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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
Sponsor:
Information provided by (Responsible Party):
UniQure Biopharma B.V.

Brief 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®


Condition or disease Intervention/treatment
Lipoprotein Lipase Deficiency Familial Hyperlipoproteinemia Type 1 Familial Hyperchylomicronemia Other: Observational study

Detailed 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.

Study Type : Observational [Patient Registry]
Actual Enrollment : 16 participants
Observational Model: Other
Time Perspective: Prospective
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



Intervention Details:
  • Other: Observational study
    Post-Authorization Safety Study


Primary Outcome Measures :
  1. 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




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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients treated with GLYBERA®, either during their participation in a clinical trial or in the commercial setting till October 25th, 2017
Criteria

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:


Information from the National Library of Medicine

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


Locations
Germany
Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin
Berlin, Germany, 13353
Sponsors and Collaborators
UniQure Biopharma B.V.
Investigators
Study Chair: Maurizio Averna, Prof Universitaria Policlinico Paolo Giaccone, Palermo

Responsible Party: UniQure Biopharma B.V.
ClinicalTrials.gov Identifier: NCT03293810     History of Changes
Other Study ID Numbers: REG-uQ-Glyb-001
First Posted: September 26, 2017    Key Record Dates
Last Update Posted: February 16, 2018
Last Verified: February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Hyperlipoproteinemias
Hyperlipidemias
Hyperlipoproteinemia Type I
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn