To Reduce the LDL-C Level in Hypercholesteremia Adult PatientsPhase 1NCT05905068
Adult Subjects With Elevated Low-Density Lipoprotein-Cholesterol to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects
A Phase 1 study testing RN0191 INJECTION in people with To Reduce the LDL-C Level in Hypercholesteremia Adult Patients and Combination for Subgroup High LDL-c Patients With Other Comorbidities, run by Ikaria Bioscience Pty Ltd at 1 site in Australia.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- To Reduce the LDL-C Level in Hypercholesteremia Adult Patients, Combination for Subgroup High LDL-c Patients With Other Comorbidities
- What is being tested?
- RN0191 INJECTION
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 1 siteAustralia
- Study dates
- 14 Jul 2023to 14 May 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Male and female subjects, aged 18 to 60 years, inclusive.
- Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for females and >50 kg for males.
- Serum LDL-C ≥100mg/dL (2.6 mmol/L) at screening and Day -1.
- Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.
- 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)
- 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.
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- 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.
- 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.
- Willing to comply with protocol required visit schedule and visit requirements and provide written informed consent
Who cannot join
- Male and female subjects, aged 18 to 60 years, inclusive.
- Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for females and >50 kg for males .
- Serum LDL-C ≥100mg/dL (2.6 mmol/L) at screening and Day -1.
- Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.
- 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).
- 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).
Show all 9Show fewer
- 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.
- 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.
- Willing to comply with protocol required visit schedule and visit requirements and provide written informed consent.
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
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)
Read the detailed description
This is a randomized, placebo-controlled, single ascending-dose (SAD) study of RN0191 administered SC to adult subjects with elevated low-density lipoprotein-cholesterol. Subjects who have signed an Ethics Committee (EC)-approved informed consent form (ICF) and have met all the protocol eligibility criteria during screening may be enrolled into the study. The study only has one part: SAD cohorts with Sentinel Dosing
The starting dose for subjects in Cohort 1 will be 25 mg RN0191 (or placebo). The following are the planned dose levels for subsequent cohorts in the SAD phase:
- Cohort 2: 100 mg RN0191 or placebo
- Cohort 3: 300 mg RN0191 or placebo
- Cohort 4: 500 mg RN0191 or placebo
- Conditions
- To Reduce the LDL-C Level in Hypercholesteremia Adult Patients, Combination for Subgroup High LDL-c Patients With Other Comorbidities
- Being tested
- RN0191 INJECTION
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RN0191 INJECTIONDrug
a sterile solution in each bottle for subcutaneous (SC) injection
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 will be 25 mg RN0191 (or placebo) | Active comparator | Not described Receives: Drug: RN0191 INJECTION |
| Cohort 1 will be 100 mg RN0191 (or placebo) | Active comparator | Not described Receives: Drug: RN0191 INJECTION |
| Cohort 1 will be 300 mg RN0191 (or placebo) | Active comparator | Not described Receives: Drug: RN0191 INJECTION |
| Cohort 1 will be 500 mg RN0191 (or placebo) | Active comparator | Not described Receives: Drug: RN0191 INJECTION |
What is being measured
The primary outcome is the main question the study is designed to answer.
Exploratory objectives are presented in the body of the protocol.
- To determine the safety and tolerability of RN0191 administered as escalating single subcutaneously (SC) doses in adult subjects with elevated low-density lipoprotein-cholesterol
Measured: For each subject in the study, the duration of the study clinic visits is approximately 21 weeks from screening to Day 85/EOS examination.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2023
- Study start14 Jul 2023
- Primary completion24 Feb 2024
- Study completion14 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for To Reduce the LDL-C Level in Hypercholesteremia Adult PatientsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- This is a randomized, placebo-controlled, single ascending-dose (SAD) study of RN0191 administered SC to adult subjects with elevated low-density lipoprotein-cholesterol. Subjects who have signed an Ethics Committee (EC)-approved informed consent form (ICF) and have met all the protocol eligibility criteria during screening may be enrolled into the study. In this SAD study, adult subjects with elevated LDL-C will be enrolled in up to 4 cohorts. Each cohort will comprise 8 subjects randomized in a 3:1 ratio (1:1 ratio for sentinel subjects; 5:1 ratio for non-sentinel subjects) to receive a single dose of RN0191 (n=6) or placebo (n=2), respectively.
