Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
NCT ID: NCT06597006
Last Updated: 2025-12-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
9 participants
INTERVENTIONAL
2025-02-28
2029-04-15
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Inclisiran
Year 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)
Inclisiran
Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 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.
Placebo
Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)
Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Interventions
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Inclisiran
Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 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.
Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* HoFH diagnosed by genetic confirmation
Exclusion Criteria
* On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)
* Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation
* Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.
* Documented evidence of a null (negative) mutation in both LDLR alleles
* Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
* History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \<15% reduction in LDL-C)
* Treatment with mipomersen or lomitapide (within 5 months of screening)
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Heterozygous familial hypercholesterolemia (HeFH)
* Body weight (at the screening and/or randomization (Day 1) visit) \<16 kg for participants 6 to \<12 years (at screening) or \<11 kg for participants 2 to \<6 years (at screening)
* 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
2 Years
11 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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UC San Francisco Medical Center
San Francisco, California, United States
UC San Francisco Medical Center
San Francisco, California, United States
Childrens National Hospital
Washington D.C., District of Columbia, United States
Washington Univ School Of Medicine
St Louis, Missouri, United States
Novartis Investigative Site
Vienna, , Austria
Novartis Investigative Site
Beijing, Beijing Municipality, China
Novartis Investigative Site
Frankfurt am Main, Hesse, Germany
Novartis Investigative Site
Ioannina, , Greece
Novartis Investigative Site
Kota Bharu, Kelantan, Malaysia
Novartis Investigative Site
Amsterdam, North Holland, Netherlands
Novartis Investigative Site
Bloemfontein, Free State, South Africa
Novartis Investigative Site
Taichung, , Taiwan
Novartis Investigative Site
Taipei, , Taiwan
Novartis Investigative Site
Adana, Saricam, Turkey (Türkiye)
Novartis Investigative Site
Ankara, Yenimahalle, Turkey (Türkiye)
Novartis Investigative Site
Izmir, , Turkey (Türkiye)
Novartis Investigative Site
Southampton, , United Kingdom
Countries
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Central Contacts
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Novartis Pharmaceuticals
Role: CONTACT
Facility Contacts
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Other Identifiers
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2024-514595-41
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
CKJX839C12304
Identifier Type: -
Identifier Source: org_study_id
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