Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects

December 29, 2025 updated by: Ikaria Bioscience Pty Ltd

A Phase 1, Randomized, Placebo-controlled, Single Ascending Dose and Phase 2 Randomized, Placebo Controlled, Parallel-Group, Single-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic Effects, and Immunogenicity of RN0361 in Adult Healthy Subjects and Hypertriglyceridemic Subjects

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of single doses of RN0361 in Adult healthy subjects and Adult Hypertriglyceridemic Subjects.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

108

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Queensland
      • Morayfield, Queensland, Australia
        • University of the Sunshine Coast Clinical Trials
      • Sippy Downs, Queensland, Australia, 4556
        • University of The Sunshine Coast Clinical Trials, Sippy Downs
    • Victoria
      • Altona N., Victoria, Australia, 3025
        • Altona Clinical Research
      • Melbourne, Victoria, Australia, 3004
        • Nucleus Network Melbourne
  • United States
    • Florida
      • Tamarac, Florida, United States, 33321
        • TBC Research LLC,
    • Kentucky
      • Versailles, Kentucky, United States, 40383
        • Versailles Family Medicine / Avacare
    • Minnesota
      • Dilworth, Minnesota, United States, 56529
        • Axis Clinicals USA
    • Texas
      • Houston, Texas, United States, 77061
        • Synergy Groups Medical
      • Webster, Texas, United States, 77598
        • Tranquil
    • Utah
      • Pleasant View, Utah, United States, 84404
        • Ogden Clinic, Mountain View / Avacare

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Description

Phase I：

Inclusion Criteria:

  • Willing to provide written informed consent before any study-specific procedures.
  • Comply with the study requirements and restrictions as listed in the Informed Consent Form and the protocol.
  • Fasting serum triglyceride levels > 80 mg/dL and fasting LDL-C ≥70 mg/dL at screening
  • Female participants must either be nonchildbearing or, if of childbearing potential, not pregnant, not breastfeeding, and using effective contraception. Male participants must use condoms and ensure their partners use contraception if they are of childbearing potential.
  • Participants must avoid sperm or egg donation during the study

Exclusion Criteria:

  • History or presence of any serious or uncontrolled disease
  • clinically significant health concerns
  • Recent vaccination with live vaccines, except for influenza, or plans to receive such during the study.
  • Positive tests for alcohol or drugs of abuse at screening.
  • History of multiple drug allergies or allergic reactions to specific components used in the study.

Phase II:

Inclusion Criteria:

  • Willing to provide written informed consent before any study-specific procedures.
  • Comply with the study requirements and restrictions as listed in the Informed Consent Form and the protocol.
  • Fasting serum triglyceride levels ≥ 300 mg/dL at screening
  • Female participants must either be nonchildbearing or, if of childbearing potential, not pregnant, not breastfeeding, and using effective contraception. Male participants must use condoms and ensure their partners use contraception if they are of childbearing potential.

Exclusion Criteria:

  • History or presence of any serious or uncontrolled disease
  • Active pancreatitis within 12 weeks prior to Day 1
  • Uncontrolled hypertension (sitting blood pressure) >160/100 mm Hg
  • Uncontrolled diabetes
  • Symptomatic heart failure (NYHA II-IV)
  • Positive serologic test of HBV, HCV, or HIV
  • Alcohol or drugs abuse
  • History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
  • History of intolerance to SC injection or relevant abdominal scarring (surgical, burns, etc)
  • History of malignancy within the last 2 years prior to the date of consent

Note: Additional inclusion/exclusion ceiteria may apply, per protocol.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RN0361
subcutaneous injections
Drug: RN0361
Placebo
Placebo Comparator: Placebo
calculated volume to match active treatment
Drug: RN0361
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects.
Time Frame: Up to Day85
Phase 1
Up to Day85
Absolute and percent change from baseline in APOC3 and fasting triglycerides (TG) in subjects with HTG
Time Frame: Up to Day183
Phase 2
Up to Day183

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Plasma pharmacokinetics (PK) parameters (Cmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (Tmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (AUC 0-24) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (t1/2) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (AUC0-inf) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (Vz/F) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Plasma pharmacokinetics (PK) parameters (λz) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to 48 hours post-dose
Phase 1
Up to 48 hours post-dose
Urine pharmacokinetics (PK) parameters (CLR) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Urine pharmacokinetics (PK) parameters (Aet) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Urine pharmacokinetics (PK) parameters (Aeu) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Urine pharmacokinetics (PK) parameters (FE) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Absolute and percent change from baseline in APOC3 in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Absolute and percent change from baseline in TG in adult healthy subjects.
Time Frame: Up to Day 85
Phase 1
Up to Day 85
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in subjects with HTG.
Time Frame: Up to Day 183
Phase 2
Up to Day 183
Absolute and percent change from baseline in remnant cholesterol and VLDL-C in subjects with HTG.
Time Frame: Up to Day 183
Phase 2
Up to Day 183

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ikaria Bioscience Pty Ltd

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 30, 2024

Primary Completion (Estimated)

June 3, 2026

Study Completion (Estimated)

August 26, 2026

Study Registration Dates

First Submitted

June 6, 2024

First Submitted That Met QC Criteria

June 18, 2024

First Posted (Actual)

June 24, 2024

Study Record Updates

Last Update Posted (Actual)

December 30, 2025

Last Update Submitted That Met QC Criteria

December 29, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RN0361-101

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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