Lipid Metabolism, Inborn Errors clinical trials
The registry lists 24 studies for Lipid Metabolism, Inborn Errors across more than 10 countries. 3 are recruiting or about to start. 8 of them are mainly about Lipid Metabolism, Inborn Errors (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-04. Counting its 24 narrower conditions too, such as Fabry Disease: 1,040 studies, 215 open.
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Who runs them
Organon and Co (4) · Kastle Therapeutics, LLC (4) · Akcea Therapeutics (2) · CENTOGENE GmbH Rostock (2) · Oregon Health and Science University (1) · Rigshospitalet, Denmark (1) · Ionis Pharmaceuticals, Inc. (1) · NewAmsterdam Pharma (1)
What is being tested here
Drugs under test in Lipid Metabolism, Inborn Errors studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
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.
The study
- Jan 2027Screening for Disorders of Lipid Metabolism and Familial Hyperlipidemia in Children and Adolescents With Increased Cardiovascular Risk Due to Chronic Diseases Such as Congenital Heart Disease, Chronic Inflammatory Bowel Disease, Celiac Disease and Rheumatic Disorders · Alexandra Miriam Kiess
Who these studies take
Of the 2 Lipid Metabolism, Inborn Errors studies recruiting now, 0 (0%) set no upper age limit and 2 (100%) stop at 75 or younger. 2 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 1 (50%) | 1 (50%) |
| 18 | 1 (50%) | 1 (50%) |
| 30 | 0 (0%) | 0 (0%) |
| 40 | 0 (0%) | 0 (0%) |
| 50 | 0 (0%) | 0 (0%) |
| 65 | 0 (0%) | 0 (0%) |
| 75 | 0 (0%) | 0 (0%) |
| 85 | 0 (0%) | 0 (0%) |
Across all 24 studies, recruiting or not: 29% stop at 75 or younger and 25% at 65; 67% take children; 0% are for women only and 0% for men only; 17% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 24 of 24. search them.
- EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Not yet recruitingNCT07894887Phase 1/21 country
- Study of Zodasiran in Adolescent Participants With Homozygous Familial HypercholesterolemiaRecruitingNCT07473843Phase 33 countries
- Screening for Disorders of Lipid Metabolism and Familial Hyperlipidemia in Children and Adolescents With Increased Cardiovascular Risk Due to Chronic Diseases Such as Congenital Heart Disease, Chronic Inflammatory Bowel Disease, Celiac Disease and Rheumatic DisordersRecruitingNCT07716488Not applicable1 country
- Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTActive, not recruitingNCT05739643Phase 1/21 country
- Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.CompletedNCT05425745Phase 310 countries
- Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)Approved for marketingNCT063602371 country
- Mendelian Reverse Cholesterol Transport StudyTerminatedNCT01782027Not applicable1 country
- Sitosterolemia Extension Study (0653-004)(COMPLETED)CompletedNCT00092820Phase 3
- Sitosterolemia Extension Study (0653-003)(COMPLETED)CompletedNCT00092807Phase 3
- An Investigational Drug Study to Lower Non-Cholesterol Sterol Levels Associated With Sitosterolemia (0653-062)(COMPLETED)CompletedNCT00092898Phase 3
- Investigational Drug in Patients With Hypercholesterolemia or in Patients With Sitosterolemia (0653-026)(COMPLETED)TerminatedNCT00092833Phase 3
- Biomarker for Homozygous Familial Hypercholesterolemia (BioHoFH)WithdrawnNCT031988973 countries
- Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)No longer availableNCT03544060
- Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher DiseaseCompletedNCT024166617 countries
- Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)CompletedNCT03360747Phase 21 country
- Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT02112994Phase 215 countries
- Fat and Sugar Metabolism During Exercise in Patients With Metabolic MyopathyStatus unknownNCT02635269Not applicable1 country
- Dietary Cholesterol and Defects in Cholesterol Synthesis in Mevalonate Kinase DeficiencyTerminatedNCT002602991 country
- Measure Liver Fat Content After ISIS 301012 (Mipomersen) AdministrationCompletedNCT00362180Phase 21 country
- Open Label Extension of ISIS 301012 (Mipomersen) to Treat Familial HypercholesterolemiaCompletedNCT00477594Phase 21 country
- Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial HypercholesterolemiaCompletedNCT00607373Phase 37 countries
- An Open-label Extension Study to Assess the Long-term Safety and Efficacy of ISIS 301012 (Mipomersen) in Patients With Familial Hypercholesterolemia or Severe-HypercholesterolemiaCompletedNCT00694109Phase 37 countries
- Anti-oxLDL IgM Antibodies as a Novel Therapy for Metabolic Lipid DiseasesStatus unknownNCT02707211Early Phase 11 country
- Genetic Study of SitosterolemiaCompletedNCT000044811 country