← Eichor
Condition: Lipid Metabolism Inborn Errors Synced from ClinicalTrials.gov · July 28, 2026

Recruiting studies: Lipid Metabolism Inborn Errors

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Safety Evaluation of Gene Therapy Drug in the Treatment of Primary Hypertriglyceridemic Patients With Recurrent Pancreat
Phase 1 · 1 location(s) · GeneCradle Inc · NCT05860569A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeedin
Phase N/A · 1 location(s) · Amicus Therapeutics · NCT04252066Krabbe Disease Global Patient Registry
Phase N/A · 1 location(s) · State University of New York at Buffalo · NCT02993796Fabry Cardiomyopathy: Identification of Early Myocardial Structural and Tissue Abnormalities Using Multiparametric MRI
NA · 1 location(s) · University Health Network, Toronto · NCT04856059Screening for Familial Hypercholesterolemia in Children
NA · 1 location(s) · Centre Hospitalier Universitaire, Amiens · NCT06555120''Comparative Evaluation of Change in Bite Force and Levels of Bone Turnover Markers CTX and BALP in Hyperdivergent and
NA · 1 location(s) · Postgraduate Institute of Dental Sciences Rohtak · NCT064568272-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH)
Phase 2 · 1 location(s) · Vanderbilt University Medical Center · NCT04941599World Data on Ambroxol for Patients With GD and GBA Related PD
Phase N/A · 3 location(s) · Shaare Zedek Medical Center · NCT04388969Comparing Direct vs Indirect Methods for Cascade Screening
NA · 1 location(s) · University of Maryland, Baltimore · NCT05348564Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory
Phase 1 · 2 location(s) · UNC Lineberger Comprehensive Cancer Center · NCT03721068Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
Phase N/A · 7 location(s) · Chiesi Italia · NCT07109375A Long-term Follow-up Study of Gaucher Disease
Phase N/A · 1 location(s) · Duke University · NCT03190837Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT00344331Postprandial Fatty Acid Metabolism in Subjects With Lipoprotein Lipase Deficiency
NA · 1 location(s) · Université de Sherbrooke · NCT04227678Registry of Patients Diagnosed With Lysosomal Storage Diseases
Phase N/A · 1 location(s) · University of California, San Francisco · NCT05619900Gaucherite - A Study to Stratify Gaucher Disease
Phase N/A · 8 location(s) · Cambridge University Hospitals NHS Foundation Trust · NCT03240653A Dose-exploration Study of EDP167 in HoFH
Phase 2 · 1 location(s) · Eddingpharm (Zhuhai) Co., Ltd. · NCT07489209Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Phase N/A · 1 location(s) · Universitätsklinikum Hamburg-Eppendorf · NCT04613089UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)
Phase N/A · 1 location(s) · University of California, San Francisco · NCT06669949Lysosomal Acid Lipase (LAL) Deficiency Registry
Phase N/A · 104 location(s) · Alexion Pharmaceuticals, Inc. · NCT01633489IBI306 Monotherapy in Non-Familial Hypercholesterolemia and Mixed Hyperlipidemia
Phase 3 · 1 location(s) · Innovent Biologics (Suzhou) Co. Ltd. · NCT07473960Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Cor
Phase N/A · 13 location(s) · Fondazione SISA (Societa Italiana per lo Studio della Arteri · NCT07447648PMMHRI - Familial Hypercholesterolemia Registry
Phase N/A · 1 location(s) · Polish Mother Memorial Hospital Research Institute · NCT06571630FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
Phase N/A · 1 location(s) · xCures · NCT06539169Integrating Whole Genome Sequencing and Digital Twins Into the Management of Hypercholesterolemia in Emiratis
NA · 1 location(s) · Abu Dhabi Health Services Company · NCT06535542Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
Phase 2/Phase 3 · 10 location(s) · Chiesi Farmaceutici S.p.A. · NCT05710692Evaluation of HEArt invoLvement in Patients With FABRY Disease
Phase N/A · 1 location(s) · Wuerzburg University Hospital · NCT03362164The Spanish Familial Hypercholesterolaemia Cohort Study
Phase N/A · 1 location(s) · Fundación Hipercolesterolemia Familiar · NCT02693548
Look up another condition
Patient organization or foundation? You can brand this page for your community — your logo, your resources, same honest registry data, kept current daily. Ask about branding →

This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.