Hyperlipoproteinemia Type I clinical trials
The registry lists 21 studies for Hyperlipoproteinemia Type I across more than 10 countries. 3 are recruiting or about to start. 18 of them are mainly about Hyperlipoproteinemia Type I (how this is decided). Per ClinicalTrials.gov, last updated 2026-06-05.
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Who runs them
Amsterdam Molecular Therapeutics (4) · Akcea Therapeutics (3) · Arrowhead Pharmaceuticals (3) · UniQure Biopharma B.V. (2) · University of Texas Southwestern Medical Center (1) · CENTOGENE GmbH Rostock (1) · Shanghai Jiao Tong University School of Medicine (1) · Ionis Pharmaceuticals, Inc. (1)
What is being tested here
Drugs under test in Hyperlipoproteinemia Type I studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Heparin | 1 | 0 | 1 |
| CS-121 | 1 | 0 | 1 |
| Plozasiran | 0 | 1 | 2 |
| Mycophenolic Acid | 0 | 2 | 2 |
| Cyclosporine | 0 | 2 | 2 |
| Orlistat | 0 | 0 | 1 |
| ISIS 304801 | 0 | 1 | 1 |
| Vupanorsen | 0 | 0 | 1 |
| Pradigastat | 0 | 0 | 1 |
| CAT-2003 | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Dec 2026Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency · Université de Sherbrooke
- Oct 2027CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia · Shanghai Jiao Tong University School of Medicine
Who these studies take
Of the 2 Hyperlipoproteinemia Type I studies recruiting now, 0 (0%) set no upper age limit and 2 (100%) stop at 75 or younger. 1 (50%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (50%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (50%) | 1 (50%) |
| 18 | 2 (100%) | 2 (100%) |
| 30 | 1 (50%) | 1 (50%) |
| 40 | 1 (50%) | 1 (50%) |
| 50 | 1 (50%) | 1 (50%) |
| 65 | 1 (50%) | 1 (50%) |
| 75 | 1 (50%) | 1 (50%) |
| 85 | 0 (0%) | 0 (0%) |
Across all 21 studies, recruiting or not: 48% stop at 75 or younger and 19% at 65; 29% take children; 0% are for women only and 0% for men only; 29% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 21 of 21. search them.
- Treatment Protocol of Plozasiran in Adults With High-Risk Severe Hypertriglyceridemia (SHTG) and in Adults and Adolescents With FCSAvailableNCT067964261 country
- Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase DeficiencyRecruitingNCT04227678Not applicable1 country
- CS-121 APOC3 Base Editing in Children and Adolescents With HyperchylomicronemiaRecruitingNCT07371767Early Phase 11 country
- Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)CompletedNCT05089084Phase 321 countries
- Study of ARO-APOC3 in Healthy Volunteers, Hypertriglyceridemic Patients and Patients With Familial Chylomicronemia Syndrome (FCS)CompletedNCT03783377Phase 13 countries
- Baby Detect : Genomic Newborn ScreeningCompletedNCT056874741 country
- Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)Approved for marketingNCT063602371 country
- Glybera Registry, Lipoprotein Lipase Deficient (LPLD) PatientsCompletedNCT032938101 country
- Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)WithdrawnNCT031988973 countries
- Orlistat for the Treatment of Type I HyperlipoproteinemiaCompletedNCT02767531Phase 21 country
- Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)No longer availableNCT03544060
- The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia SyndromeCompletedNCT02658175Phase 311 countries
- Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)CompletedNCT03360747Phase 21 country
- Safety, Tolerability,Pharmacokinetics(PK)and Pharmacodynamics(PD)Assessment of LCQ908 in Patients With Severe HypertriglyceridemiaCompletedNCT01146522Phase 1/21 country
- Lipoprotein Lipase Enzyme Activity Assay Validation and Clinical AssessmentCompletedNCT02656095
- Alipogene Tiparvovec for the Treatment of LPLD PatientsWithdrawnNCT02904772Phase 22 countries
- Pilot Study To Assess CAT-2003 in Patients With ChylomicronemiaCompletedNCT02098278Phase 21 country
- Duration of Effect of Alipogene Tiparvovec Treatment, Which Was Administered in Other StudiesTerminatedNCT014479011 country
- 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 StudiesStatus unknownNCT014485771 country
- Safety and Efficacy in LPL-Deficient Subjects of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein Lipase [S447X]Status unknownNCT01109498Phase 2/31 country
- Efficacy and Safety of Human Lipoprotein Lipase (LPL)[S447X] Expressed by an Adeno-Associated Viral Vector in LPL-deficient SubjectsCompletedNCT00891306Phase 2/31 country