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HypertriglyceridemiaPhase 1/2NCT06471543

Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects

A Phase 1/2 study testing RN0361 in people with Hypertriglyceridemia and Familial Chylomicronemia Syndrome, run by Ikaria Bioscience Pty Ltd at 10 sites in 2 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record

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Trial 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. Read more about this study

Who is it for?
Ages 18 to 70 yearsHealthy volunteers can take part
What is being tested?
RN0361
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
108 participants
Where
10 sitesin 2 countries
Study dates
30 Sep 2024to 26 Aug 2026
Phase
Phase 1/2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • Healthy volunteers accepted
  • Adult, Older adult

Who may be able to join

  • 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
  • Willing to provide written informed consent before any study-specific procedures.
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  • 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.

Who cannot join

  • 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:
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  • 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.

Trial locations and contacts

10 sites in 2 countries.

10 sites in 2 countries

United States6 sites

  • Axis Clinicals USA

    Dilworth, Minnesota, 56529

  • Synergy Groups Medical

    Houston, Texas, 77061

  • Ogden Clinic, Mountain View / Avacare

    Pleasant View, Utah, 84404

  • TBC Research LLC,

    Tamarac, Florida, 33321

  • Versailles Family Medicine / Avacare

    Versailles, Kentucky, 40383

  • Tranquil

    Webster, Texas, 77598

Other trials in United States

Australia4 sites

  • Altona Clinical Research

    Altona N., Victoria, 3025

  • Nucleus Network Melbourne

    Melbourne, Victoria, 3004

  • University of the Sunshine Coast Clinical Trials

    Morayfield, Queensland

  • University of the Sunshine Coast Clinical Trials, Sippy Downs

    Sippy Downs, Queensland, 4556

Other trials in Australia

About this study

Official title: 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

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.

Being tested
RN0361
Medical subject headings
Hypertriglyceridemia, Familial hyperchylomicronemia syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RN0361Drug

    Placebo

Study groups in NCT06471543
Study groupRoleWhat they receive
RN0361Experimentalsubcutaneous injections
Receives: Drug: RN0361
PlaceboPlacebo comparatorcalculated volume to match active treatment
Receives: Drug: RN0361

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Phase 1

    Measured: Up to Day85

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

    Phase 2

    Measured: Up to Day183

Secondary outcomes (15)

  1. Plasma pharmacokinetics (PK) parameters (Cmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  2. Plasma pharmacokinetics (PK) parameters (Tmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  3. 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.

    Phase 1

    Measured: Up to 48 hours post-dose

  4. Plasma pharmacokinetics (PK) parameters (t1/2) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  5. Plasma pharmacokinetics (PK) parameters (AUC0-inf) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  6. Plasma pharmacokinetics (PK) parameters (Vz/F) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  7. Plasma pharmacokinetics (PK) parameters (λz) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to 48 hours post-dose

  8. Urine pharmacokinetics (PK) parameters (CLR) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

  9. Urine pharmacokinetics (PK) parameters (Aet) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

  10. Urine pharmacokinetics (PK) parameters (Aeu) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

  11. Urine pharmacokinetics (PK) parameters (FE) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

  12. Absolute and percent change from baseline in APOC3 in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

  13. Absolute and percent change from baseline in TG in adult healthy subjects.

    Phase 1

    Measured: Up to Day 85

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

    Phase 2

    Measured: Up to Day 183

  15. Absolute and percent change from baseline in remnant cholesterol and VLDL-C in subjects with HTG.

    Phase 2

    Measured: Up to Day 183

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Jun 2024
  2. Study start30 Sep 2024
  3. Primary completion3 Jun 2026 (estimated)
  4. Study completion26 Aug 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
108 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06471543
Sponsor's protocol ID
RN0361-101
Status last verified
Dec 2025
First submitted
6 Jun 2024
Last updated
30 Dec 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

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