Familial Hypercholesterolemia - HeterozygousPhase 3ORION-16NCT04652726
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
A Phase 3 study testing Inclisiran in people with Familial Hypercholesterolemia - Heterozygous, run by Novartis Pharmaceuticals at 59 sites in 26 countries.
Trial summary
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C). Read more about this study
- Who is it for?
- Ages 12 to 17 years
- Conditions the study is for
- Familial Hypercholesterolemia - Heterozygous
- What is being tested?
- Inclisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 141 participants
- Where
- 59 sitesin 26 countries
- Study dates
- 27 Jan 2021to 27 Nov 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 12 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
- Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
- On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening
Who cannot join
- Homozygous familial hypercholesterolemia (HoFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Recent and/or planned use of other investigational medicinal products or devices
Trial locations and contacts
59 sites in 26 countries.
59 sites in 26 countries
United States6 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Childrens Hospital Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Primary Children's Medical Center
Salt Lake City, Utah, 84113
Tucson Medical Center
Tucson, Arizona, 85712
Wake Forest U of Health Sciences
Winston-Salem, North Carolina, 27157
Spain5 sites
Hospital Abente y Lago
A Coruña, 15001
Hospital Reina Sofia
Córdoba, Andalusia, 14004
Hospital Virgen de la Vcitoria
Málaga, Andalusia, 29010
Hospital Central de Asturias
Oviedo, Principality of Asturias, 33011
Novartis Investigative Site
Pamplona, Navarre, 31008
Brazil4 sites
Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio
Fortaleza, Ceará, 60430275
Nucleo de Pesquisa Clinica do Rio Grande do Sul
Porto Alegre, Rio Grande do Sul, 90430-001
Setor de Lípides, Aterosclerose e Biologia
São Paulo, São Paulo, 04023-900
Heart Institute (InCOr) HCMFUSP
São Paulo, São Paulo, 05403 000
Italy4 sites
Novartis Investigative Site
Milan, MI, 20162
Novartis Investigative Site
Modena, MO, 41124
Novartis Investigative Site
Roma, RM, 00161
Novartis Investigative Site
Roma, RM, 00165
Turkey (Türkiye)4 sites
Novartis Investigative Site
Adana, 01330
Gazi University Medical Faculty
Ankara, 06500
Novartis Investigative Site
Istanbul, TUR, 34098
Novartis Investigative Site
Izmir, 35100
France3 sites
Novartis Investigative Site
Besançon, 25030
Novartis Investigative Site
Bron, 69677
Novartis Investigative Site
Toulouse, 31059
Germany3 sites
KKIM UK Frankfurt/Main
Frankfurt, 60590
Universitaetsklinikum Freiburg
Freiburg im Breisgau, 79106
Universitaetsmedizin Mannheim
Mannheim, Baden-Wurttemberg, 68305
Greece3 sites
Hippokrateion General Hospital of Athens Greece
Athens, 115 27
Metropolitan Hospital
Athens, 18547
University General Hospital of Ioannina
Ioannina, GR, 455 00
Russia3 sites
Institute of the complex problems of cardiovascular disease
Kemerovo, 650002
Novartis Investigative Site
Moscow, 127412
Institute of Internal Prev. Med.
Novosibirsk, 630090
South Africa3 sites
Novartis Investigative Site
Bloemfontein, Free State, 9301
Novartis Investigative Site
Cape Town, 7925
Novartis Investigative Site
Somerset West, Western Cape, 7130
Czechia2 sites
Novartis Investigative Site
Prague, 12808
Novartis Investigative Site
Prague, 150 06
Israel2 sites
Lipid Research
Jerusalem, 9112001
Lipids Center Sheba Medical Center, Israel
Ramat Gan, 52621
Netherlands2 sites
Novartis Investigative Site
Amsterdam, 1105 AZ
Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
Poland2 sites
Novartis Investigative Site
Gdansk, 80 952
Novartis Investigative Site
Lodz, 93-338
Taiwan2 sites
Far Eastern Memorial Hospital
New Taipei City, 22060
Taipei Veterans General Hospital
Taipei, 11217
Argentina1 site
Novartis Investigative Site
Formosa, Formosa Province, P3600
Slovenia1 site
University Medical Centre Ljubljana, Div. of Pediatric Dept. of Endocrinology, Diabetes and Metabolic Diseases
Ljubljana, 1000
About this study
Official title: Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).
