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A Phase 2 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of ARO-APOC3 in Adults With Dyslipidemia

Dyslipidemia active ID: NCT05413135

This is an open-label extension of the parent studies AROAPOC3-2001 and AROAPOC3-2002. Adult participants with dyslipidemia who completed the blinded 12-month period from either parent study and continue to meet eligibility criteria have the option to be enrolled into this study. Eligible enrolled participants will initially receive open-label ARO-APOC3 at the assigned dose level until a final dose is selected, at which point all participants will be transitioned to the selected dosing regimen.

Inclusion Criteria

  • Adults who are nonpregnant, nonlactating and do not plan to become pregnant during the study
  • Able and willing to provide written informed consent
  • Completed the 48-week study treatment period in the parent study

Timeline

  • June 2022

    Study First Posted

  • July 2022

    Study Start Date

  • October 2025

    Estimated study completion date

Trial Details

Start date:

July 2022

End date:

October 2025

Locations:

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

Participants:

536

Eligibility criteria:

18 Years and older (Adult, Older Adult), All sexes, No healthy volunteers

active

Dyslipidemia ID: NCT05413135

This is an open-label extension of the parent studies AROAPOC3-2001 and AROAPOC3-2002. Adult participants with dyslipidemia who completed the blinded 12-month period from either parent study and continue to meet eligibility criteria have the option to be enrolled into this study. Eligible enrolled participants will initially receive open-label ARO-APOC3 at the assigned dose level until a final dose is selected, at which point all participants will be transitioned to the selected dosing regimen.

Phase 2 Study to Evaluate the Safety and Efficacy of ARO-ANG3 in Subjects With Homozygous Familial Hypercholesterolemia (HOFH)(Gateway)

Homozygous Familial Hypercholesterolemia active ID: NCT05217667

Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

Inclusion Criteria

  • Fasting LDL-C >100 mg/dL at Screening
  • Weight of ≥ 40 kg and body mass index ≥ 18.5 and ≤ 40 kg/m2
  • Diagnosis of HoFH based on a supportive genetic test or clinical diagnosis
  • On stable maximally tolerated lipid lowering therapy
  • Willing to abide by stable low-fat, low-cholesterol, heart-healthy diet for at least 4 weeks prior to Day 1
  • Participants of childbearing potential (males & females) must agree to use highly-effective contraception during the study and for at least 24 weeks from the 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 medications for > 2 menstrual cycles prior to Day 1
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • February 2022

    Study First Posted

  • April 2022

    Study Start Date

  • July 2023

    Estimated study completion date

Trial Details

Start date:

April 2022

End date:

July 2023

Locations:

Australia, Canada, South Africa, United States

Participants:

18

Eligibility criteria:

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

active

Homozygous Familial Hypercholesterolemia ID: NCT05217667

Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks. Participants who complete the first 36 week treatment period may opt to continue in an additional 24-month extension period during which they will receive up to 8 doses open-label doses of ARO-ANG3.

A Phase 1/2a Study Evaluating the Effects of ARO-RAGE in Healthy Subjects and Patients With Inflammatory Lung Disease

Inflammatory Pulmonary Diseases recruiting ID: NCT05276570

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung disease. In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or placebo. In Part 2 of the study, adult participants with inflammatory lung disease will receive 2 doses of ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

Inclusion Criteria

  • Normal pulmonary function tests at Screening (NHVs only)
  • Confirmed diagnosis of asthma based on source verifiable medical record (asthma patients only)
  • No abnormal finding of clinical relevance at Screening (other than asthma for asthma patients)
  • Stable dose of asthma controller medications for at least 4 weeks prior to Screening (asthma patients only)
  • Non-smoking
  • Able to produce an induced sputum sample at Screening
  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. Males must not donate sperm during the study and for at least 12 weeks following the last dose of study drug
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • March 2022

    Study First Posted

  • June 2022

    Study Start Date

  • February 2024

    Estimated Study Completion Date

Trial Details

Start date:

June 2022

End date:

February 2024

Locations:

Australia, New Zealand

Participants:

121

Eligibility criteria:

18 Years to 65 Years, All Sexes, Accepts Healthy Volunteers

recruiting

Inflammatory Pulmonary Diseases ID: NCT05276570

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung disease. In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or placebo. In Part 2 of the study, adult participants with inflammatory lung disease will receive 2 doses of ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative safety and pharmacodynamic data from Part 1.

