| Not Yet Recruiting | Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia NCT07473843 | Arrowhead Pharmaceuticals | Phase 3 |
| Not Yet Recruiting | Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolem NCT07133815 | Beijing Suncadia Pharmaceuticals Co., Ltd | Phase 2 |
| Recruiting | A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (Y NCT07037771 | Arrowhead Pharmaceuticals | Phase 3 |
| Completed | A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy a NCT06723652 | Beijing Suncadia Pharmaceuticals Co., Ltd | Phase 3 |
| Completed | Evaluate the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia NCT06009393 | Beijing Suncadia Pharmaceuticals Co., Ltd | Phase 2 |
| Recruiting | NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia NCT06125847 | First Affiliated Hospital Xi'an Jiaotong University | EARLY_Phase 1 |
| Completed | Safety and Effectiveness of Evinacumab for the Treatment of Homozygous Familial Hypercholesterolemia NCT05611528 | Daniel Gaudet | Phase 3 |
| Active Not Recruiting | Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) NCT05217667 | Arrowhead Pharmaceuticals | Phase 2 |
| Completed | Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterol NCT04233918 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH NCT04034485 | LIB Therapeutics LLC | Phase 3 |
| Completed | A Study to Evaluate Safety and Efficacy of IBI306, a PCSK9 Monoclonal Antibody in Chinese Subjects With Homozy NCT04031742 | Innovent Biologics (Suzhou) Co. Ltd. | Phase 2 / Phase 3 |
| Completed | A Study to Evaluate the Safety and Efficacy of the PCSK9 Inhibitor AK102 in Patients With HoFH NCT03933293 | Akeso | Phase 2 |
| Completed | Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C NCT03814187 | Novartis Pharmaceuticals | Phase 3 |
| Completed | A Study of Inclisiran in Participants With Homozygous Familial Hypercholesterolemia (HoFH) NCT03851705 | Novartis Pharmaceuticals | Phase 3 |
| Withdrawn | Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) NCT03455777 | Akcea Therapeutics | Phase 2 |
| Completed | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterol NCT03409744 | Regeneron Pharmaceuticals | Phase 3 |
| Unknown | HDL Acute Lipid Optimization in Homozygous Familial Hypercholesterolemia NCT03135184 | HDL Therapeutics | N/A |
| Completed | Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia NCT03399786 | Regeneron Pharmaceuticals | Phase 3 |
| Completed | Study of cardiovAscular Contrasted Phenotypes in Patients With FamIliaI hypercholesteRolemia NCT03234127 | Nantes University Hospital | N/A |
| Completed | Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH) NCT03156621 | Regeneron Pharmaceuticals | Phase 3 |
| Recruiting | HoFH, the International Clinical Collaborators Registry NCT04815005 | University of Pennsylvania | — |
| Completed | A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) NCT02963311 | The Medicines Company | Phase 2 |
| Withdrawn | Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterol NCT02765841 | Aegerion Pharmaceuticals, Inc. | Phase 3 |
| Withdrawn | Effects of Lomitapide on Carotid and Aortic Atherosclerosis NCT02399852 | Aegerion Pharmaceuticals, Inc. | — |
| Completed | Study to Evaluate the Effects of MBX-8025 in Patients With HoFH NCT02472535 | Gilead Sciences | Phase 2 |
| Completed | Study of REGN1500 in Participants With Homozygous Familial Hypercholesterolemia (HoFH) NCT02265952 | Regeneron Pharmaceuticals | Phase 2 |
| Recruiting | LOWER: Lomitapide Observational Worldwide Evaluation Registry NCT02135705 | Amryt Pharma | — |
| Terminated | Efficacy and Tolerability of Anacetrapib Added to Ongoing Lipid-Lowering Therapy in Adult Participants With Ho NCT01841684 | Merck Sharp & Dohme LLC | Phase 3 |
| Completed | Comparisons of Two Low-density Lipoprotein Apheresis Systems in Patients With Homozygous Familial Hypercholest NCT02286596 | Laval University | — |
| Completed | Trial Evaluating PCSK9 Antibody in Subjects With LDL Receptor Abnormalities NCT01588496 | Amgen | Phase 2 / Phase 3 |
| Completed | Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects NCT01412034 | Cerenis Therapeutics, SA | Phase 2 |
| Recruiting | The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository NCT01109368 | The Rogosin Institute | — |
| Completed | A Safety and Efficacy Study of AEGR-733 to Treat Homozygous Familial Hypercholesterolemia (FH) NCT00730236 | Aegerion Pharmaceuticals, Inc. | Phase 3 |
| Completed | Evaluation of the Safety, Tolerability and Efficacy of Ezetimibe on a Select Population of Filipinos With Hype NCT00704535 | Organon and Co | — |
| Completed | Safety, Tolerability and Efficacy of Microsomal Triglyceride Protein (MTP) Inhibitor NCT01556906 | Aegerion Pharmaceuticals, Inc. | Phase 2 |
| Completed | Gene Analysis and Treatment Optimization in Chinese Homozygous Familial Hypercholesterolemia NCT01878604 | Central South University | — |