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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05905068




Registration number
NCT05905068
Ethics application status
Date submitted
2/06/2023
Date registered
15/06/2023
Date last updated
26/01/2024

Titles & IDs
Public title
Adult Subjects With Elevated Low-Density Lipoprotein-Cholesterol to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects
Scientific title
A Phase 1, Randomized, Single-blinded, Placebo-controlled, Single-ascending-dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of RN0191 in Adult Subjects With Elevated Low-Density Lipoprotein-Cholesterol
Secondary ID [1] 0 0
RN0191-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
To Reduce the LDL-C Level in Hypercholesteremia Adult Patients 0 0
Combination for Subgroup High LDL-c Patients With Other Comorbidities 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RN0191 INJECTION

Active Comparator: Cohort 1 will be 25 mg RN0191 (or placebo) -

Active Comparator: Cohort 1 will be 100 mg RN0191 (or placebo) -

Active Comparator: Cohort 1 will be 300 mg RN0191 (or placebo) -

Active Comparator: Cohort 1 will be 500 mg RN0191 (or placebo) -


Treatment: Drugs: RN0191 INJECTION
a sterile solution in each bottle for subcutaneous (SC) injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Exploratory objectives are presented in the body of the protocol.
Timepoint [1] 0 0
For each subject in the study, the duration of the study clinic visits is approximately 21 weeks from screening to Day 85/EOS examination.

Eligibility
Key inclusion criteria
1. Male and female subjects, aged 18 to 60 years, inclusive.

2. Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for
females and >50 kg for males.

3. Serum LDL-C =100mg/dL (2.6 mmol/L) at screening and Day -1.

4. Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.

5. Adequate complete blood counts (complete blood counts [CBCs]; if outside the reference
range, CBC values that are not clinically relevant and are acceptable to the
Investigator)

6. Female subjects are eligible to participate if they are confirmed either not women of
child-bearing potential (WOCBP), or have a negative urine pregnancy test at Day 1, are
not breastfeeding, and willing and able to abide by the contraception guidelines.

7. Male subjects who can produce viable sperm are eligible to participate if they agree
to use an adequate method of contraception as per the contraceptive guidance from
Screening (signing the ICF) until at least 3 months post dose. Subjects with a
partner(s) who is (are) not of childbearing potential are exempt from these
requirements.

8. Male subjects with a pregnant or breastfeeding partner must agree to remain abstinent
from penile-vaginal intercourse or use a male condom during each episode of penile
penetration. In addition, male subjects must refrain from donating sperm from
Screening (signing the ICF) until at least 3 months post dose.

9. Willing to comply with protocol required visit schedule and visit requirements and
provide written informed consent
Minimum age
18 Years
Maximum age
60 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Male and female subjects, aged 18 to 60 years, inclusive.

2. Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for
females and >50 kg for males .

3. Serum LDL-C =100mg/dL (2.6 mmol/L) at screening and Day -1.

4. Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.

5. Adequate complete blood counts (complete blood counts [CBCs]; if outside the reference
range, CBC values that are not clinically relevant and are acceptable to the
Investigator).

6. Female subjects are eligible to participate if they are confirmed either not women of
child-bearing potential (WOCBP), or have a negative urine pregnancy test at Day 1, are
not breastfeeding, and willing and able to abide by the contraception guidelines
(signing the ICF).

7. Male subjects who can produce viable sperm are eligible to participate if they agree
to use an adequate method of contraception as per the contraceptive guidance in
Appendix 3 from Screening (signing the ICF) until at least 3 months post dose.
Subjects with a partner(s) who is (are) not of childbearing potential are exempt from
these requirements.

8. Male subjects with a pregnant or breastfeeding partner must agree to remain abstinent
from penile-vaginal intercourse or use a male condom during each episode of penile
penetration. In addition, male subjects must refrain from donating sperm from
Screening (signing the ICF) until at least 3 months post dose.

9. Willing to comply with protocol required visit schedule and visit requirements and
provide written informed consent.

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
Other
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
14/07/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
27/07/2024
Actual
Sample size
Target
32
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Brisban
Recruitment hospital [1] 0 0
Nucleus Networks - Herston
Recruitment postcode(s) [1] 0 0
4006 - Herston

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

Ethics approval
Ethics application status

Summary
Brief summary
Objectives: The primary and secondary objectives of the study are presented below.
Exploratory objectives are presented in the body of the protocol.

Primary:

• To determine the safety and tolerability of RN0191 administered as escalating single
subcutaneously (SC) doses in adult subjects with elevated low-density lipoprotein-cholesterol

Secondary:

- To evaluate the single-dose pharmacokinetics (PK) of RN0191 in adult subjects with
elevated low-density lipoprotein-cholesterol

- To evaluate the pharmacodynamic (PD) effect of RN0191 on serum levels of low-density
lipoprotein-cholesterol (LDL-C)

- To evaluate the PD effect of RN0191 on plasma levels of proprotein convertase
subtilisin/kexin type 9 (PCSK9)
Trial website
https://clinicaltrials.gov/ct2/show/NCT05905068
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Kristi McLendon, MD
Address 0 0
Nucleus Network
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
jian jiang, MS
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/ct2/show/NCT05905068