Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
24/06/2024

Titles & IDs
Public title
Study of RN0361 in Adult Subjects
Scientific title
A Phase 1, Randomized, Single-blinded, Placebo-controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic Effects and Immunogenicity of RN0361 in Adult Subjects With Elevated Triglycerides
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 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
solution
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [1] 0 0
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.
Primary outcome [2] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [2] 0 0
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.
Primary outcome [3] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [3] 0 0
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.
Primary outcome [4] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [4] 0 0
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.
Primary outcome [5] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [5] 0 0
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.
Primary outcome [6] 0 0
Safety and tolerability of RN0361 as evaluated when administered as single and multiple ascending SC doses in healthy adult subjects with elevated triglycerides
Timepoint [6] 0 0
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 [1] 0 0
Maximum plasma concentration (Cmax) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite
Timepoint [1] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [2] 0 0
Time to reach maximum plasma concentration (tmax) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite
Timepoint [2] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [3] 0 0
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
Timepoint [3] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [5] 0 0
Terminal elimination half-life (t1/2) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite
Timepoint [5] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [6] 0 0
Elimination rate constant (?z) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite
Timepoint [6] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [7] 0 0
Apparent volume of distribution (Vz/F) of single-dose and multiple-dose plasma PK parameters for RN0361 and its metabolite
Timepoint [7] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [8] 0 0
Renal clearance (CLR) of single-dose and multiple-dose urine PK parameters for RN0361 and its metabolite AS3'N-1
Timepoint [8] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [9] 0 0
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
Timepoint [9] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [10] 0 0
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
Timepoint [10] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [11] 0 0
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
Timepoint [11] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.
Secondary outcome [12] 0 0
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
Timepoint [12] 0 0
Up to Day 85 in Part 1 [SAD] and Day 113 in Part 2 [MAD]. Additionally, there may be an extra follow-up period.

Eligibility
Key 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
Minimum age
18 Years
Maximum age
60 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.

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


Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
30/09/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2026
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network Melbourne - Melbourne
Recruitment postcode(s) [1] 0 0
3004 - Melbourne

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

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/study/NCT06471543
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
Esther J JIANG
Address 0 0
Country 0 0
Phone 0 0
13916015172
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06471543