GENIALL
Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)
Sponsor study identifier
observational
16
1 country
1
Study Start
June 27, 2014
Actual (reported)Primary Completion
June 2023
Actual (reported)Study Completion
June 2023
Actual (reported)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®
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
June 2023
Actual (reported)Click on a node to explore related trials.
Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
June 27, 2014
Actual (reported)May 20, 2015
Actual (reported)September 26, 2017
Actual (reported)June 2023
Actual (reported)June 2023
Actual (reported)Long-term collection of Safety and Efficacy of GLYBERA®, as measured by collection of Adverse Events, Immunological responses and information on Pancreatitis-events
Adverse Events will be collected as reported by the patients during routine visits/contacts. Immunological responses defined as antibody formation and T-cell responses against the AAV1-capsid and against the LPLS447X transgene product, measured just before dosing and at 6 and 12 months post-dosing. Pancreatitis-events will be collected as reported by the patients during routine visits/contacts
15 years
Post-Authorization Safety Study
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Patients treated with GLYBERA®, either during their participation in a clinical trial or in the commercial setting till October 25th, 2017
Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin
Berlin, 13353, Germany
Maurizio Averna, Prof
Universitaria Policlinico Paolo Giaccone, Palermo
16 participants (actual enrollment reported by the registry).
First Submitted
May 20, 2015
First Posted
September 26, 2017
Study Start
June 27, 2014
Actual (reported)Contact the study team to confirm eligibility.
Primary Completion
June 2023
Actual (reported)Study Completion
June 2023
Actual (reported)Last Updated
November 27, 2023
Record last verified: 2023-11
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Source: ClinicalTrials.gov.
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