HyperlipidemiaPhase 1ORION-14NCT04774003
Study of Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inclisiran in Chinese Participants With Elevated Serum LDL-C
A Phase 1 study testing 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) and 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) in people with Hyperlipidemia, run by Novartis Pharmaceuticals at 3 sites in China.
Trial summary
Study to evaluate the pharmacokinetics and pharmacodynamics of inclisiran treatment given as single subcutaneous injection in Chinese participants with elevated low-density lipoprotein cholesterol (LDL-C) despite treatment with LDL-C lowering therapies Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hyperlipidemia
- What is being tested?
- 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) and 300 mg inclisiran sodium (equivalent to 284 mg inclisiran)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 3 sitesChina
- Study dates
- 26 Feb 2021to 18 Oct 2021
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent must be obtained before any assessment is performed.
- Male or female participants ≥ 18 years of age at screening
- Participants should meet fasting serum LDL-C ≥ 100 mg/dL (≥ 2.6 mmol/L) at screening
- Participants should meet fasting triglyceride < 400 mg/dL (< 4.52 mmol/L) at screening
- Participants should be receiving a maximally tolerated dose of statin#.
- For all participants, all the lipid-lowering therapy/ies (such as but not limited to statins and/or ezetimibe) should have remained stable (stable dose and no medication change) for ≥ 30 days before screening with no planned medication or dose change during study participation. #Maximum tolerated dose was defined as the maximum dose of statin that could be taken on a regular basis without intolerable AEs.
- Participants not receiving statin must have a documented evidence of intolerance to all doses of at least 2 different statins (or the corresponding local definition of complete intolerance to statins)
Who cannot join
- Participants diagnosed with any of following: homozygous familial hypercholesterolemia, New York Heart Association class III \& IV heart failure, Type 2 diabetes, severe hypertension, active liver disease, HIV infection or any uncontrolled or serious disease;
- History of drug abuse or unhealthy alcohol use, malignancy of any organ system, or or allergy to the investigational compound/compound class;
- Major adverse cardiovascular event within 3 months prior to randomization;
- Calculated glomerular filtration rate ≤30 mL/min by estimated glomerular filtration rate (eGFR) using standardized clinical methodology;
- Use of other investigational drugs or planned use of other investigational products or devices;
- Women of child-bearing potential unless they are using basic methods of contraception during dosing of investigational drug (total abstinence, sterilization, barrier methods, hormonal contraception, intrauterine device);
- Treatment with monoclonal antibodies inhibiting PCSK9 within 90 days prior to screening.
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
3 sites in 1 country.
China3 sites
Novartis Investigative Site
Beijing, 100029
Novartis Investigative Site
Changsha, Hunan, 410003
Novartis Investigative Site
Chengdu, Sichuan, 610041
About this study
Official title: A Placebo-controlled, Participant, Investigator and Sponsor Blinded, Randomized Study to Evaluate the Pharmacokinetics and Pharmacodynamics of Inclisiran Treatment Given as Single Subcutaneous Injection in Chinese Participants With Elevated Low-density Lipoprotein Cholesterol (LDL-C) Despite Treatment With LDL-C Lowering Therapies (ORION-14)
Study to evaluate the pharmacokinetics and pharmacodynamics of inclisiran treatment given as single subcutaneous injection in Chinese participants with elevated low-density lipoprotein cholesterol (LDL-C) despite treatment with LDL-C lowering therapies
Read the detailed description
The purpose of the study is to characterize pharmacokinetics, pharmacodynamics, safety and tolerability of inclisiran in Chinese participants with elevated serum LDL-C to support inclisiran registration in China.Inclisiran is a long acting RNA therapeutic agent that inhibits the synthesis of PCSK9, leading to reduced circulating LDL-C levels. Three pivotal Phase III studies have been conducted primarily in non-Asian participants to support New Drug Application/Marketing Authorization Application approval of inclisiran globally. This study design is based on the hypothesis that the global inclisiran clinical data primarily obtained in Caucasian participants could be appropriately extrapolated to Chinese participants.
