Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients — GENIALL  

Lipoprotein Lipase Deficiency Clinical Trial

Official title: Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)

Status Completed

Clinical Trial Summary

Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive disorder, characterized by loss-of function mutations in the LPL gene, leading to the inability to produce functionally active lipoprotein lipase (LPL). LPL is the key enzyme in the metabolism of triglyceride (TG)-rich lipoproteins (chylomicrons (CM) and very low-density lipoproteins (VLDL)). LPLD results in extremely high concentrations of circulating TG-rich lipoproteins. No drug therapy for LPLD is currently available. Clinical management of LPLD patients consists of severe dietary fat restriction and the use of medium-chain triglycerides to substitute for normal dietary fats. Alipogene tiparvovec (Glybera®) received marketing authorisation from the European commission on 25 October 2012. Glybera® aims to correct lipoprotein lipase deficiency sufficiently to decrease the morbidity and lower the risk of inherent complications of LPLD, in adult patients genetically diagnosed with LPLD. The Glybera Registry is designed to collect the long-term safety and efficacy data of GLYBERA®

Clinical Trial Description

All patients treated with GLYBERA®, in a clinical trial and when GLYBERA® was commercially available who are currently participating in the LPLD Registry (Long term follow up of safety and efficacy in LPLD), will be asked to continue their participation in this Glybera Registry to collect long-term safety and efficacy data. ;

Related Conditions & MeSH terms

• Familial Hyperchylomicronemia
• Familial Hyperlipoproteinemia Type 1
• Hyperlipidemias
• Hyperlipoproteinemia Type I
• Hyperlipoproteinemias
• Lipoprotein Lipase Deficiency

• NCT number: NCT03293810
• Study type: Observational [Patient Registry]
• Source: UniQure Biopharma B.V.
• Status: Completed
• Start date: June 27, 2014
• Completion date: June 2023