Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

16 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-06-27

Study Completion Date

2023-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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®

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Lipoprotein Lipase Deficiency Familial Hyperlipoproteinemia Type 1 Familial Hyperchylomicronemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Observational study

Post-Authorization Safety Study

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Maurizio Averna, Prof

Role: STUDY_CHAIR

Universitaria Policlinico Paolo Giaccone, Palermo

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Interdisciplinary Metabolism Center, Lipid Out-Patient-Clinic, Lipid Apheresis, Charité, University of Berlin

Berlin, , Germany

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Germany

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

REG-uQ-Glyb-001

Identifier Type: -

Identifier Source: org_study_id