A Phase 1/2a Study Evaluating the Effects of ARO-MUC5AC Inhalation Solution in Healthy Subjects and Patients With Muco-Obstructive Lung Disease

Chronic Obstructive Pulmonary Disease active ID: NCT05292950

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of ARO-MUC5AC in normal healthy volunteers (NHVs), patients with moderate-to-severe asthma and patients with moderate-to-severe chronic obstructive pulmonary disease (COPD). In part 1 NHVs will receive a single dose of ARO-MUC5AC or placebo. In part 2 of the study, NHVs, adult patients with asthma, and adult patients with COPD will receive 3 doses of ARO-MUC5AC or placebo.

Inclusion Criteria

  • Normal pulmonary function tests at Screening (NHVs only)
  • Confirmed diagnosis of asthma or COPD based on source verifiable medical record (asthma and COPD patients only)
  • No abnormal finding of clinical relevance at Screening (NHVs only)
  • Stable dose of asthma controller medications for at least 28 days prior to Screening (asthma patients only)
  • Documented treatment with an inhaled corticosteroid and at least 1 additional maintenance asthma controller medication for at least 3 months prior to Screening (asthma patients only)
  • Non-smoking (NHVs and asthma patients)
  • Current smoker or ex-smoker with smoking history of ≥ 10 pack-years (COPD patients only)
  • All COPD treatments have been stable for at least one month prior to Screening (COPD patients only)
  • Able to produce an induced sputum sample at Screening
  • Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. Males must not donate sperm during the study and for at least 12 weeks following the last dose of study drug
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • March 2022

    Study First Posted

  • June 2022

    Study Start Date

  • February 2024

    Estimated study completion date

Trial Details

Start date:

June 2022

End date:

February 2024

Locations:

Australia, New Zealand, Poland, Thailand

Participants:

104

Eligibility criteria:

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

active

Chronic Obstructive Pulmonary Disease ID: NCT05292950

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of ARO-MUC5AC in normal healthy volunteers (NHVs), patients with moderate-to-severe asthma and patients with moderate-to-severe chronic obstructive pulmonary disease (COPD). In part 1 NHVs will receive a single dose of ARO-MUC5AC or placebo. In part 2 of the study, NHVs, adult patients with asthma, and adult patients with COPD will receive 3 doses of ARO-MUC5AC or placebo.

A Phase 1 Study Evaluating the Effects of ARO-RAGE Injection for Subcutaneous Administration in Healthy Subjects

Asthma recruiting ID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

Inclusion Criteria

  • Normal pulmonary function tests at Screening prior to sputum induction
  • Normal 12-lead electrocardiogram (ECG) at Screening
  • Non-smoking
  • Able to produce an induced sputum sample at Screening
  • Participants of child-bearing potential (male and female) must use highly effective contraception and cannot donate sperm or eggs during the study or for at least 12 weeks following the end of the study or last dose of study drug, whichever is later. Women must have a negative pregnancy test and cannot be breastfeeding
  • Willing to provide written informed consent and to comply with study requirements

Timeline

  • September 2022

    Study First Posted

  • November 2022

    Study Start Date

  • December 2023

    Estimated study completion date

Trial Details

Start date:

November 2022

End date:

December 2023

Locations:

New Zealand

Participants:

50

Eligibility criteria:

18 Years to 55 Years (Adult), All Sexes, Accepts Healthy Volunteers

recruiting

Asthma ID: NCT05533294

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of ARO-RAGE Injection in normal healthy volunteers.

A Phase 1/2a Study Evaluating the Effects of ARO-MMP7 Inhalation Solution in Healthy Subjects and Patients With Idiopathic Pulmonary Fibrosis

Idiopathic Pulmonary Fibrosis recruiting ID: NCT05537025

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-MMP7 in normal healthy volunteers (NHVs) and in participants with idiopathic pulmonary fibrosis (IPF). The study will initiate with NHVs receiving single ascending doses of ARO-MMP7. Following evaluation of safety and pharmacodynamic (PD) data, participants will receive multiple doses of ARO-MMP7.

Inclusion Criteria (NHVs)

  • Normal pulmonary function tests at Screening
  • Normal electrocardiogram (ECG) at Screening
  • Non-smoking
  • Female participants cannot be pregnant or lactating
  • Male and female participants of childbearing potential must agree to use highly effective contraception and must not donate eggs/sperm during the study and for at least 90 days following end of study or last dose of study drug, whichever is later.

Inclusion Criteria (IPF Participants)

  • Age ≥ 45 years at Screening
  • Clinical diagnosis consistent with IPF based upon established criteria confirmed by review of high-resolution computed tomography (HRCT) and surgical lung biopsy findings (if available)
  • Safely able to undergo bronchoscopy
  • Stable IPF disease at Screening with minimum life expectancy of ≥ 12 months from Screening
  • Female participants cannot be pregnant or lactating
  • Male and female participants of childbearing potential must agree to use highly effective contraception and must not donate eggs/sperm during the study and for at least 90 days following end of study or last dose of study drug, whichever is later.

Timeline

  • September 2022

    Study First Posted

  • January 2023

    Study Start Date

  • August 2024

    Estimated study completion date

Trial Details

Start date:

January 2023

End date:

August 2024

Locations:

New Zealand

Participants:

77

Eligibility criteria:

18 Years and older (Adult, Older Adult), all sexes, accepts healthy volunteers

recruiting

Idiopathic Pulmonary Fibrosis ID: NCT05537025

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-MMP7 in normal healthy volunteers (NHVs) and in participants with idiopathic pulmonary fibrosis (IPF). The study will initiate with NHVs receiving single ascending doses of ARO-MMP7. Following evaluation of safety and pharmacodynamic (PD) data, participants will receive multiple doses of ARO-MMP7.

A Phase 1 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-SOD1 in Adult Patients with amyotrophic lateral sclerosis Harboring a Superoxide Dismutase 1 Mutation Considered to be Causative of amyotrophic lateral sclerosis

ALS active ID: NCT05949294

This is a Phase 1 dose escalation study in adults harboring a SOD1 mutation considered to be causative of ALS. This study will initially use single ascending doses (SADs) to evaluate the safety, tolerability, and PK/PD profiles of ARO-SOD1. The study is primarily intended to evaluate safety as well as effects on CSF SOD1 levels as a biomarker of PD effect. This study will be initiated in symptomatic subjects with ALS carrying a SOD1 gene mutation thought to be causative of ALS, and ARO-SOD1 administered by intrathecal (IT) infusion. After all subjects in a cohort have completed the study as described in the clinical protocol, subjects may be rescreened into a higher dose cohort that is open for enrollment, if subject’s CSF SOD1 level has returned to ≥70% of the predose level, or if the level has returned to ≥50% but <70% of the predose level 12 weeks after the last CSF collection. Subjects not able or willing to rescreen may enroll into an OLE study.

Inclusion Criteria

  • Diagnosis of ALS based on source-verifiable medical record
  • Pathogenic or likely pathogenic SOD1 mutation based on source-verifiable medical records or genetic testing during Screening
  • Slow Vital Capacity (SVC) ≥ 50% of predicted value adjusted for sex, age, and height (from a sitting position) at Screening
  • Able to complete at least 6 months of follow-up
  • If taking any medication or supplement to treat ALS or ALS symptoms, must be on stable dose for ≥ 4 weeks prior to Day 1 and expected to remain at that dose until final study visit and not expected to start these medications after the first dose of ARO-SOD1
  • Able and willing to provide written informed consent and to comply with all study assessments
  • Participants of childbearing potential must agree to use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study drug, whichever is later. Males must not donate sperm and females must not donate eggs during the study and for at least 12 weeks following the end of the study or last dose of study drug, whichever is later.

Timeline

  • July 2023

    Study First Posted

  • October 2023

    Study Start Date

  • January 2025

    Estimated Study Completion Date

Trial Details

Start date:

October 2023

End date:

January 2025

Locations:

Not Provided

Participants:

32

Eligibility criteria:

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

active

ALS ID: NCT05949294

This is a Phase 1 dose escalation study in adults harboring a SOD1 mutation considered to be causative of ALS. This study will initially use single ascending doses (SADs) to evaluate the safety, tolerability, and PK/PD profiles of ARO-SOD1. The study is primarily intended to evaluate safety as well as effects on CSF SOD1 levels as a biomarker of PD effect. This study will be initiated in symptomatic subjects with ALS carrying a SOD1 gene mutation thought to be causative of ALS, and ARO-SOD1 administered by intrathecal (IT) infusion. After all subjects in a cohort have completed the study as described in the clinical protocol, subjects may be rescreened into a higher dose cohort that is open for enrollment, if subject’s CSF SOD1 level has returned to ≥70% of the predose level, or if the level has returned to ≥50% but <70% of the predose level 12 weeks after the last CSF collection. Subjects not able or willing to rescreen may enroll into an OLE study.