Question 1

Are there clinical trials for children with Familial Hypercholesterolemia (HeFH and HoFH), such as the ORION-20 (NCT06597019) and ORION-19 (NCT06597006) Inclisiran studies at UCSF?

Accepted Answer

Yes. At the UCSF Pediatric Lipid Clinic, Site Principal Investigator Dr. Martin Thelin, MD, PhD is actively recruiting children for two pivotal Phase 3 trials of Inclisiran (a twice-yearly PCSK9-targeted siRNA therapy): ORION-20 (NCT06597019) for children aged 6 to 11 years with Heterozygous FH (HeFH), and ORION-19 (NCT06597006) for children aged 2 to 11 years with Homozygous FH (HoFH). UCSF is also recruiting patients with Familial Chylomicronemia Syndrome (FCS) for the Ionis Olezarsen trial.

Question 2

When should a child be screened for high cholesterol, what are pediatric LDL goals, and is statin therapy safe for growing children?

Accepted Answer

All children should undergo universal lipid screening once between ages 9 and 11 and again between ages 17 and 21. If a parent has Familial Hypercholesterolemia (FH) or premature heart disease, cascade screening should begin at age 2. In children with FH, the clinical treatment target is LDL-C < 100–130 mg/dL, and decades of pediatric clinical trials show that FDA-approved statins started at ages 8–10 are safe and do not impair linear growth, puberty, or cognitive development.

clinical trials

Novartis (Inclisiran)

Ionis (Olezarsen)