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Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients

NCT03293810 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 16

Last updated 2023-11-27

No results posted yet for this study

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®

Conditions

  • Lipoprotein Lipase Deficiency
  • Familial Hyperlipoproteinemia Type 1
  • Familial Hyperchylomicronemia

Interventions

OTHER

Observational study

Post-Authorization Safety Study

Sponsors & Collaborators

  • UniQure Biopharma B.V.

    lead INDUSTRY

Principal Investigators

  • Maurizio Averna, Prof · Universitaria Policlinico Paolo Giaccone, Palermo

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-06-27
Primary Completion
2023-06-30
Completion
2023-06-30

Countries

  • Germany

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03293810 on ClinicalTrials.gov