Condition (MeSH)
Homozygous Familial Hypercholesterolemia
ClinicalTrials.gov lists 67 trials for Homozygous Familial Hypercholesterolemia or a narrower condition beneath it.
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Trials per phase (log scale)
67 of the 67 trials are tagged with Homozygous Familial Hypercholesterolemia itself; the rest are tagged with a narrower condition beneath it.
When did Homozygous Familial Hypercholesterolemia trials start? (24)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 8
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 4 |
| 2025 | 4 |
| 2024 | 4 |
| 2023 | 4 |
| 2022 | 1 |
| 2021 | 1 |
| 2020 | 2 |
| 2019 | 8 |
| 2018 | 5 |
| 2017 | 4 |
Showing 10 of 24 earlier years.
No forecast is made for this condition: it averages 3.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 6 trials still running at the 2026-10-09 snapshot; registered median of 22 months across 47 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Homozygous Familial Hypercholesterolemia trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 1 |
| Phase 2 | 14 |
| Phase 2/3 | 4 |
| Phase 3 | 27 |
| Not applicable | 4 |
| Observational | 15 |
How many Homozygous Familial Hypercholesterolemia trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 11 |
| Active, not recruiting | 4 |
| Completed | 41 |
| Terminated | 4 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Homozygous Familial Hypercholesterolemia trials? (13)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Aegerion Pharmaceuticals, Inc. | 6 |
| Novartis Pharmaceuticals | 6 |
| Regeneron Pharmaceuticals | 5 |
| Kastle Therapeutics, LLC | 4 |
| Amgen | 3 |
Showing 5 of 13 sponsors.
Which drugs are being tested for Homozygous Familial Hypercholesterolemia? (9)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Lomitapide | 8 |
| Mipomersen | 4 |
| Evolocumab | 4 |
| Evinacumab | 4 |
| ROSUVASTATIN | 3 |
Showing 5 of 9 drugs.
Interventions as named in the registry (6)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Inclisiran (Leqvio®) 300mg | 5 |
| SHR-1918 | 3 |
| VSA003 | 1 |
| ER Niacin | 1 |
| Statin | 1 |
Showing 5 of 6 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Homozygous Familial Hypercholesterolemia trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Coronary artery disease | 147 | 9 |
| Angina pectoris | 117 | 8 |
| Acute myocardial infarction | 100 | 5 |
| COVID-19 | 98 | 1 |
| Pneumonia | 78 | 4 |
Showing 5 of 10 adverse events.
Which publications cover Homozygous Familial Hypercholesterolemia? (7)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, non-inferiority trial. | The lancet. Diabetes & endocrinology | 2025 | Yes |
| Efficacy, Safety, and Tolerability of Inclisiran in Patients With Homozygous Familial Hypercholesterolemia: Results From the ORION-5 Randomized Clinical Trial. | Circulation | 2024 | Yes |
| Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia. | Circulation | 2024 | Yes |
| Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. | Liver international : official journal of the International Association for the Study of the Liver | 2023 | Yes |
| Worldwide experience of homozygous familial hypercholesterolaemia: retrospective cohort study. | Lancet (London, England) | 2022 | Yes |
Showing 5 of 7 publications.
Prevalence (18)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Homozygous familial hypercholesterolemia | Point prevalence | 1-9 / 1 000 000 | Denmark | Not stated | Exact |
| Homozygous familial hypercholesterolemia | Point prevalence | 1-9 / 1 000 000 | Worldwide | Not stated | Exact |
| Homozygous familial hypercholesterolemia | Point prevalence | 1-9 / 1 000 000 | Denmark | Not stated | Exact |
| Homozygous familial hypercholesterolemia | Point prevalence | 1-9 / 1 000 000 | Netherlands | Not stated | Exact |
| Homozygous familial hypercholesterolemia | Point prevalence | 1-9 / 1 000 000 | Spain | Not stated | Exact |
Showing 5 of 18 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Homozygous familial hypercholesterolemia | 391665 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Homozygous familial hypercholesterolemia | 391665 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.