A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers
Alpha 1-Antitrypsin Deficiency
complete
ID: NCT03362242
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
Inclusion Criteria
Women of child bearing 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
Non-smoker for at least one year
Normal lung function
No abnormal finding of clinical relevance at Screening
Normal AAT level at Screening visit
Trial Details
Eligibility criteria:
18 Years to 55 Years (Adult), All Sexes, Accepts Healthy Volunteers
complete
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Effect of ARO-AAT on Serum Alpha-1 Antitrypsin Levels in Normal Adult Volunteers
Alpha 1-Antitrypsin Deficiency
ID: NCT03362242
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-AAT in healthy adult volunteers.
A Phase 1/2a Single Dose-Escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetic Effects of ARO-HBV in Normal Adult Volunteers and Multiple Escalating Doses Evaluating Safety, Tolerability and Pharmacodynamic Effects in HBV Patients
Hepatitis B
complete
ID: NCT03365947
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-HBV in healthy adult volunteers and participants with hepatitis B virus (HBV).
Inclusion Criteria for Parts A & B
Women of childbearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use contraception.
Willing to provide written informed consent and comply with study requirements
Additional Inclusion Criteria for Part B
Diagnosis of chronic HBV infection
HbsAg at screening > or = 50 IU/mL
Liver Elastography score < or = 10.5
Trial Details
Locations:
Australia, Hong Kong, New Zealand
Eligibility criteria:
18 Years to 65 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers
complete
A Phase 1/2a Single Dose-Escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetic Effects of ARO-HBV in Normal Adult Volunteers and Multiple Escalating Doses Evaluating Safety, Tolerability and Pharmacodynamic Effects in HBV Patients
Hepatitis B
ID: NCT03365947
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-HBV in healthy adult volunteers and participants with hepatitis B virus (HBV).
A Phase 1 Single and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-ANG3 in Adult Healthy Volunteers and in Dyslipidemic Patients
Dyslipidemia
complete
ID: NCT03747224
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.
Inclusion Criteria
Women of child bearing 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
On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
Normal electrocardiogram (ECG) at Screening
Trial Details
Locations:
Australia, New Zealand
Eligibility criteria:
18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers
complete
A Phase 1 Single and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-ANG3 in Adult Healthy Volunteers and in Dyslipidemic Patients
Dyslipidemia
ID: NCT03747224
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial hypercholesterolemia and severe hypertriglyceridemia.
A Phase 1b Dose-Finding Study of ARO-HIF2 in Patients With Advanced Clear Cell Renal Cell Carcinoma
complete
ID: NCT04169711
The purpose of this study is to evaluate the safety and efficacy of ARO-HIF2 injection (also referred to as ARO-HIF2) and to determine the recommended Phase 2 dose in the treatment of patients with advanced clear cell renal cell carcinoma (ccRCC).
Inclusion Criteria
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
Histologically confirmed locally advanced or metastatic clear cell renal cell carcinoma that has progressed during or after at least two prior therapeutic regimens which must include vascular endothelial growth factor (VEGF)-targeted therapy and checkpoint inhibitor therapy or that has otherwise failed such therapies, is measurable disease per RECIST 1.1 criteria, is biopsy accessible
Eastern Cooperative Oncology Group (ECOG) Performance Status 0 or 1
Estimated life expectancy of longer than 3 months
Adequate organ function at screening
Trial Details
Eligibility criteria:
18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers
complete
A Phase 1b Dose-Finding Study of ARO-HIF2 in Patients With Advanced Clear Cell Renal Cell Carcinoma
ID: NCT04169711
The purpose of this study is to evaluate the safety and efficacy of ARO-HIF2 injection (also referred to as ARO-HIF2) and to determine the recommended Phase 2 dose in the treatment of patients with advanced clear cell renal cell carcinoma (ccRCC).
A Phase 1/2a Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-HSD in Normal Healthy Volunteers as Well as in Patients With NASH or Suspected NASH
NASH
complete
ID: NCT04202354
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple doses of ARO-HSD in healthy adult volunteers and in patients with NASH or suspected NASH.
Inclusion Criteria
Women of child bearing 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
On a stable diet for at least 4 weeks with no plans to significantly alter diet or weight over course of study
Normal electrocardiogram (ECG) at Screening
No abnormal finding of clinical relevance (other than NASH, suspected NASH in patients) at Screening that could adversely impact subject safety during the study or adversely impact study results
Trial Details
Eligibility criteria:
19 Years to 65 Years (Adult, Older Adult), All Sexes, Accepts healthy volunteers
complete
A Phase 1/2a Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-HSD in Normal Healthy Volunteers as Well as in Patients With NASH or Suspected NASH
NASH
ID: NCT04202354
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple doses of ARO-HSD in healthy adult volunteers and in patients with NASH or suspected NASH.
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome
complete
ID: NCT03783377
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
Inclusion Criteria
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
Normal electrocardiogram (ECG) at screening
Hypertriglyceridemic patients must have a history of fasting serum triglycerides of at least 300 mg/dL (3.38 mmol/L) at screening or verifiable diagnosis of FCS
Trial Details
Locations:
Australia, Canada, New Zealand
Eligibility criteria:
18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers
complete
A Phase 1 Single and Multiple Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Effects of ARO-APOC3 in Adult Healthy Volunteers as Well as in Severely Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome
ID: NCT03783377
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple doses of ARO-APOC3 in healthy adult volunteers and in patients with severe hypertriglyceridemia and familial chylomicronemia syndrome (FCS).
