1 published article · Updated continuously
12 trials tracked for Heterozygous Familial Hypercholesterolemia: 9 in phase 3 or 4 and 6 with published results. The most-cited published study has 822 citations.
Showing the 12 most-cited and recently-updated of 12 trials. Browse the full registry →
Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.
Several pharmacological interventions have demonstrated significant reductions in LDL-C for patients with Heterozygous Familial Hypercholesterolemia. Inclisiran demonstrated substantial and sustained reductions in LDL-C, with a -41.15% change at Day 510 1, a -50.02% reduction from the initial feeder study baseline 5, and a -27.14% reduction at Day 330 6. Additionally, Inclisiran was associated with significant decreases in PCSK9 levels 11.
Other established therapies include Bococizumab, which achieved a -54.2% reduction in LDL-C at Week 12 2, and Evolocumab, which showed a -44.53% reduction in LDL-C at Week 24 3. Lipid-lowering medications (LMT) including atorvastatin, simvastatin, or rosuvastatin resulted in a -48.7% reduction in LDL-C at Week 24 8. Mipomersen sodium also demonstrated a -28.02% reduction in LDL-C and a -26.31% reduction in Apolipoprotein B 9.
Clinical data also indicate that placebo groups showed a -53.7% reduction in calculated LDL-C (pre-apheresis) at Week 6, which was associated with a significant change in the standardized rate of apheresis treatments 4. Additionally, a fixed-dose combination of EZ and Atorva was evaluated, with 82.9% of participants experiencing at least one adverse event 7.
AI synthesis of 11 cited trials, updated Oct 6, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.
