Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
Phase 1
Recruiting
- Conditions
- HypertriglyceridemiaFamilial Chylomicronemia Syndrome
- Interventions
- Registration Number
- NCT06471543
- Lead Sponsor
- Ikaria Bioscience Pty Ltd
- Brief Summary
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
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 108
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description RN0361 RN0361 subcutaneous injections Placebo RN0361 calculated volume to match active treatment
- Primary Outcome Measures
Name Time Method Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects. Up to Day85 Phase 1
Absolute and percent change from baseline in APOC3 and fasting triglycerides (TG) in subjects with HTG Up to Day183 Phase 2
- Secondary Outcome Measures
Name Time Method Plasma pharmacokinetics (PK) parameters (Cmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Plasma pharmacokinetics (PK) parameters (Tmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Plasma pharmacokinetics (PK) parameters (AUC 0-24) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Plasma pharmacokinetics (PK) parameters (Vz/F) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Urine pharmacokinetics (PK) parameters (Aet) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to Day 85 Phase 1
Urine pharmacokinetics (PK) parameters (Aeu) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to Day 85 Phase 1
Urine pharmacokinetics (PK) parameters (FE) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to Day 85 Phase 1
Absolute and percent change from baseline in TG in adult healthy subjects. Up to Day 85 Phase 1
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in subjects with HTG. Up to Day 183 Phase 2
Absolute and percent change from baseline in remnant cholesterol and VLDL-C in subjects with HTG. Up to Day 183 Phase 2
Plasma pharmacokinetics (PK) parameters (t1/2) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Plasma pharmacokinetics (PK) parameters (AUC0-inf) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Plasma pharmacokinetics (PK) parameters (λz) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to 48 hours post-dose Phase 1
Urine pharmacokinetics (PK) parameters (CLR) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects. Up to Day 85 Phase 1
Absolute and percent change from baseline in APOC3 in adult healthy subjects. Up to Day 85 Phase 1
Trial Locations
- Locations (9)
Lifeline Primary Care/Avacare
🇺🇸Lilburn, Georgia, United States
Versailles Family Medicine / Avacare
🇺🇸Versailles, Kentucky, United States
Axis Clinicals USA
🇺🇸Dilworth, Minnesota, United States
Tranquil
🇺🇸Webster, Texas, United States
Ogden Clinic, Mountain View / Avacare
🇺🇸Pleasant View, Utah, United States
University of the Sunshine Coast Clinical Trials
🇦🇺Morayfield, Queensland, Australia
University of the Sunshine Coast Clinical Trials, Sippy Downs
🇦🇺Sippy Downs, Queensland, Australia
Altona Clinical Research
🇦🇺Altona N., Victoria, Australia
Nucleus Network Melbourne
🇦🇺Melbourne, Victoria, Australia
Lifeline Primary Care/Avacare🇺🇸Lilburn, Georgia, United StatesAziz Pirani, MD, DoctorContactazizpirani@cctresearch.com