- Conditions
- Hyperlipidemia
- Being tested
- 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran), Placebo, 300 mg inclisiran sodium (equivalent to 284 mg inclisiran)
- Keywords
- Inclisiran, KJX839, LDL-C, Pharmacokinetics and pharmacodynamics, Chinese participants with elevated serum LDL-C
- Medical subject headings
- Hyperlipidemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran)Drug
Also called KJX839
100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) in 0.5 mL solution. Subcutaneous administration at Day 1
PlaceboDrug
Inclisiran sodium 0mg (equivalent to inclisiran 0 mg) in 1.5 mL solution. Subcutaneous administration at Day 1
300 mg inclisiran sodium (equivalent to 284 mg inclisiran)Drug
Also called KJX839
300 mg inclisiran sodium (equivalent to 284 mg inclisiran) in 1.5 mL solution. Subcutaneous administration at Day 1
| Study group | Role | What they receive |
|---|---|---|
| 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) | Experimental | 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) x 1 dose (n=15) at Day 1 Receives: Drug: 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) |
| 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) | Experimental | 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) x 1 dose (n=15) at Day 1 Receives: Drug: 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) |
| Placebo | Placebo comparator | Placebo x 1 dose (n=10) at Day 1 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
PK parameters (Cmax) maximum peak observed plasma inclisiran concentration in treated participants
Pharmacokinetics parameters of inclisiran
Measured: 0-48 hours post-dose
PK parameters (Tmax) time to reach maximum peak plasma inclisiran concentration in treated participants
Pharmacokinetics parameters of inclisiran
Measured: 0-48 hours post-dose
PK parameters (T1/2) the elimination half-life associated with the terminal slope of a semi-logarithmic concentration-time curve in inclisiran treated participants
Pharmacokinetics parameters of inclisiran
Measured: 0-48 hours post-dose
PK parameters (AUC) area under the plasma concentration-time curve in inclisiran treated participants
Pharmacokinetics parameters of inclisiran
Measured: 0-48 hours post-dose
Percentage change in Proprotein convertase subtilisin kexin 9 (PCSK9) from baseline overtime
Pharmacodynamics effects of inclisiran
Measured: Baseline to Days 5, 8, 15, 30, 60 and 90
Percentage change in Low density lipoprotein cholesterol (LDL-C) from baseline overtime
Pharmacodynamics effects of inclisiran
Measured: Baseline to Days 5, 8, 15, 30, 60 and 90
Secondary outcomes (3)
Percent change from baseline to Days 30, 60 and 90 in PD parameter Proprotein convertase subtilisin kexin 9 (PCSK9)
Pharmacodynamics differences between inclisiran and placebo
Measured: Baseline to Days 30, 60 and 90
Percent change from baseline to Days 30, 60 and 90 in PD parameter Low density lipoprotein cholesterol (LDL-C)
Pharmacodynamics differences between inclisiran and placebo
Measured: Baseline to Days 30, 60 and 90
Rate of formation of anti-drug antibodies to Inclisiran
Immunogenicity of inclisiran
Measured: Baseline, Days 30 and 90
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2021
- Study start26 Feb 2021
- Primary completion18 Oct 2021
- Study completion18 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HyperlipidemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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
- Parallel groups
- Model description
- This is a placebo-controlled study of inclisiran sodium given as a single subcutaneous injection of either 100 mg and 300 mg.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774003
- Sponsor's protocol ID
- CKJX839A12105
- Acronym
- ORION-14
- Status last verified
- Mar 2025
- First submitted
- 18 Feb 2021
- Last updated
- 25 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Luo Z, Huang Z, Sun F, Guo F, Wang Y, Kao S, Yang G, Huang J, Li J, Zhao S, He Y. The clinical effects of inclisiran, a first-in-class LDL-C lowering siRNA therapy, on the LDL-C levels in Chinese patients with hypercholesterolemia. J Clin Lipidol. 2023 May-Jun;17(3):392-400. doi: 10.1016/j.jacl.2023.04.010. Epub 2023 Apr 29.
PubMed 37164838
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo-controlled, Participant, Investigator and Sponsor Blinded, Randomized Study to Evaluate the Pharmacokinetics and Pharmacodynamics of Inclisiran Treatment Given as Single Subcutaneous Injection in Chinese Participants With Elevated Low-density Lipoprotein Cholesterol (LDL-C) Despite Treatment With LDL-C Lowering Therapies (ORION-14)
- ClinicalTrials.gov ID
- NCT04774003
- Acronym / sponsor ID
- ORION-14 / CKJX839A12105
- Conditions
- Hyperlipidemia
- Keywords
- Inclisiran, KJX839, LDL-C, Pharmacokinetics and pharmacodynamics, Chinese participants with elevated serum LDL-C
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 26 Feb 2021 (actual)
- Primary completion
- 18 Oct 2021 (actual)
- Study completion
- 18 Oct 2021 (actual)
- First posted
- 26 Feb 2021 (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.
Study to evaluate the pharmacokinetics and pharmacodynamics of inclisiran treatment given as single subcutaneous injection in Chinese participants with elevated low-density lipoprotein cholesterol (LDL-C) despite treatment with LDL-C lowering therapies
Detailed description
The purpose of the study is to characterize pharmacokinetics, pharmacodynamics, safety and tolerability of inclisiran in Chinese participants with elevated serum LDL-C to support inclisiran registration in China.Inclisiran is a long acting RNA therapeutic agent that inhibits the synthesis of PCSK9, leading to reduced circulating LDL-C levels. Three pivotal Phase III studies have been conducted primarily in non-Asian participants to support New Drug Application/Marketing Authorization Application approval of inclisiran globally. This study design is based on the hypothesis that the global inclisiran clinical data primarily obtained in Caucasian participants could be appropriately extrapolated to Chinese participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- This is a placebo-controlled study of inclisiran sodium given as a single subcutaneous injection of either 100 mg and 300 mg.
| Arm | Type | Receives |
|---|---|---|
| 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) | Experimental | Drug: 300 mg inclisiran sodium (equivalent to 284 mg inclisiran)300 mg inclisiran sodium (equivalent to 284 mg inclisiran) x 1 dose (n=15) at Day 1 |
| 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) | Experimental | Drug: 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran)100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) x 1 dose (n=15) at Day 1 |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo x 1 dose (n=10) at Day 1 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Written informed consent must be obtained before any assessment is performed.
