Cerliponase alfa Observational Study

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational

Study drug and medical condition

• Name of medicine:
  BRINEURA
• Study drug International non-proprietary name (INN) or common name:
  CERLIPONASE ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB17) cerliponase alfa

  cerliponase alfa
• Medical condition to be studied:
  Neuronal ceroid lipofuscinosis
• Additional medical condition(s):
  Neuronal ceroid lipofuscinosis type 2 (CLN2)

Population studied

• Age groups:
  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  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)
• Special population of interest:
  Hepatic impaired

  Immunocompromised

  Renal impaired
• Estimated number of subjects:
  45

Study design details

• Main study objective:
  To evaluate long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).
• Data analysis plan:
  All analysis will be descriptive. Assessments are collected based on the country, institution, and individual patient standard of care.