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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 (2 to less than 12 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-19)

Status
Recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2024-514595-41-00
Acronym
CKJX839C12304
Enrollment
6
Registered
2025-01-21
Start date
2025-08-05
Completion date
Unknown
Last updated
2025-10-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

homozygous familial hypercholesterolemia

Brief summary

Percentage change in LDL-C from baseline to Day 330 (Year 1)

Detailed description

Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1), Percent change and absolute change in LDL-C, PCSK9, total cholesterol, Apo B, non-HDL-C, Lp(a), triglycerides, HDL-C, VLDL-C and Apo A1 from baseline to each assessment time up to Day 720 (Year 2), Incidence, severity and relationship to study drug of treatment-emergent AEs and SAEs; vital signs; laboratory parameters; ADA measurement; growth (height, weight, BMI); pubertal development (sexual hormones and Tanner staging)

Interventions

DRUGKJX839
DRUGPlacebo to KJX839 (Inclisiran) 0 mg/1.5 mL Solution for injection in vial

Sponsors

Novartis Pharma AG
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
Percentage change in LDL-C from baseline to Day 330 (Year 1)—

Secondary

MeasureTime frame
Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1), Percent change and absolute change in LDL-C, PCSK9, total cholesterol, Apo B, non-HDL-C, Lp(a), triglycerides, HDL-C, VLDL-C and Apo A1 from baseline to each assessment time up to Day 720 (Year 2), Incidence, severity and relationship to study drug of treatment-emergent AEs and SAEs; vital signs; laboratory parameters; ADA measurement; growth (height, weight, BMI); pubertal development (sexual hormones and Tanner staging)—

Countries

Austria, France, Germany, Greece, Netherlands, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026