A European, non-interventional, multicentre, registry-based post-authorisation safety study to evaluate the long-term safety of Loargys treatment in arginase 1 deficiency patients in standard clinical care

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  LOARGYS
• Study drug International non-proprietary name (INN) or common name:
  PEGZILARGINASE
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB24) pegzilarginase

  pegzilarginase
• Medical condition to be studied:
  Arginase deficiency

Population studied

• Short description of the study population:
  Adult and paediatric patients aged 2 years and older enrolled in the PASS with a confirmed diagnosis of ARG1-D and prescribed treatment with Loargys
• Age groups:
  • Paediatric Population (< 18 years)

    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)
• Estimated number of subjects:
  15

Study design details

• Study design:
  A non-interventional, non-comparative, multi-centre, prospective, registry-based PASS, conducted in collaboration with the E-IMD. It will be based on registry data using observational methods to collect uniform data prospectively in patients with ARG1-D to monitor the long-term safety of Loargys.
• Main study objective:
  The objective of the study is to evaluate the long-term safety of Loargys treatment in patients with ARG1-D.
• Setting:
  European E-IMD centres in countries where Loargys is available.
• Outcomes:
  • Occurrence of Adverse Events, Serious Adverse Events, Adverse Drug Reactions; • Occurrence of severe hypersensitivity reactions; • Occurrences of prolonged hypoargininaemia; • Occurrence of medication errors during non-healthcare professional administration; • Occurrence and clinical course of hypersensitivity reactions during non-healthcare professional administration; • Exposure and outcomes of pregnancy and lactation;