PICOLO-FH trial
Project data
Project financing: 16 866 421,02 PLN, financed by the Medical Research Agency (ABM) - ABM/2023/1
Project description:
The clinical trial, with the acronym PICOLO-FH, is the world’s first multicentre study to compare the efficacy and safety of combination therapy using statin and alirocumab or statin and inclisiran against standard statin therapy in children diagnosed with heterozygous familial hypercholesterolaemia (heFH).
The study is a three-arm, parallel, randomised, head-to-head study with two experimental groups (arm I: rosuvastatin + alirocumab; arm II: rosuvastatin + inclisiran) and one comparison group (arm III: rosuvastatin). In the comparison group, the statin treatment used is now the standard of therapy for heFH in children according to EAS and PTL recommendations. The two experimental arms are a combination therapy of statin with inclisiran or alirocumab, which are not yet registered in children.
The mainstay of treatment for patients with heFH is the use of statins. Unfortunately, in patients with heFH, statin monotherapy seems to be an insufficient treatment, as despite their use, the risk of cardiovascular incidents is still increased several times in this group compared to the healthy population. The cause of heFH is loss-of-function mutations in the LDLR or apoB genes or gain-of-function mutations in PCSK9. Therefore, the use of a statin and a drug that inhibits the PCSK9 protein is reasonable and desirable in terms of the ethiopathogenesis of heFH. Currently, the only drug inhibiting the PCSK9 protein registered in children with heFH is evolocumab. However, despite its registration in the pediatric population with heFH and its proven clinical benefit, it is not used in this group due to its high price and low awareness among parents and physicians. The situation is different in adult patients with heFH, who can receive any of the three available PCSK9 protein inhibitor drugs free of charge in our country under the drug programme from November 2022.
The use of therapies planned in the study will ensure that therapeutic targets for LDL-C concentrations are already achieved in childhood and will reduce the duration of exposure to high LDL-C concentrations, resulting in a reduction in the occurrence of cardiovascular events.
As planned, the research will be carried out in two research centers in Poland: Regional Specialist Hospital of Wł. Biegański in Łódź and University Clinical Centre in Gdańsk.
- Beneficiary
Medical University of Lodz (Leader)
- Coordinating Principal Investigator
Marlena Broncel, MD, PhD