Phase 1a Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RN5681 in Healthy Volunteers

NCT07347678 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: RN5681-101
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of single doses of RN5681 in Adult healthy subjects

Conditions

High Cholesterol

Outcome Measures

Primary Outcomes (2)

• in SAD cohort, To assess the safety and tolerability of subcutaneously (SC) administered RN5681

  Incidence, severity, and causal relationship of adverse events (AEs) as reported by the Investigator, with severity determined according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v6.0

  From the enrollment to the end of treatment at Day180

• in POC cohort, To assess the metabolic markers of efficacy

  Percent change from Baseline in serum LDL-C and Lp(a) at Day 180

  From enrollment to the end of treatment at Day 180

Study Arms (2)

RN5681

EXPERIMENTAL

Drug: RN5681 Investigational product subcutaneous injections

Drug: RN5681

Placebo

PLACEBO COMPARATOR

Drug: Placebo control 0.9% normal saline subcutaneous injection

Drug: Placebo control

Interventions

RN5681 DRUG

Investigational Product

RN5681

Placebo control DRUG

0.9% normal saline SC injection

Placebo

Eligibility Criteria

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

You may qualify if:

• Body mass index (BMI) 18 to 35 kg/m2
• Fasting LDL-C ≥70 mg/dL (1.81 mmol/L) (all SAD cohorts); fasting LDL-C ≥100 mg/dL (2.59 mmol/L) (POC cohort only)
• Lp(a) at Screening: SAD cohort: ≥25 nmol/L POC cohort: ≥100 nmol/L
• Fasting triglycerides \<400 mg/dL (4.51 mmol/L) at Screening
• No clinically significant abnormalities of hepatic or renal function

You may not qualify if:

• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>1.5× the upper limit of normal at screening
• Hemoglobin A1c (HbA1c) ≥6.5% at screening
• Current regular smoker (defined as \>2 cigarettes/day or \>10 cigarettes/week) within 3 months prior to screening
• Use of any siRNA, antisense oligonucleotide (ASO), cell and gene therapy, or clustered regularly interspaced short palindromic repeats (CRISPR) agent in the prior 12 months
• Received any prescription lipid-lowering medication, including but not limited to statins, ezetimibe, and PCSK9 inhibitors to alter serum lipids within 30 days before screening

Sponsors & Collaborators

• Ikaria Bioscience Pty Ltd: lead
• Shanghai Rona Therapeutics Co., Ltd.: collaborator

Study Sites (2)

Q-Pharm Pty Ltd.

Brisbane, Queensland, 4006, Australia

Location

Nucleus Network Pty Ltd.

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Hypercholesterolemia

Condition Hierarchy (Ancestors)

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

Central Study Contacts

Dan Xiang

CONTACT

+86 18516063568; dan.xiang@ronatherapeutics.com

Jie Zeng

CONTACT

jie.zeng@ronatherapeutics.com; jie.zeng@ronatherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2026
• First Posted: January 16, 2026
• Study Start: March 31, 2026
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: January 16, 2026

Record last verified: 2025-01

Locations

AU (2)