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Home › Conditions › familial hypercholesterolemia

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 · 86 cited
  3. Phase 3 Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies Completed · 52 cited
  4. Phase 3 Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) Completed · 49 cited
  5. Phase 3 Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies. Completed · 44 cited
  6. Phase 3 A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH) Completed · 44 cited
Show 12 more trials
  1. Phase 3 Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia Completed · 34 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.

What the trials found Updated — new results For clinicians

Familial hypercholesterolemia: what the trials found

Several pharmacological interventions have demonstrated significant reductions in LDL-C for patients with familial hypercholesterolemia. Enlicitide Decanoate showed a mean reduction of 58.2% at week 24 and 55.3% at week 52 1. Evolocumab achieved substantial reductions, including -36.01% in HeFH participants and -14.29% in HoFH participants by week 80 7, with additional trials showing significant LDL-C decreases of up to 55.24% at week 12 16.

Other established therapies include Obicetrapib, which demonstrated a reduction of approximately 36.05% in LDL-C over 180 days 8 and showed superior efficacy compared to both placebo and Ezetimibe monotherapy 6. Lomitapide resulted in an LDL-C reduction of 51.1% along with significant decreases in Total Cholesterol (43.9%) and Apolipoprotein B (60.9%) 9.

Standard lipid-lowering agents also showed efficacy: Atorvastatin reduced LDL-C by up to 44.887% 11, while Rosuvastatin achieved reductions of up to 50.2% in a 12-week period 12. Ezetimibe demonstrated an average reduction of 33.8% in LDL-C with low rates of discontinuation due to adverse events 10.

Non-pharmacological interventions, such as HELP Secura (apheresis treatment), are also established for this condition 3.

Recent results — preliminary, needs further review

  • Enclitide Chloride showed a reduction in LDL-C of up to 59.7% at week 8, though results are not yet corroborated 13.
  • Inclisiran demonstrated an average LDL-C reduction of approximately 46.74% and a significant decrease in PCSK9 levels (up to 71.80%) 14.
  • ALN-PCSSC showed reductions in LDL-C ranging from -35.5% to -49% at various intervals up to day 180 15.

For the clinician treating this condition

  • Enlicitide Decanoate and Evolocumab are established options for significant LDL-C reduction in both HeFH and HoFH populations.
  • Obicetrapib and Lomitapide provide robust reductions in LDL-C and associated markers like Apolipoprotein B.
  • Standard therapies including Atorvastatin, Rosuvastatin, and Ezetimibe remain established for managing lipid levels.

AI synthesis of 13 cited trials, updated Jun 26, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Narrative review on PCSK9 inhibitors for familial hypercholesterolemia
Drug Pipeline Sys. Review
Narrative review on PCSK9 inhibitors for familial hypercholesterolemia Review explores PCSK9 inhibitors for familial hypercholesterolemia
This narrative review synthesizes evidence on PCSK9 inhibitors, including evolocumab, for familial hypercholesterolemia. The authors discuss…
This review looks at PCSK9 inhibitors like evolocumab for people with familial hypercholesterolemia. It discusses how these drugs work and t…
Frontiers May 27, 2026
Lipid lowering therapy patterns in patients with ASCVD and familial hypercholesterolemia in Israel
Cardiology Cohort
Lipid lowering therapy patterns in patients with ASCVD and familial hypercholesterolemia in Israel Lipid lowering therapy patterns observed in Israeli patients
This retrospective database study examined lipid lowering therapy patterns among patients with atherosclerotic cardiovascular disease, ASCVD…
A look at how patients with high cholesterol and heart disease manage their lipid lowering therapy and treatment adherence in Israel.
Frontiers Apr 30, 2026

Research across Hyperlipidemia

Related studies from across the Hyperlipidemia family.

