A Phase 1a, Randomized, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RN5681 in Healthy Volunteers
Overview
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Ikaria Bioscience Pty Ltd
- Enrollment
- 60
- Locations
- 3
- Primary Endpoint
- in SAD cohort, To assess the safety and tolerability of subcutaneously (SC) administered RN5681
Overview
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
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Single (Participant)
Eligibility Criteria
- Ages
- 18 Years to 65 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •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
Exclusion Criteria
- •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
Arms & Interventions
Placebo
Drug: Placebo control 0.9% normal saline subcutaneous injection
Intervention: Placebo control (Drug)
RN5681
Drug: RN5681 Investigational product subcutaneous injections
Intervention: RN5681 (Drug)
Outcomes
Primary Outcomes
in SAD cohort, To assess the safety and tolerability of subcutaneously (SC) administered RN5681
Time Frame: From the enrollment to the end of treatment at Day180
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
in POC cohort, To assess the metabolic markers of efficacy
Time Frame: From enrollment to the end of treatment at Day 180
Percent change from Baseline in serum LDL-C and Lp(a) at Day 180
Secondary Outcomes
No secondary outcomes reported