Comparison of inclisiran or alirocumab to standard therapy in pediatric heterozygous familial hypercholesterolemia (HeFH) – the head-to-head PICOLO-FH clinical trial
Key facts
- Where
- Gdansk és Lodz, LengyelországSee the 3 sites
- Phase
- 3. fázis
Where in Lengyelország
- Uniwersyteckie Centrum KliniczneGdansk, LengyelországTaking people
- Instytut Centrum Zdrowia Matki PolkiLodz, LengyelországTaking people
- Wojewodzki Specjalistyczny Szpital Im Dr Wl BieganskiegoLodz, LengyelországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Medical University Of Lodz
- Trial code
- EU 2024-514523-42-00
- Record updated
- 2026. október 30.
- Original description
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.Read full original descriptionShow less
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.
Inclusion: Obtain informed written consent for the patient to participate in the study, for genetic testing and for the processing of personal data; Age 8 - <18 years; LDL level from screening visit (V1): (a) LDL≥ 190 mg/dl (>4.921 mmol/L) regardless of family history or (b) LDL ≥160 mg/dl + positive family history (in first-degree relatives and/or siblings: LDL >190 mg/dl (>4.921 mmol/L) and/or with premorbid (i.e. men <55 yrs, women <60 yrs) atherosclerotic cardiovascular disease, and/or corneal stroma, and/or tendonitis) or (c) LDL ≥130 mg/dl + molecularly confirmed mutation in at least one parent and/or subject; Negative serum pregnancy test (beta-HCG) in menstruating girls; Consent to the use of contraceptive methods as described in the study protocol.
Exclusion: Any current treatment in another clinical trial or less than 30 days from the end of treatment in the other trial or 2x the half-life of the drug in the study; LDL - apheresis within the last month prior to inclusion in the study; Use of ‘ prohibited’ drugs (see section 7.2.6 of the protocol for details); Uncontrolled hypertension; Lipid disorders identified as secondary in the investigator's assessment due to: a. BMI ≥ 85th percentile according to the centile grids of Warsaw children (Palczewska-Niedźwiecka); b. poorly compensated diabetes mellitus, defined as HbA1c >8%; c. decompensated hypothyroidism; d. nephrotic syndrome e. anorexia f. liver dysfunction; g. other medical reasons; Fasting triglycerides > 350 mg/d (l>3.95 mmol/l); Chronic kidney disease (eGFR <30 ml/min/1.73m2); Body weight <23kg.