Inclisiran reduces LDL cholesterol by up to 54.7% in patients with atherosclerotic cardiovascular disease
Nephrology RCT
Inclisiran reduces LDL cholesterol by up to 54.7% in patients with atherosclerotic cardiovascular disease Trial shows consistent cholesterol reduction for heart disease patients
This Phase 3 randomized controlled trial evaluated inclisiran in 3,660 patients with heterozygous familial hypercholesterolemia and atherosc…
A large trial shows that a new injection can significantly lower bad cholesterol in people with high risk for heart disease.
Jul 4, 2026
Inclisiran plus usual care improves LDL-C goal attainment after acute coronary syndrome
Genetics & Precision Medicine RCT
Inclisiran plus usual care improves LDL-C goal attainment after acute coronary syndrome Inclisiran helps heart patients hit cholesterol goals fast
This phase 3b open-label RCT in 400 patients discharged after acute coronary syndrome found that adding inclisiran to usual care led to sign…
A new shot helped 3 in 4 recent heart attack patients reach LDL targets within 90 days, far more than usual care alone.
May 20, 2026
Exploratory analysis of RYGB and SG shows variable LDL cholesterol response in patients with obesity and high LDL cholesterol
Nutrition & Obesity Medicine RCT
Exploratory analysis of RYGB and SG shows variable LDL cholesterol response in patients with obesity and high LDL cholesterol Two bariatric surgeries lower cholesterol through completely different biological paths
This exploratory analysis of a randomized clinical trial (BASALTO) evaluated 30 patients with obesity and high LDL cholesterol undergoing Ro…
New research shows that two popular weight loss surgeries lower bad cholesterol in very different ways that could change how doctors pick a …
May 1, 2026
Meta-analysis shows dietary nitrate supplementation lowers systolic blood pressure and cholesterol in adults with obesity
Diabetes & Endocrinology Meta-analysis
Meta-analysis shows dietary nitrate supplementation lowers systolic blood pressure and cholesterol in adults with obesity Beetroot and Greens May Lower Blood Pressure and Cholesterol
This systematic review and meta-analysis evaluated dietary nitrate supplementation in adults with obesity.
Eating more nitrate-rich vegetables like beets and spinach may lower blood pressure and cholesterol in people with obesity, even if the scal…
Frontiers Apr 29, 2026
Narrative review proposes framework for TWAS signature-matching in drug prioritization
Genetics & Precision Medicine
Narrative review proposes framework for TWAS signature-matching in drug prioritization New method finds better drug targets using your DNA data
This narrative review and framework proposal examines the TWAS signature-matching approach with varying parameters, including TWAS method, e…
A new gene-based method can point doctors to better drugs, but the right settings are critical for accuracy.
medRxiv Apr 24, 2026
Bempedoic acid 180 mg/day reduces LDL-C in Japanese patients with hypercholesterolemia over 52 weeks
Drug Pipeline Phase III
Bempedoic acid 180 mg/day reduces LDL-C in Japanese patients with hypercholesterolemia over 52 weeks New Cholesterol Option Works Well for Statin Intolerant Patients
This multicenter, open-label, single-arm Phase 3 study evaluated bempedoic acid 180 mg/day in 130 Japanese patients with hypercholesterolemi…
A new cholesterol drug safely lowers bad cholesterol for one year in patients who cannot take standard statin medications.
Apr 17, 2026
Night shift work linked to elevated total cholesterol in male miners, cross-sectional analysis finds
Psychiatry Cohort
Night shift work linked to elevated total cholesterol in male miners, cross-sectional analysis finds Your Shift Work Raises Cholesterol Even If You Sleep Well
A cross-sectional analysis of 921 male underground miners in the Kailuan Group found night shift work (0:00–7:59) was associated with higher…
Night shift work raises cholesterol levels even when workers sleep well, proving that fixing your sleep isn't enough to protect your heart h…
Frontiers Apr 14, 2026
NHANES data show prevalence of high total cholesterol among US adults aged 20+
Infectious Disease
NHANES data show prevalence of high total cholesterol among US adults aged 20+ How many American adults have high cholesterol? A new look at national data
An observational analysis of NHANES 2015-2018 data examined the prevalence of high total cholesterol among US adults aged ≥20 years.
A new look at national data reveals how many American adults aged 20 and older have high total cholesterol, a silent driver of heart disease…
CDC Apr 6, 2026
Meta-analysis finds water-only fasting decreases HDL, increases LDL and total cholesterol
Diabetes & Endocrinology Meta-analysis
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.
Frontiers Apr 4, 2026
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…
CT.gov Apr 3, 2026

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 →