11 completed, 2 recruiting, 2 unknown, 2 withdrawn. 7 Phase 2, 4 Phase 3, 2 Phase 1, 1 Early Phase 1, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 7 | Completed 5, Unknown 1, Withdrawn 1 |
| Phase 3 | 4 | Completed 3, Unknown 1 |
| Phase 1 | 2 | Completed 2 |
| Early Phase 1 | 1 | Recruiting 1 |
| Not applicable | 1 | Recruiting 1 |
Follow Hyperlipoproteinemia Type I: new trials by email Literature Review of Hyperlipoproteinemia Type I trials Research Report on Hyperlipoproteinemia Type I
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 |
|---|---|---|---|---|---|---|
| CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia CS-121 - Hyperchylomicronemia | Early Phase 1 | Recruiting | Shanghai Jiao Tong University School of Medicine | 15 | 2026-01-26 | 2026-01-28 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) Plozasiran, Placebo - Familial Chylomicronemia | Phase 3 | Completed | Arrowhead Pharmaceuticals | 75 | 2021-12-14 | 2026-06-05 |
| Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency Heparin, liquid meal - Lipoprotein Lipase Deficiency | Not applicable | Recruiting | Université de Sherbrooke | 16 | 2019-12-09 | 2026-02-10 |
| Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS) ARO-APOC3, sterile normal saline (0.9% NaCl) - Hypertriglyceridemia, Familial Chylomicronemia | Phase 1 | Completed | Arrowhead Pharmaceuticals | 112 | 2019-03-08 | 2025-12-24 |
| Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH) Lipoprotein Lipase Deficiency, Inborn Error of Lipid Metabolism | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) AKCEA-ANGPTL3-LRx - Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | Phase 2 | Completed | Akcea Therapeutics | 3 | 2017-12-21 | 2021-01-07 |
| Alipogene Tiparvovec for the Treatment of LPLD Patients alipogene tiparvovec, Prednisolone, Cyclosporins - LPL Deficiency | Phase 2 | Withdrawn | UniQure Biopharma B.V. | 2016-10-01 | 2017-08-17 | |
| Lipoprotein Lipase Enzyme Activity Assay Validation and Clinical Assessment Heparin - Lipoprotein Lipase Deficiency | Completed | University of Colorado, Denver | 12 | 2016-03-01 | 2019-07-11 | |
| The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome Volanesorsen - Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency | Phase 3 | Completed | Akcea Therapeutics | 68 | 2015-12-23 | 2021-08-26 |
| Orlistat for the Treatment of Type I Hyperlipoproteinemia Orlistat - Hyperlipoproteinemia Type I, Hypertriglyceridemia | Phase 2 | Completed | University of Texas Southwestern Medical Center | 2 | 2015-12-01 | 2023-02-08 |
| Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients Observational study - Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1 | Completed | UniQure Biopharma B.V. | 16 | 2014-06-27 | 2023-11-27 | |
| Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia CAT-2003, Placebo - Lipoprotein Lipase Deficiency, Familial, Hyperlipoproteinemia Type I | Phase 2 | Completed | Catabasis Pharmaceuticals | 13 | 2014-03-01 | 2016-07-21 |
| Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other Studies Hyperlipoproteinemia Type I | Terminated | Amsterdam Molecular Therapeutics | 19 | 2011-09-01 | 2015-03-30 | |
| Study to Re-assess and Re-confirm Data Previously Recorded About the Incidence and Severity of Acute Abdominal "Pancreatitis" Episodes in Lipoprotein Lipase Deficient (LPLD) Subjects Previously Enrolled on AMT Clinical Studies Lipoprotein Lipase Deficiency | Unknown | Amsterdam Molecular Therapeutics | 22 | 2010-11-01 | 2011-10-07 | |
