Trials / Completed
CompletedNCT03293810
Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients
Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)
- Status
- Completed
- Phase
- —
- Study type
- Observational
- Enrollment
- 16 (actual)
- Sponsor
- UniQure Biopharma B.V. · Industry
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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®
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| OTHER | Observational study | Post-Authorization Safety Study |
Timeline
- Start date
- 2014-06-27
- Primary completion
- 2023-06-01
- Completion
- 2023-06-01
- First posted
- 2017-09-26
- Last updated
- 2023-11-27
Locations
1 site across 1 country: Germany
Source: ClinicalTrials.gov record NCT03293810. Inclusion in this directory is not an endorsement.