For Healthcare Professionals

Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients

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About the study

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®
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

  1. 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
  2. Who are currently participating in the LPLD Registry

EXCLUSION CRITERIA

  1. None
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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.
Phone iconCall +31 20 240 6000Email iconEmail Study Center

Study’s details


Contition

Familial Hyperchylomicronemia,Familial Hyperlipoproteinemia Type 1,Lipoprotein Lipase Deficiency

Age (in years)

0 - 200

Participants needed

16

Est. Completion Date

Oct 2033

Treatment type

Observational [Patient Registry]


Sponsor

UniQure Biopharma B.V.

ClinicalTrials.gov identifier

NCT03293810

Study number

REG-uQ-Glyb-001

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