Glybera Registry, long-term safety and efficacy follow-up in Lipoprotein Lipase deficient (LPLD) patients treated with alipogene tiparvovec (GLYBERA®)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Disease epidemiology

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective, non-interventional, observational study

Study drug and medical condition

• Name of medicine:
  GLYBERA
• Medical condition to be studied:
  Lipoprotein deficiency

  Lipid metabolism disorder

  Hyperchylomicronaemia

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  16

Study design details

• Main study objective:
  To assess long-term safety of Glybera®; To assess the long-term clinical response of Glybera®; To assess the epidemiology of the disease and the demographics of LPLD patients
• Data analysis plan:
  The Glybera Registry data analyses are descriptive in nature, consistent with the main objectives. As such, there was no hypothesis-driven analysis and no need for Type I error adjustment.