| Safety, Tolerability,Pharmacokinetics(PK)and Pharmacodynamics(PD)Assessment of LCQ908 in Patients With Severe Hypertriglyceridemia LCQ908, Placebo - Hyperlipoproteinemia | Phase 1 / Phase 2 | Completed | Novartis Pharmaceuticals | 8 | 2010-05-01 | 2020-12-17 |
| Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient Subjects Alipogene Tiparvovec (AMT-011), Human LPL [S447X], mycophenolate mofetil, cyclosporine - Familial Lipoprotein Lipase Deficiency | Phase 2 / Phase 3 | Completed | Amsterdam Molecular Therapeutics | 5 | 2009-02-01 | 2011-09-29 |
| Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X] Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Mycophenolate mofetil, cyclosporine - Familial Lipoprotein Lipase Deficiency | Phase 2 / Phase 3 | Unknown | Amsterdam Molecular Therapeutics | 14 | 2007-08-01 | 2011-09-30 |
| Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) Volanesorsen - Familial Chylomicronemia | No longer available | Akcea Therapeutics | 2021-09-16 | |||
| Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCS Plozasiran - Familial Chylomicronemia, High Risk Severe Hypertriglyceridemia (SHTG) | Available | Arrowhead Pharmaceuticals | 2026-03-03 |
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Familial hyperchylomicronemia syndrome (4) · Hyperlipoproteinemias (4) · Hypertriglyceridemia (4) · Lipid Metabolism, Inborn Errors (4) · Dyslipidemias (3) · Genetic Diseases, Inborn (3) · Hyperlipidemias (3) · Lipid Metabolism Disorders (3) · Metabolic Diseases (3) · Metabolism, Inborn Errors (3) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (1) · 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency (1) · Acidemia, isovaleric (1) · Acyl-CoA Dehydrogenase Family, Member 9, Deficiency of (1)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (21) · Nutritional and Metabolic Diseases (21) · Dyslipidemias (18) · Genetic Diseases, Inborn (18) · Hyperlipidemias (18) · Lipid Metabolism Disorders (18) · Metabolic Diseases (18) · Metabolism, Inborn Errors (18) · Hyperlipoproteinemias (17) · Lipid Metabolism, Inborn Errors (17) · Digestive System Diseases (2) · Eye Diseases (2) · Pancreatic Diseases (2) · Abnormalities, Multiple (1) · Adrenal Gland Diseases (1)
Alipogene tiparvovec (3) · Mycophenolic Acid (3) · Cyclosporine (2) · Heparin (2) · ISIS 304801 (2) · plozasiran (2) · Cyclosporins (1) · IONIS-ANGPTL3-LRx (1) · Methylprednisolone (1) · Methylprednisolone Hemisuccinate (1) · Observation (1) · Orlistat (1) · Prednisolone (1) · olezarsen (1) · pradigastat (1)
Amsterdam Molecular Therapeutics (4) · Akcea Therapeutics (3) · Arrowhead Pharmaceuticals (3) · UniQure Biopharma B.V. (2) · CENTOGENE GmbH Rostock (1) · Catabasis Pharmaceuticals (1) · Centre Hospitalier Universitaire de Liege (1) · Ionis Pharmaceuticals, Inc. (1) · Novartis Pharmaceuticals (1) · Shanghai Jiao Tong University School of Medicine (1) · University of Colorado, Denver (1) · University of Texas Southwestern Medical Center (1) · Université de Sherbrooke (1)
Canada (12) · United States (6) · Germany (4) · Australia (2) · Belgium (2) · France (2) · Israel (2) · New Zealand (2) · Spain (2) · Argentina (1) · Austria (1) · Brazil (1) · China (1) · Croatia (1) · India (1)
Abhimanyu Garg, MD (1) · Daniel Gaudet, MD PhD (1) · Daniel Gaudet, MD, Ph.D. (1) · Daniel Gaudet, MD, PhD (1) · Laurent Servais (1) · Maurizio Averna, Prof (1) · Peter Bauer, Prof. (1) · Robert Eckel, MD (1)
PMID 22438229, PMID 22717743, PMID 25889044, PMID 29659879, PMID 38320498, PMID 39225259, PMID 41923352
2015 (1), UL1TR001082 (1)