Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects

NCT06471543 | Active Not Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: RN0361-101
• Study Type: interventional
• Target: 108
• Locations: 2 countries
• Sites: 10

Brief Summary

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.

Conditions

Hypertriglyceridemia; Familial Chylomicronemia Syndrome

Outcome Measures

Primary Outcomes (2)

• Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects.

  Phase 1

  Up to Day85

• Absolute and percent change from baseline in APOC3 and fasting triglycerides (TG) in subjects with HTG

  Phase 2

  Up to Day183

Secondary Outcomes (15)

• Plasma pharmacokinetics (PK) parameters (Cmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

  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.

  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.

  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.

  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.

  Up to 48 hours post-dose

Study Arms (2)

RN0361

EXPERIMENTAL

subcutaneous injections

Drug: RN0361

Placebo

PLACEBO COMPARATOR

calculated volume to match active treatment

Drug: RN0361

Interventions

RN0361 DRUG

Placebo

Placebo; RN0361

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• 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.
• 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)

Sponsors & Collaborators

• Ikaria Bioscience Pty Ltd: lead

Study Sites (10)

TBC Research LLC,

Tamarac, Florida, 33321, United States

Location

Versailles Family Medicine / Avacare

Versailles, Kentucky, 40383, United States

Location

Axis Clinicals USA

Dilworth, Minnesota, 56529, United States

Location

Synergy Groups Medical

Houston, Texas, 77061, United States

Location

Tranquil

Webster, Texas, 77598, United States

Location

Ogden Clinic, Mountain View / Avacare

Pleasant View, Utah, 84404, United States

Location

University of the Sunshine Coast Clinical Trials

Morayfield, Queensland, Australia

Location

University of the Sunshine Coast Clinical Trials, Sippy Downs

Sippy Downs, Queensland, 4556, Australia

Location

Altona Clinical Research

Altona N., Victoria, 3025, Australia

Location

Nucleus Network Melbourne

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Hypertriglyceridemia; Familial hyperchylomicronemia syndrome

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 6, 2024
• First Posted: June 24, 2024
• Study Start: September 30, 2024
• Primary Completion (Estimated): June 3, 2026
• Study Completion (Estimated): August 26, 2026
• Last Updated: December 30, 2025

Record last verified: 2025-12

Locations

US (6); AU (4)