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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

Timeline

  • November 2018

    Study First Posted

  • January 2019

    Study Start Date

  • May 2021

    Acrtual study completion date

Trial Details

Start date:

January 2019

End date:

May 2021

Locations:

Australia, New Zealand

Participants:

93

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

complete

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

Timeline

  • November 2019

    Study First Posted

  • August 2020

    Study Start Date

  • January 2022

    Actual study completion date

Trial Details

Start date:

August 2020

End date:

January 2022

Locations:

United States

Participants:

26

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

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).

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

Timeline

  • December 2019

    Study First Posted

  • March 2020

    Study Start Date

  • September 2021

    Actual study completion date

Trial Details

Start date:

March 2020

End date:

September 2021

Locations:

New Zealand

Participants:

50

Eligibility criteria:

19 Years to 65 Years (Adult, Older Adult), All Sexes, Accepts healthy volunteers

complete

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

Timeline

  • December 2018

    Study First Posted

  • March 2019

    Study Start Date

  • February 2021

    Actual study completion date

Trial Details

Start date:

March 2019

End date:

February 2021

Locations:

Australia, Canada, New Zealand

Participants:

112

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

complete

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 active 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

Timeline

  • May 2019

    Study First Posted

  • August 2019

    Study Start Date

  • November 2021

    Actual study completion date

Trial Details

Start date:

August 2019

End date:

November 2021

Locations:

Germany, Italy, Netherlands, Portugal, Spain, United States

Participants:

40

Eligibility criteria:

18 Years to 75 Years (Adult, Older Adult), All Sexes, No Healthy Volunteers

active

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 active 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

Timeline

  • January 2021

    Study First Posted

  • May 2021

    Study Start Date

  • March 2023

    Actual study completion date

Trial Details

Start date:

May 2021

End date:

March 2023

Locations:

Australia, Canada, Germany, Hungary, Netherlands, New Zealand, Poland, United States

Participants:

229

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

active

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

Timeline

  • April 2021

    Study First Posted

  • June 2021

    Study Start Date

  • August 2022

    Estimated study completion date

Trial Details

Start date:

June 2021

End date:

August 2022

Locations:

Australia, Canada, New Zealand, United States

Participants:

204

Eligibility criteria:

18 Years and older (Adult, Older Adult), All Sexes, No Healthy Volunteers

active

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

Participants:

353

Eligibility criteria:

18 Years to 65 Years, All Sexes, No Healthy Volunteers

complete

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.

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and/or Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease

C3 Glomerulopathy recruiting ID: NCT05083364

The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

Inclusion Criteria

  • Willing to provide written informed consent and to comply with study requirements
  • Female participants must be non-pregnant/non-lactating
  • Healthy volunteers must be willing to be vaccinated with a meningococcal and pneumococcal vaccine. C3G and IgAN participants must have been vaccinated or willing to undergo vaccination
  • All participants must be willing to be vaccinated or have a history of vaccination for Haemophilus influenzae
  • Body Mass Index (BMI) between 18.0 and 35.0 kg/m2
  • 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety at discretion of investigator
  • Participants of childbearing potential must use highly effective contraception during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Males must not donate sperm during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later.
  • No abnormal finding of clinical relevance at the Screening evaluation that, in the opinion of the investigator, could adversely impact participant safety or study results

Timeline

  • October 2021

    Study First Posted

  • February 2022

    Study Start Date

  • December 2024

    Estimated Study Completion Date

Trial Details

Start date:

February 2022

End date:

December 2024

Locations:

Australia, Georgia, Germany, Korea, New Zealand, Spain, Thailand, United Kingdom,

Participants:

60

Eligibility criteria:

18 Years to 70 Years (Adult, Older Adult), All Sexes, Accepts Healthy Volunteers

recruiting

C3 Glomerulopathy ID: NCT05083364

The purpose of AROC3-1001 is to evaluate the safety, tolerability, pharmacokinetics and/or pharmacodynamics in adult healthy volunteers (HVs) and in adult patients with complement-mediated renal disease (C3 Glomerulopathy [C3G] and IgA Nephropathy [IgAN]). In Part 1 of the study, HVs will receive either one or two doses of ARO-C3 or placebo. In Part 2 of the study, adult patients with C3G/IgAN will receive 3 open-label doses of ARO-C3. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome (PALISADE)

Familial Chylomicronemia Syndrome active ID: NCT05089084

The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of ARO-APOC3 or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive ARO-APOC3.

Inclusion Criteria

  • Fasting TG ≥ 10 mmol/L (≥ 880 mg/dL) at screening refractory to standard lipid lowering therapy
  • Diagnosis of FCS
  • Willing to follow dietary counseling as per investigator judgement 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 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

Timeline

  • October 2021

    Study First Posted

  • January 2022

    Study Start Date

  • April 2024

    Estimated study completion date

Trial Details

Start date:

January 2022

End date:

April 2024

Locations:

Argentina, Australia, Austria, Belgium, Canada, Croatia, France, Germany, Ireland, Israel, Japan, Korea, Republic of, Mexico, New Zealand, Oman, Poland, Serbia, Singapore, Spain, Turkey, United States

Participants:

72

Eligibility criteria:

18 Years and older, All Sexes, No Healthy Volunteers

active

Familial Chylomicronemia Syndrome ID: NCT05089084

The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of ARO-APOC3 or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive ARO-APOC3.