Read the detailed description
This was a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.
- Being tested
- Inclisiran, Placebo
- Keywords
- Heterozygous familial hypercholesterolemia (HeFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InclisiranDrug
Also called KJX839
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
PlaceboDrug
Also called Saline solution
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
| Study group | Role | What they receive |
|---|---|---|
| Inclisiran | Experimental | Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630) Receives: Drug: Inclisiran |
| Placebo | Placebo comparator | Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
Measured: Baseline and Day 330
Secondary outcomes (26)
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330
Measured: Baseline, after Day 90 up to Day 330
Absolute Change in LDL-C From Baseline to up Day 330 (Part 1/Year 1)
Absolute change in LDL-C from baseline to Day 330.
Measured: Baseline and Day 330
Percent Change in Apo B From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in apolipoprotein B (Apo B) from baseline to Day 330.
Measured: Baseline and Day 330
Percent Change in Lp(a) From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in lipoprotein (a) [Lp(a)] from baseline to Day 330.
Measured: Baseline and Day 330
Percent Change in Non-HDL-C From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to Day 330.
Measured: Baseline and Day 330
Percent Change in Total Cholesterol From Baseline up to Day 330 (Part 1/Year 1)
Percentage change in total cholesterol from baseline to Day 330.
Measured: Baseline and Day 330
Percent Change in LDL-C From Baseline up to Day 720
Percentage change in LDL-C from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in LDL-C From Baseline up to Day 720
Absolute change in LDL-C from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Apo B From Baseline up to Day 720
Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Apo B From Baseline up to Day 720
Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Lp(a) From Baseline up to Day 720
Percentage change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Lp(a) From Baseline up to Day 720
Absolute change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Non-HDL-C From Baseline up to Day 720
Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Non-HDL-C From Baseline up to Day 720
Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Total Cholesterol From Baseline up to Day 720
Percentage change in total cholesterol from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Total Cholesterol From Baseline up to Day 720
Absolute change in total cholesterol from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Triglycerides From Baseline up to Day 720
Percentage change in triglycerides from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Triglycerides From Baseline up to Day 720
Absolute change in triglycerides from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in HDL-C From Baseline up to Day 720
Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in HDL-C From Baseline up to Day 720
Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in VLDL-C From Baseline up to Day 720
Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolut Change in VLDL-C From Baseline up to Day 720
Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in Apo A1 From Baseline up to Day 720
Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolute Change in Apo A1 From Baseline up to Day 720
Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Percent Change in PCSK9 From Baseline up to Day 720
Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Absolut Change in PCSK9 From Baseline up to Day 720
Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.
Measured: Baseline, up to Day 720
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2020
- Study start27 Jan 2021
- Primary completion9 Nov 2023
- Study completion27 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Familial Hypercholesterolemia - HeterozygousStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Parallel (Year 1) to single-group (Year 2)
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 141 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
- NCT04652726
- Sponsor's protocol ID
- CKJX839C12301
- Other IDs
- 2020-002757-18
- Acronym
- ORION-16
- Status last verified
- Dec 2025
- First submitted
- 2 Dec 2020
- Last updated
- 13 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Publications
Wiegman A, Peterson AL, Bruckert E, Defesche JC, Schweizer A, Bergeron J, Chueca MJ, Roeters van Lennep JE, Santos RD, Schwab KO, Lesogor A, Zhang S, Hegele RA. Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial. Lancet Diabetes Endocrinol. 2026 Mar;14(3):233-242. doi: 10.1016/S2213-8587(25)00351-1. Epub 2026 Jan 27.
PubMed 41616799Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
PubMed 35175352Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
PubMed 33990512
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
- ClinicalTrials.gov ID
- NCT04652726
- Acronym / sponsor ID
- ORION-16 / CKJX839C12301
- Keywords
- Heterozygous familial hypercholesterolemia (HeFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 141 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Jan 2021 (actual)
- Primary completion
- 9 Nov 2023 (actual)
- Study completion
- 27 Nov 2024 (actual)
- First posted
- 3 Dec 2020 (actual)
- Results
- Posted 10 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).
