Long-term efficacy and safety follow-up of MLD patients treated with atidarsagene autotemcel (LongTERM-MLD)

Study type

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

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational long term follow up

Study drug and medical condition

• Name of medicine:
  LIBMELDY
• Medical condition to be studied:
  Metachromatic leukodystrophy

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
• Estimated number of subjects:
  72

Study design details

• Main study objective:
  Evaluate the durability of clinical efficacy and long-term safety following treatment with Libmeldy.
• Data analysis plan:
  Full statistical methods are detailed in a separate statistical analysis plan (SAP).