Study of RN0361 in Adult Subjects
NCT Number: NCT06471543
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE1
Conditions: Hypertriglyceridemia|Familial Chylomicronemia
Interventions: DRUG: RN0361
Sponsor: Ikaria Bioscience Pty Ltd
Sex: ALL
Age: ADULT
Enrollment: 60
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: SINGLE (PARTICIPANT)|Primary Purpose: TREATMENT
Start Date: 2024-09-30
Completion Date: 2026-06-30
Locations: Nucleus Network Melbourne, Melbourne, Victoria, 3004, Australia

Summary:
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics， pharmacodynamics and immunogenicity of single and multiple doses of RN0361 in Adult subjects.

Primary Outcome Measures: Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Incidence and frequency of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361, Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.|Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Number of Participants with clinically significant changes in clinical laboratory safety tests (hematology, biochemistry, coagulation, and urinalysis) related to treatment of RN0361, Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.|Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Number of Participants with clinically significant changes in vital signs (oral body temperature, blood pressure, heart rate, and respiration rate), Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.|Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Number of Participants with clinically significant changes in 12-lead electrocardiograms (ECGs), Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.|Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Number of Participants with clinically significant changes in physical examinations, Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.|Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides, Incidence and frequency of injection site reactions (ISRs), Up to Day85 in Single Ascending-Dose (SAD) and Day113 in the Multiple Ascending Dose (MAD) part. Additionally, there may be an extra follow-up period.
Secondary Outcome Measures: Maximum plasma concentration (Cmax) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Time to reach maximum plasma concentration (tmax) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Area under the plasma concentration-time curve from time zero to 24h post-dose (AUC0-24) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Area under the plasma concentration versus time curve from time zero extrapolated to infinity (AUC0-inf) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Terminal elimination half-life (t1/2) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Elimination rate constant (λz) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Apparent volume of distribution (Vz/F) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Renal clearance (CLR) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1, Single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Amount of drug excreted in the urine over the collection interval t (Aet) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1, Single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Cumulative amount of drug excreted in urine at the end of each interval (Aeu) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1, Single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Fraction of dose excreted in the urine over the collection interval (FE) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1, Single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.|Cumulative fraction of dose excreted in the urine over collection intervals (FEu) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1, Single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite, Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.