Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects

NCT ID: NCT06471543

Last Updated: 2025-12-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

108 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-30

Study Completion Date

2026-08-26

Brief Summary

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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.

Detailed Description

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Conditions

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Hypertriglyceridemia Familial Chylomicronemia Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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RN0361

subcutaneous injections

Group Type EXPERIMENTAL

RN0361

Intervention Type DRUG

Placebo

Placebo

calculated volume to match active treatment

Group Type PLACEBO_COMPARATOR

RN0361

Intervention Type DRUG

Placebo

Interventions

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RN0361

Placebo

Intervention Type DRUG

Eligibility Criteria

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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


* 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
* 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:


* 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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Ikaria Bioscience Pty Ltd

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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TBC Research LLC,

Tamarac, Florida, United States

Site Status

Versailles Family Medicine / Avacare

Versailles, Kentucky, United States

Site Status

Axis Clinicals USA

Dilworth, Minnesota, United States

Site Status

Synergy Groups Medical

Houston, Texas, United States

Site Status

Tranquil

Webster, Texas, United States

Site Status

Ogden Clinic, Mountain View / Avacare

Pleasant View, Utah, United States

Site Status

University of the Sunshine Coast Clinical Trials

Morayfield, Queensland, Australia

Site Status

University of the Sunshine Coast Clinical Trials, Sippy Downs

Sippy Downs, Queensland, Australia

Site Status

Altona Clinical Research

Altona N., Victoria, Australia

Site Status

Nucleus Network Melbourne

Melbourne, Victoria, Australia

Site Status

Countries

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United States Australia

Other Identifiers

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RN0361-101

Identifier Type: -

Identifier Source: org_study_id