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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06471543




Registration number
NCT06471543
Ethics application status
Date submitted
6/06/2024
Date registered
24/06/2024
Date last updated
4/06/2025

Titles & IDs
Public title
Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
Scientific 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
Secondary ID [1] 0 0
RN0361-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertriglyceridemia 0 0
Familial Chylomicronemia Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RN0361

Experimental: RN0361 - subcutaneous injections

Placebo comparator: Placebo - calculated volume to match active treatment


Treatment: Drugs: RN0361
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in adult healthy subjects.
Timepoint [1] 0 0
Up to Day85
Primary outcome [2] 0 0
Absolute and percent change from baseline in APOC3 and fasting triglycerides (TG) in subjects with HTG
Timepoint [2] 0 0
Up to Day183
Secondary outcome [1] 0 0
Plasma pharmacokinetics (PK) parameters (Cmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [1] 0 0
Up to 48 hours post-dose
Secondary outcome [2] 0 0
Plasma pharmacokinetics (PK) parameters (Tmax) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [2] 0 0
Up to 48 hours post-dose
Secondary outcome [3] 0 0
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.
Timepoint [3] 0 0
Up to 48 hours post-dose
Secondary outcome [4] 0 0
Plasma pharmacokinetics (PK) parameters (t1/2) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [4] 0 0
Up to 48 hours post-dose
Secondary outcome [5] 0 0
Plasma pharmacokinetics (PK) parameters (AUC0-inf) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [5] 0 0
Up to 48 hours post-dose
Secondary outcome [6] 0 0
Plasma pharmacokinetics (PK) parameters (Vz/F) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [6] 0 0
Up to 48 hours post-dose
Secondary outcome [7] 0 0
Plasma pharmacokinetics (PK) parameters (?z) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [7] 0 0
Up to 48 hours post-dose
Secondary outcome [8] 0 0
Urine pharmacokinetics (PK) parameters (CLR) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [8] 0 0
Up to Day 85
Secondary outcome [9] 0 0
Urine pharmacokinetics (PK) parameters (Aet) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [9] 0 0
Up to Day 85
Secondary outcome [10] 0 0
Urine pharmacokinetics (PK) parameters (Aeu) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [10] 0 0
Up to Day 85
Secondary outcome [11] 0 0
Urine pharmacokinetics (PK) parameters (FE) of a single ascending SC dose of RN0361 and its metabolite AS3'N-1 in adult healthy subjects.
Timepoint [11] 0 0
Up to Day 85
Secondary outcome [12] 0 0
Absolute and percent change from baseline in APOC3 in adult healthy subjects.
Timepoint [12] 0 0
Up to Day 85
Secondary outcome [13] 0 0
Absolute and percent change from baseline in TG in adult healthy subjects.
Timepoint [13] 0 0
Up to Day 85
Secondary outcome [14] 0 0
Number and incidence of adverse events (AEs)/serious adverse events (SAEs) possibly or probably related to treatment of RN0361 in subjects with HTG.
Timepoint [14] 0 0
Up to Day 183
Secondary outcome [15] 0 0
Absolute and percent change from baseline in remnant cholesterol and VLDL-C in subjects with HTG.
Timepoint [15] 0 0
Up to Day 183

Eligibility
Key inclusion criteria
Phase I:



* 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
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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.



* 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/09/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/12/2026
Actual
Sample size
Target
108
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
University of the Sunshine Coast Clinical Trials - Morayfield
Recruitment hospital [2] 0 0
University of the Sunshine Coast Clinical Trials, Sippy Downs - Sippy Downs
Recruitment hospital [3] 0 0
Altona Clinical Research - Altona N.
Recruitment hospital [4] 0 0
Nucleus Network Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
- Morayfield
Recruitment postcode(s) [2] 0 0
4556 - Sippy Downs
Recruitment postcode(s) [3] 0 0
3025 - Altona N.
Recruitment postcode(s) [4] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Kentucky
Country [3] 0 0
United States of America
State/province [3] 0 0
Minnesota
Country [4] 0 0
United States of America
State/province [4] 0 0
Texas
Country [5] 0 0
United States of America
State/province [5] 0 0
Utah

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ikaria Bioscience Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Dan xiang
Address 0 0
Country 0 0
Phone 0 0
18516063568
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06471543