NCT03293810: GENIALL

Glybera Registry, Long-term Safety and Efficacy Follow-up in Lipoprotein Lipase Deficient (LPLD) Patients Treated With Alipogene Tiparvovec (GLYBERA®)

Completed Last updated 23 November 2023

What this trial tests

trial testing Observational study in Lipoprotein Lipase Deficiency in 16 participants. Completed in 1 June 2023.

Timeline

27 June 2014

Primary endpoint
1 June 2023

1 June 2023

Quick facts

Lead sponsor	UniQure Biopharma B.V.
Status	Completed
Study type	OBSERVATIONAL
Enrollment	16
Start date	27 June 2014
Primary completion	1 June 2023
Estimated completion	1 June 2023
Sites	1 location across Germany

Drugs / interventions tested

• Observational study — full drug profile →

Conditions studied

• Lipoprotein Lipase Deficiency — all drugs for Lipoprotein Lipase Deficiency →
• Familial Hyperlipoproteinemia Type 1 — all drugs for Familial Hyperlipoproteinemia Type 1 →
• Familial Hyperchylomicronemia — all drugs for Familial Hyperchylomicronemia →

Sponsor

UniQure Biopharma B.V. — full company profile →

Who can join

Eligibility, any sex, with Lipoprotein Lipase Deficiency or Familial Hyperlipoproteinemia Type 1. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• 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

Sponsor's own description

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®

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

1. Nanodelivery of nucleic acids.
   Mendes BB, Conniot J, Avital A, Yao D, et al · · 2022 · cited 401× · PMID 35480987 · DOI 10.1038/s43586-022-00104-y
2. Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology.
   Weng Y, Huang Q, Li C, Yang Y, et al · · 2020 · cited 72× · PMID 31927331 · DOI 10.1016/j.omtn.2019.12.004
3. Barriers to Treg therapy in Europe: From production to regulation.
   Hennessy C, Deptula M, Hester J, Issa F. · · 2023 · cited 11× · PMID 36744143 · DOI 10.3389/fmed.2023.1090721