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Clinical Trials/NCT06471543
NCT06471543Active, not recruitingPhase 1

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

Ikaria Bioscience Pty Ltd10 sites in 2 countries108 target enrollmentStarted: September 30, 2024Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
108
Locations
10
Primary Endpoint
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects.
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

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.

Study Design

Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Eligibility Criteria

Ages
18 Years to 70 Years (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
Yes

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.
  • •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.

Arms & Interventions

RN0361

Experimental

subcutaneous injections

Intervention: RN0361 (Drug)

Placebo

Placebo Comparator

calculated volume to match active treatment

Intervention: RN0361 (Drug)

Outcomes

Primary Outcomes

Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects.

Time Frame: Up to Day85

Phase 1

Absolute and percent change from baseline in APOC3 and fasting triglycerides (TG) in subjects with HTG

Time Frame: Up to Day183

Phase 2

Secondary Outcomes

  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • 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)
  • Absolute and percent change from baseline in APOC3 in adult healthy subjects.(Up to Day 85)
  • Absolute and percent change from baseline in TG in adult healthy subjects.(Up to Day 85)
  • 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)
  • Absolute and percent change from baseline in remnant cholesterol and VLDL-C in subjects with HTG.(Up to Day 183)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (10)

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