A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]
Alpha-1 Liver Disease
complete
ID: NCT03945292
Participants will be enrolled to receive multiple subcutaneous injections of Fazirsiran Injection (also referred to as TAK-999 Injection or ARO-AAT Injection) or placebo. Eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. However, any participant with a biopsy result within 1 year of screening showing no fibrosis does not require a pre-dose biopsy. Only participants who have liver fibrosis will undergo a post-dose biopsy and may continue treatment in an open-label phase.
Inclusion Criteria
Diagnosis of AATD
Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year
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
Non-smoker for at least 1 year
No abnormal finding of clinical relevance at Screening
Trial Details
Start date:
August 7, 2019
End date:
September 18, 2023
Locations:
Germany, Italy, Netherlands, Portugal, Spain, United States
Eligibility criteria:
18 Years to 75 Years (Adult, Older Adult), All Sexes, No Healthy Volunteers
complete
A Placebo-Controlled, Multi-dose, Phase 2 Study to Determine the Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]
Alpha-1 Liver Disease
ID: NCT03945292
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)
Severe Hypertriglyceridemia
complete
ID: NCT04720534
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.
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
Trial Details
Locations:
Australia, Canada, Germany, Hungary, Netherlands, New Zealand, Poland, United States
Eligibility criteria:
18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers
complete
A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia (SHASTA-2)
Severe Hypertriglyceridemia
ID: NCT04720534
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.
A Double-blind, Placebo-controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-ANG3 in Adults With Mixed Dyslipidemia (ARCHES-2)
Dyslipidemia
active
ID: NCT04832971
The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.
Inclusion Criteria
Based on medical history, evidence of TG ≥ 150 mg/dL but ≤ 499 mg/dL
Fasting levels at Screening of LDL-C ≥ 70 mg/dL OR non-HDL-C ≥ 100 mg/dL after at least 4 weeks of stable diet and stable optimal statin therapy
Mean fasting TG ≥ 150 mg/dL and ≤ 499 mg/dL during Screening collected at two separate and consecutive visits and at least 7 days apart and not more than 17 days apart
Willing to follow diet counseling and maintain a stable diet per Investigator judgment based on local standard of care
Participants of childbearing potential must agree to use highly-effective contraception during the study and for at least 24 weeks from 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 medication for ≥ 2 menstrual cycles prior to Day 1
Men must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
Able and willing to provide written informed consent and to comply with study requirements
Trial Details
Locations:
Australia, Canada, New Zealand, United States
Eligibility criteria:
18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers
active
A Double-blind, Placebo-controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-ANG3 in Adults With Mixed Dyslipidemia (ARCHES-2)
Dyslipidemia
ID: NCT04832971
The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.
A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Mixed Dyslipidemia (MUIR)
Dyslipidemia
complete
ID: NCT04998201
Participants who have met all protocol eligibility criteria will be randomly assigned to treatment (ARO-APOC3 or placebo) in a double-blind fashion and will be evaluted for safety and efficacy over 48 weeks. Participants will be counseled to remain on a specified diet throughout the study, as recommended by the Investigator in accordance with local standard of care. After week 48, participants will be eligible and invited to consent and continue in an open-label extension study. All placebo participants who opt to continue will switch to active drug (ARO-APOC3) during the extension study.
Inclusion Criteria
Based on medical history, evidence of TG ≥ 150 mg/dL but ≤ 499 mg/dL on more than one occasion
Fasting levels at Screening of non-HDL-C ≥ 100 mg/dL OR LDL-C ≥ 70 mg/dL after at least 4 weeks of stable diet and stable optimal statin therapy
Mean fasting TG ≥ 150 mg/dL and ≤ 499 mg/dL during Screening collected at two separate and consecutive visits and at least 7 days apart and not more than 14 days apart
Willing to follow diet counseling as per Investigator judgment based on local standard of care
Participants of childbearing potential (males & females) must use highly-effective contraception during the study and for at least 24 weeks following the last dose of study medication. Males must not donate sperm and females must ot donate eggs during the study and for at least 24 weeks following the last dose of study medication.
Women of childbearing potential must have a negative pregnancy test at Screening and cannot be breastfeeding
Women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to Day 1
Willing to provide written informed consent and to comply with study requirements
Timeline
August 10, 2021
Study First Posted
September 28, 2021
Study Start Date
August 14, 2023
Study Completion Date
Trial Details
Start date:
September 2021
End date:
August 14, 2023
Locations:
Australia, Canada, Hungary, New Zealand, Poland, United States
Eligibility criteria:
18 Years to 65 Years, All Sexes, No Healthy Volunteers
complete
A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Mixed Dyslipidemia (MUIR)
Dyslipidemia
ID: NCT04998201
Participants who have met all protocol eligibility criteria will be randomly assigned to treatment (ARO-APOC3 or placebo) in a double-blind fashion and will be evaluted for safety and efficacy over 48 weeks. Participants will be counseled to remain on a specified diet throughout the study, as recommended by the Investigator in accordance with local standard of care. After week 48, participants will be eligible and invited to consent and continue in an open-label extension study. All placebo participants who opt to continue will switch to active drug (ARO-APOC3) during the extension study.