Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients (GENIALL)
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|ClinicalTrials.gov Identifier: NCT03293810|
Recruitment Status : Active, not recruiting
First Posted : September 26, 2017
Last Update Posted : February 16, 2018
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®
|Condition or disease||Intervention/treatment|
|Lipoprotein Lipase Deficiency Familial Hyperlipoproteinemia Type 1 Familial Hyperchylomicronemia||Other: Observational study|
|Study Type :||Observational [Patient Registry]|
|Actual Enrollment :||16 participants|
|Target Follow-Up Duration:||15 Years|
|Official Title:||Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)|
|Study Start Date :||June 2014|
|Estimated Primary Completion Date :||February 2029|
|Estimated Study Completion Date :||June 2029|
- Other: Observational study
Post-Authorization Safety Study
- Long-term collection of Safety and Efficacy of GLYBERA®, as measured by collection of Adverse Events, Immunological responses and information on Pancreatitis-events [ Time Frame: 15 years ]
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03293810
|Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin|
|Berlin, Germany, 13353|
|Study Chair:||Maurizio Averna, Prof||Universitaria Policlinico Paolo Giaccone, Palermo|