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ikaria Bioscience Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kristi McLendon, MD, Principal investigator, Nucleus Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05905068
- Sponsor's protocol ID
- RN0191-101
- Status last verified
- Oct 2024
- First submitted
- 2 Jun 2023
- Last updated
- 10 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05905068
- Acronym / sponsor ID
- RN0191-101
- Conditions
- To Reduce the LDL-C Level in Hypercholesteremia Adult Patients, Combination for Subgroup High LDL-c Patients With Other Comorbidities
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Ikaria Bioscience Pty LtdIndustry (pharma, biotech, devices)
- Study start
- 14 Jul 2023 (actual)
- Primary completion
- 24 Feb 2024 (actual)
- Study completion
- 14 May 2024 (actual)
- First posted
- 15 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Detailed description
This is a randomized, placebo-controlled, single ascending-dose (SAD) study of RN0191 administered SC to adult subjects with elevated low-density lipoprotein-cholesterol. Subjects who have signed an Ethics Committee (EC)-approved informed consent form (ICF) and have met all the protocol eligibility criteria during screening may be enrolled into the study. The study only has one part: SAD cohorts with Sentinel Dosing
The starting dose for subjects in Cohort 1 will be 25 mg RN0191 (or placebo). The following are the planned dose levels for subsequent cohorts in the SAD phase:
- Cohort 2: 100 mg RN0191 or placebo
- Cohort 3: 300 mg RN0191 or placebo
- Cohort 4: 500 mg RN0191 or placebo
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Single blind (participant)
- Number of arms
- 4
- Model description
- This is a randomized, placebo-controlled, single ascending-dose (SAD) study of RN0191 administered SC to adult subjects with elevated low-density lipoprotein-cholesterol. Subjects who have signed an Ethics Committee (EC)-approved informed consent form (ICF) and have met all the protocol eligibility criteria during screening may be enrolled into the study. In this SAD study, adult subjects with elevated LDL-C will be enrolled in up to 4 cohorts. Each cohort will comprise 8 subjects randomized in a 3:1 ratio (1:1 ratio for sentinel subjects; 5:1 ratio for non-sentinel subjects) to receive a single dose of RN0191 (n=6) or placebo (n=2), respectively.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 will be 25 mg RN0191 (or placebo) | Active comparator | Drug: RN0191 INJECTION |
| Cohort 1 will be 100 mg RN0191 (or placebo) | Active comparator | Drug: RN0191 INJECTION |
| Cohort 1 will be 300 mg RN0191 (or placebo) | Active comparator | Drug: RN0191 INJECTION |
| Cohort 1 will be 500 mg RN0191 (or placebo) | Active comparator | Drug: RN0191 INJECTION |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Male and female subjects, aged 18 to 60 years, inclusive.
- Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for females and >50 kg for males.
- Serum LDL-C ≥100mg/dL (2.6 mmol/L) at screening and Day -1.
- Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.
- 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)
Key exclusion criteria (9 listed)
- Male and female subjects, aged 18 to 60 years, inclusive.
- Body mass index between 18 and 32 kg/m2, inclusive, with body weight > 45 kg for females and >50 kg for males .
- Serum LDL-C ≥100mg/dL (2.6 mmol/L) at screening and Day -1.
- Fasting triglycerides < 400 mg/dL (<4.52 mmol/L) at screening and Day -1.
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RN0191 INJECTIONa sterile solution in each bottle for subcutaneous (SC) injection | Drug | Cohort 1 will be 100 mg RN0191 (or placebo), Cohort 1 will be 25 mg RN0191 (or placebo), Cohort 1 will be 300 mg RN0191 (or placebo), Cohort 1 will be 500 mg RN0191 (or placebo) |
Primary outcome
Exploratory objectives are presented in the body of the protocol.
Time frame: For each subject in the study, the duration of the study clinic visits is approximately 21 weeks from screening to Day 85/EOS examination.
• To determine the safety and tolerability of RN0191 administered as escalating single subcutaneously (SC) doses in adult subjects with elevated low-density lipoprotein-cholesterol
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Oct 2024.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kristi McLendon, MD principal investigator
Nucleus Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RN0191 INJECTION in people with To Reduce the LDL-C Level in Hypercholesteremia Adult Patients and Combination for Subgroup High LDL-c Patients With Other Comorbidities, run by Ikaria Bioscience Pty Ltd at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Exploratory objectives are presented in the body of the protocol. (time frame: For each subject in the study, the duration of the study clinic visits is approximately 21 weeks from screening to Day 85/EOS examination.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Oct 2024.