duration of the stud
No abnormal finding of clinical relevance at Screening that, in the opinion of investigator, could adversely impact subject safety or adversely impact study results
Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study medication whichever is later
Participants must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study medication whichever is later
Trial Details
Start date:
December 2024
Eligibility criteria:
18 to 65 Years Old, All Sexes, No Healthy Volunteers
recruiting
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-INHBE in Adult Volunteers With Obesity With and Without Diabetes Mellitus
Obesity
ID: NCT06700538
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 (Part 1), and the safety, tolerability and PD of repeat doses of ARO-INHBE in adult participants with obesity with and without type 2 diabetes mellitus receiving tirzepatide (Part 2). Part 2 will commence after evaluation of safety data from Part 1 multiple-dose cohorts through Day 43. The dose for Part 2 will be based upon evaluation of data from Part 1.
Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-3)
Severe Hypertriglyceridemia
active
ID: NCT06347003
This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.
Inclusion Criteria
Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥ 500 mg/dL (≥5.65mmol/L)
Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
Screening HbA1C ≤8.5%
Willing to follow diet counseling and maintain a stable low-fat diet
Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)
Trial Details
Eligibility criteria:
18 Years or Older, All Sexes, No Healthy Volunteers
active
Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-3)
Severe Hypertriglyceridemia
ID: NCT06347003
This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.
Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-4)
Severe Hypertriglyceridemia
active
ID: NCT06347016
This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.
Inclusion Criteria
Established diagnosis of severe hypertriglyceridemia (SHTG) and prior documented evidence (medical history) of fasting TG levels of ≥500 mg/dL (≥5.65 mmol/L)
Mean fasting TG level ≥500 mg/dL (≥5.65 mmol/L) collected at 2 separate and consecutive visits at least 7 days apart and no more than 17 days apart during the screening period
Fasting low density lipoprotein-cholesterol (LDL-C) ≤130 mg/dL (≤3.37 mmol/L) at screening
Screening HbA1C ≤8.5%
Must be on standard of care lipid-lowering medications per local guidelines (unless documented as intolerant as determined by the Investigator)
Trial Details
Eligibility criteria:
18 Years or Older, All Sexes, No Healthy Volunteers
active
Double-Blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Plozasiran in Adults with Severe Hypertriglyceridemia (SHASTA-4)
Severe Hypertriglyceridemia
ID: NCT06347016
This Phase 3 study will evaluate the safety and efficacy of plozasiran injection (ARO-APOC3) in adult participants with severe hypertriglyceridemia (SHTG). After providing informed consent eligible participants will be randomized to receive 4 doses (once every 3 months) of plozasiran or placebo, and be evaluated for efficacy and safety. After Month 12, eligible participants will be offered an opportunity to continue in an optional open-label extension under a separate protocol.
