- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03293810
Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients (GENIALL)
Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)
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®
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Berlin, Germany, 13353
- Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Sampling Method
Study Population
Description
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
Study Plan
How is the study designed?
Design Details
- Observational Models: Other
- Time Perspectives: Prospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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
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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
|
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Maurizio Averna, Prof, Universitaria Policlinico Paolo Giaccone, Palermo
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- REG-uQ-Glyb-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Amsterdam Molecular TherapeuticsThe Clinical Trial CompanyCompletedFamilial Lipoprotein Lipase DeficiencyCanada
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University of Colorado, DenverCompletedLipoprotein Lipase Deficiency
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