- Male or female participants ≥ 18 years of age at screening
- Participants should meet fasting serum LDL-C ≥ 100 mg/dL (≥ 2.6 mmol/L) at screening
- Participants should meet fasting triglyceride < 400 mg/dL (< 4.52 mmol/L) at screening
- Participants should be receiving a maximally tolerated dose of statin#.
Key exclusion criteria (8 listed)
- Participants diagnosed with any of following: homozygous familial hypercholesterolemia, New York Heart Association class III \& IV heart failure, Type 2 diabetes, severe hypertension, active liver disease, HIV infection or any uncontrolled or serious disease;
- History of drug abuse or unhealthy alcohol use, malignancy of any organ system, or or allergy to the investigational compound/compound class;
- Major adverse cardiovascular event within 3 months prior to randomization;
- Calculated glomerular filtration rate ≤30 mL/min by estimated glomerular filtration rate (eGFR) using standardized clinical methodology;
- Use of other investigational drugs or planned use of other investigational products or devices;
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 |
|---|---|---|
| 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran)Also: KJX839100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) in 0.5 mL solution. Subcutaneous administration at Day 1 | Drug | 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) |
| PlaceboInclisiran sodium 0mg (equivalent to inclisiran 0 mg) in 1.5 mL solution. Subcutaneous administration at Day 1 | Drug | Placebo |
| 300 mg inclisiran sodium (equivalent to 284 mg inclisiran)Also: KJX839300 mg inclisiran sodium (equivalent to 284 mg inclisiran) in 1.5 mL solution. Subcutaneous administration at Day 1 | Drug | 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) |
Primary outcome
PK parameters (Cmax) maximum peak observed plasma inclisiran concentration in treated participants
Time frame: 0-48 hours post-dose
Pharmacokinetics parameters of inclisiran
PK parameters (Tmax) time to reach maximum peak plasma inclisiran concentration in treated participants
Time frame: 0-48 hours post-dose
Pharmacokinetics parameters of inclisiran
PK parameters (T1/2) the elimination half-life associated with the terminal slope of a semi-logarithmic concentration-time curve in inclisiran treated participants
Time frame: 0-48 hours post-dose
Pharmacokinetics parameters of inclisiran
PK parameters (AUC) area under the plasma concentration-time curve in inclisiran treated participants
Time frame: 0-48 hours post-dose
Pharmacokinetics parameters of inclisiran
Percentage change in Proprotein convertase subtilisin kexin 9 (PCSK9) from baseline overtime
Time frame: Baseline to Days 5, 8, 15, 30, 60 and 90
Pharmacodynamics effects of inclisiran
Percentage change in Low density lipoprotein cholesterol (LDL-C) from baseline overtime
Time frame: Baseline to Days 5, 8, 15, 30, 60 and 90
Pharmacodynamics effects of inclisiran
3 secondary outcomes
Percent change from baseline to Days 30, 60 and 90 in PD parameter Proprotein convertase subtilisin kexin 9 (PCSK9)
Time frame: Baseline to Days 30, 60 and 90
Percent change from baseline to Days 30, 60 and 90 in PD parameter Low density lipoprotein cholesterol (LDL-C)
Time frame: Baseline to Days 30, 60 and 90
Rate of formation of anti-drug antibodies to Inclisiran
Time frame: Baseline, Days 30 and 90
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 25 Mar 2025.
- Derived from this study: PubMed 37164838Luo Z, Huang Z, Sun F, Guo F, Wang Y, Kao S, Yang G, Huang J, Li J, Zhao S, He Y. The clinical effects of inclisiran, a first-in-class LDL-C lowering siRNA therapy, on the LDL-C levels in Chinese patients with hypercholesterolemia. J Clin Lipidol. 2023 May-Jun;17(3):392-400. doi: 10.1016/j.jacl.2023.04.010. Epub 2023 Apr 29.
- A Plain Language Trial Summary is available on novctrd.comLink provided by the sponsor.
- Results for CKJX839A12105 from the Novartis Clinical Trials WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Hyperlipidemia
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A Study of Inclisiran to Prevent Cardiovascular Events in High-risk Primary Prevention Patients.
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RecruitingObservational study
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 100 mg inclisiran sodium (equivalent to 94.5 mg inclisiran) and 300 mg inclisiran sodium (equivalent to 284 mg inclisiran) in people with Hyperlipidemia, run by Novartis Pharmaceuticals at 3 sites in China.
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 7 inclusion and 8 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 25 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. 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. Some participants in this study may receive a placebo. 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?
PK parameters (Cmax) maximum peak observed plasma inclisiran concentration in treated participants (time frame: 0-48 hours post-dose). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04774003, which also lists 1 linked publication.
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 25 Mar 2025.