Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients

Lipoprotein Lipase Deficiency Clinical Trial

— GENIALL  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03293810
• Other study ID #: REG-uQ-Glyb-001
• Status: Completed
• Start date: June 27, 2014
• Est. completion date: June 2023

Study information

• Verified date: November 2023
• Source: UniQure Biopharma B.V.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

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®

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: June 2023
• Est. primary completion date: June 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• All patients treated with GLYBERA®, either during their participation in a clinical trial or in the commercial setting till October 25th, 2017 (= expiration date of Marketing Authorization of GLYBERA®), and

• Who are currently participating in the LPLD Registry

Exclusion Criteria:

• None

Related Conditions & MeSH terms

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

Intervention

Other:
• Observational study
Post-Authorization Safety Study

Locations

Country	Name	City	State
Germany	Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin	Berlin

Sponsors (1)

Lead Sponsor	Collaborator
UniQure Biopharma B.V.

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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