Detailed description
This was a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Parallel (Year 1) to single-group (Year 2)
| Arm | Type | Receives |
|---|---|---|
| Inclisiran | Experimental | Drug: InclisiranYear 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630) |
| Placebo | Placebo comparator | Drug: PlaceboYear 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630) |
Who may qualify
- Ages
- 12 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
- Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
- On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening
Key exclusion criteria (5 listed)
- Homozygous familial hypercholesterolemia (HoFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Recent and/or planned use of other investigational medicinal 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 |
|---|---|---|
| InclisiranAlso: KJX839Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection | Drug | Inclisiran |
| PlaceboAlso: Saline solutionSterile normal saline (0.9% sodium chloride in water for subcutaneous injection) | Drug | Placebo |
Primary outcome
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)
26 secondary outcomes
Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)
Time frame: Baseline, after Day 90 up to Day 330
Absolute Change in LDL-C From Baseline to up Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percent Change in Apo B From Baseline up to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percent Change in Lp(a) From Baseline up to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percent Change in Non-HDL-C From Baseline up to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percent Change in Total Cholesterol From Baseline up to Day 330 (Part 1/Year 1)
Time frame: Baseline and Day 330
Percent Change in LDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in LDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Apo B From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Apo B From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Lp(a) From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Lp(a) From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Non-HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Non-HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Total Cholesterol From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Total Cholesterol From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Triglycerides From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Triglycerides From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in HDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in VLDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolut Change in VLDL-C From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in Apo A1 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolute Change in Apo A1 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Percent Change in PCSK9 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Absolut Change in PCSK9 From Baseline up to Day 720
Time frame: Baseline, up to Day 720
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41616799Wiegman A, Peterson AL, Bruckert E, Defesche JC, Schweizer A, Bergeron J, Chueca MJ, Roeters van Lennep JE, Santos RD, Schwab KO, Lesogor A, Zhang S, Hegele RA. Efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolaemia (ORION-16): a two-part, randomised, multicentre clinical trial. Lancet Diabetes Endocrinol. 2026 Mar;14(3):233-242. doi: 10.1016/S2213-8587(25)00351-1. Epub 2026 Jan 27.
- Derived from this study: PubMed 35175352Reijman MD, Schweizer A, Peterson ALH, Bruckert E, Stratz C, Defesche JC, Hegele RA, Wiegman A. Rationale and design of two trials assessing the efficacy, safety, and tolerability of inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2022 Jul 20;29(9):1361-1368. doi: 10.1093/eurjpc/zwac025.
- Derived from this study: PubMed 33990512Warden BA, Duell PB. Inclisiran: A Novel Agent for Lowering Apolipoprotein B-containing Lipoproteins. J Cardiovasc Pharmacol. 2021 Aug 1;78(2):e157-e174. doi: 10.1097/FJC.0000000000001053.
- A Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- A Pediatric Plain Language Trial Summary is available on www.novctrd.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.
Related trials for Familial Hypercholesterolemia - Heterozygous
Active, not recruitingObservational study
Assessment of the Prevalence of Steatotic Liver Disease Associated With Metabolic Dysfunction in Patients With Heterozygous Familial Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia
Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
Recruiting
The ORIGIN-FH Study
Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH)
RecruitingPhase 2/3
A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)
Heterozygous Familial Hypercholesterolemia (HeFH)
Not yet recruitingPhase 2
A Study of VERVE-102 (LY4636581) in Adults With or at High Risk for Cardiovascular Disease
Atherosclerosis, Cardiovascular Disease
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
59 sites in 26 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Inclisiran in people with Familial Hypercholesterolemia - Heterozygous, run by Novartis Pharmaceuticals at 59 sites in 26 countries.
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 4 inclusion and 5 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 13 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in United States, Spain, Brazil, Italy, Turkey (Türkiye) and 21 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Jun 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1) (time frame: Baseline and Day 330). 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 NCT04652726, which also lists 3 linked publications.
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 13 Jan 2026.