Testing two cholesterol drugs against standard treatment in children with familial high cholesterol

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children and teenagers aged 8 to under 18 with heterozygous familial hypercholesterolemia (high cholesterol that runs in families) and high LDL cholesterol.

Age

8 to under 18 years

Sex

Girls and boys can join. Girls who have periods must have a negative pregnancy test and agree to use contraception.

Disease stage

Heterozygous familial hypercholesterolemia with a high LDL level at screening: LDL 190 mg/dl (4.9 mmol/L) or more, or 160 mg/dl (4.1 mmol/L) or more with a positive family history, or 130 mg/dl (3.4 mmol/L) or more with a confirmed gene mutation in a parent or the child.

Earlier treatment

People cannot join if they had LDL apheresis in the last month, are in another clinical trial or less than 30 days from the end of one, or use prohibited drugs.

Biomarker or test result

LDL cholesterol at the screening visit: 190 mg/dl (4.9 mmol/L) or more, or 160 mg/dl (4.1 mmol/L) or more with a positive family history, or 130 mg/dl or more with a confirmed mutation in at least one parent or the child.

Main reasons for not taking part

• High cholesterol caused by something else, such as BMI at or above the 85th percentile, poorly controlled diabetes (HbA1c above 8% (64 mmol/mol)), decompensated hypothyroidism, nephrotic syndrome, anorexia or liver dysfunction
• Fasting triglycerides above 350 mg/d
• Uncontrolled high blood pressure
• Chronic kidney disease (eGFR below 30 ml/min/1.73m2)
• Body weight below 23kg (51 lb (23 kg))

The registry criteria, word for word

What the trial wants to find out

Assessment the efficacy of treatment with alirocumab (S.C.) and rosuvastatin (P.O.) or inclisiran (S.C.) and rosuvastatin (P.O.), compared with standard rosuvastatin (P.O.) therapy, in achieving the therapeutic goal of LDL-C <100 mg/dl after 104 weeks of treatment in paediatric population with heterozygous familial hypercholesterolaemia.