Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
ORION-20
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 Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol
2 other identifiers
interventional
51
23 countries
66
Brief Summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Dec 2024
Typical duration for phase_3
66 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 11, 2024
CompletedFirst Posted
Study publicly available on registry
September 19, 2024
CompletedStudy Start
First participant enrolled
December 9, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 21, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
April 15, 2029
November 21, 2025
November 1, 2025
3.3 years
September 11, 2024
November 20, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage change in LDL-C from baseline to Day 330 (Year 1)
Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[percent change\] at Day 330
Baseline and Day 330
Secondary Outcomes (13)
Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)
Baseline, after Day 90 up to Day 330
Absolute change in LDL-C from baseline to Day 330 (Year 1)
Baseline and Day 330
Percent change in PCSK9 from baseline to Day 330 (Year 1)
Baseline and Day 330
Percent change in total cholesterol, non-HDL-C from baseline to Day 330 (Year 1)
Baseline and Day 330
Percent change in Apo B from baseline to Day 330 (Year 1)
Baseline and Day 330
- +8 more secondary outcomes
Study Arms (2)
Inclisiran
EXPERIMENTALYear 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)
Placebo
PLACEBO COMPARATORYear 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)
Interventions
Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Eligibility Criteria
You may qualify if:
- Male or female participants, 6 to \<12 years of age at screening
- HeFH diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
- For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
- Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.
You may not qualify if:
- Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Homozygous familial hypercholesterolemia (HoFH)
- Body weight \<16 kg at the screening and/or randomization (Day 1) visit
- Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome)
- Pregnant or nursing females
- Recent and/or planned use of other investigational medicinal products or devices
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (66)
UC San Francisco Medical Center
San Francisco, California, 94143, United States
UC San Francisco Medical Center
San Francisco, California, 94143, United States
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
Childrens National Hospital
Washington D.C., District of Columbia, 20010, United States
Excel Medical Clinical Trials LLC
Boca Raton, Florida, 33434, United States
Icahn School of Med at Mt Sinai
New York, New York, 10029, United States
Primary Childrens Medical Center
Salt Lake City, Utah, 84113, United States
Primary Childrens Medical Center
Salt Lake City, Utah, 84113, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
West Virginia Childrens Hospital
Morgantown, West Virginia, 26506, United States
West Virginia Childrens Hospital
Morgantown, West Virginia, 26506, United States
Novartis Investigative Site
Buenos Aires, C1245AAM, Argentina
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CABA, C1181ACH, Argentina
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Salzburg, 5020, Austria
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Vienna, 1090, Austria
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Brussels, 1200, Belgium
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Leuven, 3000, Belgium
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Fortaleza, Ceará, 60430-275, Brazil
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Rio de Janeiro, Rio de Janeiro, 20211-340, Brazil
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Porto Alegre, Rio Grande do Sul, 90020-020, Brazil
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São Paulo, São Paulo, 05403 000, Brazil
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Beijing, Beijing Municipality, 100013, China
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Shanghai, 200127, China
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Prague, 128 08, Czechia
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Prague, 150 06, Czechia
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Marseille, 13885, France
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Nantes, 44093, France
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Paris, 75012, France
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Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
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Frankfurt am Main, Hesse, 60590, Germany
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Hanover, 30173, Germany
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Athens, 115 27, Greece
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Ioannina, 455 00, Greece
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Hong Kong, 999077, Hong Kong
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Budapest, 1026, Hungary
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Jerusalem, 9112001, Israel
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Ramat Gan, 5265601, Israel
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Milan, MI, 20162, Italy
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Roma, RM, 00165, Italy
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Torino, TO, 10126, Italy
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Verona, VR, 3712, Italy
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Kuala Lumpur, Kuala Lumpur, 50586, Malaysia
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Amsterdam, North Holland, 1105 AZ, Netherlands
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Bialystok, 15-274, Poland
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Gdansk, 80 952, Poland
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Lodz, Łódź Voivodeship, 93-338, Poland
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Coimbra, 3000-602, Portugal
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Lisbon, 1649-035, Portugal
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Porto, 4050-651, Portugal
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Porto, 4200 319, Portugal
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Bloemfontein, Free State, 9301, South Africa
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Elche, Alicante, 03203, Spain
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Cadiz, Andalusia, 11009, Spain
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Esplugues, Barcelona, 08950, Spain
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Badalona, Catalonia, 08916, Spain
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Pamplona, Navarre, 31008, Spain
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Barcelona, 08041, Spain
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Málaga, 29011, Spain
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Seville, 41013, Spain
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Taipei, 111045, Taiwan
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Taipei, 11217, Taiwan
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Adana, Saricam, 01330, Turkey (Türkiye)
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Ankara, Yenimahalle, 06500, Turkey (Türkiye)
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Izmir, 35100, Turkey (Türkiye)
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West Midlands, Birmingham, B4 6NH, United Kingdom
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London, NW3 2QG, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Masking Details
- Masked (Year 1) to No Masking (Year 2)
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 11, 2024
First Posted
September 19, 2024
Study Start
December 9, 2024
Primary Completion (Estimated)
March 21, 2028
Study Completion (Estimated)
April 15, 2029
Last Updated
November 21, 2025
Record last verified: 2025-11
Data Sharing
- IPD Sharing
- Will share
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.