41 completed, 11 recruiting, 4 active not recruiting, 4 terminated. 31 Phase 3, 19 Phase 2, 4 Not applicable, 2 Phase 1, 1 Early Phase 1.
Follow Homozygous Familial Hypercholesterolemia: new trials by email Literature Review of Homozygous Familial Hypercholesterolemia trials Research Report on Homozygous Familial Hypercholesterolemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia Zodasiran - Homozygous Familial Hypercholesterolemia | Phase 3 | Recruiting | Arrowhead Pharmaceuticals | 10 | 2026-10-01 | 2026-10-26 |
| Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) Homozygous Familial Hypercholesterolemia (HoFH), Coronary Computed Tomography Angiography | Recruiting | Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) | 52 | 2026-04-30 | 2026-08-21 | |
| The ORIGIN-FH Study Screening for FH - Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH) | Not applicable | Recruiting | University of Wisconsin, Madison | 70 | 2026-02-14 | 2026-10-13 |
| A Dose-exploration Study of EDP167 in HoFH EDP167 - Homozygous Familial Hypercholesterolemia (HoFH) | Phase 2 | Recruiting | Eddingpharm (Zhuhai) Co., Ltd. | 20 | 2026-02-06 | 2026-05-20 |
| Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia SHR-1918 - Homozygous Familial Hypercholesterolemia | Phase 2 | Not yet recruiting | Beijing Suncadia Pharmaceuticals Co., Ltd | 55 | 2025-08-01 | 2025-08-21 |
| A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) zodasiran Injection, Placebo - Homozygous Familial Hypercholesterolemia | Phase 3 | Active not recruiting | Arrowhead Pharmaceuticals | 70 | 2025-06-17 | 2026-08-12 |
| Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia Inclisiran, Placebo - Familial Hypercholesterolemia - Homozygous | Phase 3 | Recruiting | Novartis Pharmaceuticals | 9 | 2025-02-28 | 2026-07-24 |
| A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia SHR-1918, SHR-1918 placebo - Homozygous Familial Hypercholesterolemia | Phase 3 | Completed | Beijing Suncadia Pharmaceuticals Co., Ltd | 55 | 2025-02-13 | 2025-12-15 |
| A Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of VSA003 in Chinese HoFH Patients VSA003, Placebo - Homozygous Familial Hypercholesterolemia (HoFH) | Phase 3 | Active not recruiting | Visirna Therapeutics HK Limited | 46 | 2024-11-27 | 2026-05-05 |
| Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE) | Active not recruiting | Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) | 73 | 2024-09-09 | 2026-02-20 | |
| A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias CTX310 - Cardiovascular, Metabolic Disease | Phase 1 | Recruiting | CRISPR Therapeutics AG | 90 | 2024-06-21 | 2026-09-18 |
| Specified Drug-use Survey of Leqvio for s.c. Injection. inclisiran - Familial Hypercholesterolaemia, Hypercholesterolaemia | Active not recruiting | Novartis Pharmaceuticals | 585 | 2024-03-04 | 2026-08-06 | |
| Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia SHR-1918 - Homozygous Familial Hypercholesterolemia | Phase 2 | Completed | Beijing Suncadia Pharmaceuticals Co., Ltd | 26 | 2023-12-22 | 2025-09-05 |
| NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia NGGT006 - Homozygous Familial Hypercholesterolemia | Early Phase 1 | Recruiting | First Affiliated Hospital Xi'an Jiaotong University | 21 | 2023-10-29 | 2026-09-03 |
| Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia Evinacumab - Homozygous Familial Hypercholesterolemia | Phase 3 | Completed | Daniel Gaudet | 10 | 2023-02-21 | 2025-03-25 |
| Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies Inclisiran - Heterozygous or Homozygous Familial Hypercholesterolemia | Phase 3 | Recruiting | Novartis Pharmaceuticals | 195 | 2023-02-10 | 2026-09-18 |
| Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) ARO-ANG 3 Injection - Homozygous Familial Hypercholesterolemia | Phase 2 | Terminated | Arrowhead Pharmaceuticals | 18 | 2022-04-22 | 2026-06-12 |
| Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia Inclisiran, Placebo - Familial Hypercholesterolemia - Homozygous | Phase 3 | Completed | Novartis Pharmaceuticals | 13 | 2021-02-16 | 2026-01-13 |
| Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) Lomitapide - Homozygous Familial Hypercholesterolaemia (HoFH) | Phase 3 | Completed | Amryt Pharma | 46 | 2020-12-14 | 2025-08-27 |
| Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia Evinacumab - Homozygous Familial Hypercholesterolemia | Phase 3 | Completed | Regeneron Pharmaceuticals | 20 | 2020-06-29 | 2024-07-16 |
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Hyperlipoproteinemia Type II (22) · Hypercholesterolemia (14) · Dyslipidemias (9) · Genetic Diseases, Inborn (8) · Lipid Metabolism Disorders (8) · Metabolic Diseases (8) · Hyperlipidemias (7) · Atherosclerosis (6) · Cardiovascular Diseases (6) · Hyperlipoproteinemias (4) · Lipid Metabolism, Inborn Errors (4) · Metabolism, Inborn Errors (4) · Congenital Abnormalities (3) · Infant, Newborn, Diseases (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (66) · Nutritional and Metabolic Diseases (66) · Hyperlipoproteinemias (63) · Lipid Metabolism, Inborn Errors (63) · Metabolism, Inborn Errors (63) · Hyperlipidemias (60) · Genetic Diseases, Inborn (59) · Lipid Metabolism Disorders (59) · Metabolic Diseases (59) · Dyslipidemias (58) · Hyperlipoproteinemia Type II (45) · Arterial Occlusive Diseases (6) · Arteriosclerosis (6) · Vascular Diseases (6) · Disease Attributes (3)
BMS201038 (8) · ALN-PCS (6) · evinacumab (5) · evolocumab (5) · Ezetimibe (4) · mipomersen (4) · Rosuvastatin Calcium (3) · alirocumab (3) · zodasiran (3) · Fatty Acids, Omega-3 (2) · Hydroxymethylglutaryl-CoA Reductase Inhibitors (2) · Niacin (2) · Saline Solution (2) · (2-methyl-4-(5-methyl-2-(4-trifluoromethyl-phenyl)-2H-(1,2,3)triazol-4-ylmethylsulfanyl)phenoxy)acetic acid (1) · Atorvastatin (1)
Aegerion Pharmaceuticals, Inc. (6) · Novartis Pharmaceuticals (6) · Regeneron Pharmaceuticals (5) · Kastle Therapeutics, LLC (4) · Amgen (3) · Arrowhead Pharmaceuticals (3) · Beijing Suncadia Pharmaceuticals Co., Ltd (3) · REGENXBIO Inc. (3) · Amryt Pharma (2) · AstraZeneca (2) · Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) (2) · Organon and Co (2) · University of Pennsylvania (2) · Akcea Therapeutics (1) · Akeso (1)
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Marina Cuchel, MD, PhD (4) · Mark Sumeray, MD (2) · Amy Peterson, MD, MS (1) · Andreas Birkenfeld, MD, PhD (1) · Bernd Hohenstein, MD, PhD (1) · Dan J Rader, MD (1) · David Pittrow, MD, PhD (1) · Elisabeth Steinhagen-Thiessen, MD, PhD (1) · Evan A Stein, MD PhD (1) · Janet Boylan (1)
PMID 33990512, PMID 36520008, PMID 24014831, PMID 27578134, PMID 11914243, PMID 17215532, PMID 20227758, PMID 20707601, PMID 24366918, PMID 25282520, PMID 25614280, PMID 25897792, PMID 26723464, PMID 28838366, PMID 29407879, PMID 29602595, PMID 30312929, PMID 30663562, PMID 31578082, PMID 32005605, PMID 32646561, PMID 32813947, PMID 33078867, PMID 33691480, PMID 35101175, PMID 35175352, PMID 36178485, PMID 36325897, PMID 37850379, PMID 37860863, PMID 38682459, PMID 38753448, PMID 39426393, PMID 39531353, PMID 39870096, PMID 40169529, PMID 40250039, PMID 40270128, PMID 40391436, PMID 40760109, PMID 41422812, PMID 41499141, PMID 42243546, PMID 42613196
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