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Familial hypercholesterolemia

Part of Hyperlipidemia

2 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials for familial hypercholesterolemia

18 trials tracked for familial hypercholesterolemia: 11 in phase 3 or 4 and 7 with published results. The most-cited published study has 100 citations.

18Trials tracked
11Phase 3 & 4
0Recruiting
7With published results
Phase distribution
Phase 4 1 Phase 3 10 Phase 2 4 Phase 1 1 Other / NA 2
  1. Phase 3 Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020) Completed · 100 cited
  2. Phase 3 Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies Completed · 79 cited
  3. Phase 3 Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies Completed · 48 cited
  4. Phase 3 Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) Completed · 48 cited
  5. Phase 3 Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies. Completed · 43 cited
  6. Phase 3 Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia Completed · 34 cited
Show 12 more trials
  1. Phase 3 A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH) Completed · 33 cited
  2. Phase 4 Low-Density Lipoprotein (LDL) Apheresis Using H.E.L.P. Therapy Completed
  3. Phase 3 A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653H in Japanese Participants With Hypercholesterolemia (MK-0653H-833) Completed
  4. Phase 3 Atorvastatin Three Year Pediatric Study Completed
  5. Phase 3 PLUTO: Pediatric Lipid-redUction Trial of rOsuvastatin Completed
  6. Phase 2 An Extension Trial of Inclisiran in Participants With Cardiovascular Disease and High Cholesterol Completed
  7. Phase 2 Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C) Completed
  8. Phase 2 Reduction of Low-Density Lipoprotein Cholesterol (LDL-C) With PCSK9 Inhibition in Heterozygous Familial Hypercholesterolemia Disorder Study Completed
  9. Phase 2 A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008) Completed
  10. Phase 1 Pediatric Reporting of Adult-Onset Genomic Results Completed
  11. N/A Amlodipine/Atorvastatin (Caduet®) Drug Use Investigation (Regulatory Post Marketing Commitment Plan) Completed
  12. N/A Evaluating the Efficacy of Pediatric Lipid Screening Alerts Completed

Showing the 18 most-cited and recently-updated of 18 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.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.

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Night shift work linked to elevated total cholesterol in male miners, cross-sectional analysis finds
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NHANES data show prevalence of high total cholesterol among US adults aged 20+
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An observational analysis of NHANES 2015-2018 data examined the prevalence of high total cholesterol among US adults aged ≥20 years.
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Meta-analysis finds water-only fasting decreases HDL, increases LDL and total cholesterol
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Meta-analysis finds water-only fasting decreases HDL, increases LDL and total cholesterol Does water-only fasting help your cholesterol? A new review suggests it might not
A systematic review and meta-analysis of 32 human studies examined water-only fasting effects on blood lipids.
Water-only fasting may lower good cholesterol and raise bad cholesterol, according to a new review of 32 human studies.
Phase 3 trial of oral PCSK9 inhibitor enlicitide in hypercholesterolemia reports no results
Diabetes & Endocrinology Phase III
Phase 3 trial of oral PCSK9 inhibitor enlicitide in hypercholesterolemia reports no results Can a new pill lower cholesterol as well as existing drugs?
A completed phase 3 randomized controlled trial in 301 adults with hypercholesterolemia compared oral PCSK9 inhibitor enlicitide to ezetimib…
A new cholesterol pill called enlicitide finished its final trial, but the results showing how well it lowers bad cholesterol and its safety…

Questions about familial hypercholesterolemia

What lipid lowering treatments are common for ASCVD patients with familial hypercholesterolemia in Israel?

Common lipid-lowering treatments for ASCVD patients with FH in Israel include high-intensity statins, ezetimibe, and PCSK9 inhibitors, though real-world data show many patients still have uncontrolled LDL-C.

Full answer →