Evaluating RN0361 in Healthy and Hypertriglyceridemic Adults

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

This study is testing a new drug called RN0361 to see how safe it is and how it affects healthy adults and those with high triglyceride levels.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 108 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: Ikaria Bioscience Pty Ltd (Industry-sponsored)
• Locations: 9 sites (Lilburn, Georgia and 8 other locations)
• Trial ID: NCT06471543 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of single doses of RN0361 in both healthy adults and adults with hypertriglyceridemia. Participants will be monitored for their triglyceride levels and overall health throughout the study. The trial includes both Phase 1 and Phase 2 evaluations to ensure comprehensive data collection on the drug's effects.

Who should consider this trial

Good fit: Ideal candidates include adults with fasting serum triglyceride levels greater than 80 mg/dL and those diagnosed with familial chylomicronemia syndrome.

Not a fit: Patients with serious or uncontrolled diseases or significant health concerns may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for individuals suffering from hypertriglyceridemia and related conditions.

How similar studies have performed: Other studies have shown promise in evaluating treatments for hypertriglyceridemia, but the specific approach of RN0361 is novel.

Eligibility criteria

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.

Where this trial is running

Lilburn, Georgia and 8 other locations

• Lifeline Primary Care/Avacare — Lilburn, Georgia, United States (Not_yet_recruiting)
• Versailles Family Medicine / Avacare — Versailles, Kentucky, United States (Not_yet_recruiting)
• Axis Clinicals USA — Dilworth, Minnesota, United States (Not_yet_recruiting)
• Tranquil — Webster, Texas, United States (Not_yet_recruiting)
• Ogden Clinic, Mountain View / Avacare — Pleasant View, Utah, United States (Not_yet_recruiting)
• University of the Sunshine Coast Clinical Trials — Morayfield, Queensland, Australia (Not_yet_recruiting)
• University of the Sunshine Coast Clinical Trials, Sippy Downs — Sippy Downs, Queensland, Australia (Not_yet_recruiting)
• Altona Clinical Research — Altona N., Victoria, Australia (Not_yet_recruiting)
• Nucleus Network Melbourne — Melbourne, Victoria, Australia (Recruiting)

Study contacts

• Study coordinator: Esther J JIANG

Conditions Hypertriglyceridemia; Familial Chylomicronemia Syndrome