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Comparison of inclisiran or alirocumab to standard therapy in pediatric heterozygous familial hypercholesterolemia (HeFH) – the head-to-head PICOLO-FH clinical trial
EU CTIS ID: 2024-514523-42-00
What this study is testing
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.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Obtain informed written consent for the patient to participate in the study, for genetic testing and for the processing of personal data.
- Age 10 years-15 years and 6 months.
- 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.
- Negative serum pregnancy test (beta-HCG) in menstruating girls.
- Consent to the use of contraceptive methods as described in the study protocol.
You likely can't join if
- 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).
- Uncontrolled hypertension.
- Chronic kidney disease (eGFR <30 ml/min/1.73m2).
- 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.
See the full eligibility criteria
- Obtain informed written consent for the patient to participate in the study, for genetic testing and for the processing of personal data.
- Age 10 years-15 years and 6 months.
- 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.
- Negative serum pregnancy test (beta-HCG) in menstruating girls.
- Consent to the use of contraceptive methods as described in the study protocol.
- 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).
- Uncontrolled hypertension.
- Chronic kidney disease (eGFR <30 ml/min/1.73m2).
- 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).
- Body weight <23kg
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.