A Post-Authorization Safety Study (PASS) to Assess Long-term Safety in Patients with Pompe Disease Treated with Avalglucosidase alfa in the Real-World Setting: the SAVANT (Safety assessment of AValglucosidase Alfa as a Novel Treatment) study

Study type

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

Study drug and medical condition

• Medicinal product name:
  NEXVIADYME
• Medicinal product name, other:
  Nexviadyme
• Study drug International non-proprietary name (INN) or common name:
  AVALGLUCOSIDASE ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB22) avalglucosidase alfa

  avalglucosidase alfa

Population studied

• Short description of the study population:
  Patients with Pompe Disease Treated with Avalglucosidase alfa
• 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)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  Evaluate the important identified risk of IARs including hypersensitivity and anaphylactic reactions
• Outcomes:
  - Incidence rate of participants with IARs (infusion associated reactions) including hypersensitivity and anaphylactic reactions at home setting and/or clinic/hospital setting; - Event rate of IARs including hypersensitivity and anaphylactic reactions at home setting and/or clinic/hospital setting; - Cumulative incidence of IARs leading to treatment discontinuation ; - Incidence rate and event rate of medication errors at home setting and/or clinic/hospital setting; - Cumulative incidence of medication errors leading to AE (adverse event)/SAE (serious adverse event)/treatment discontinuation at home setting and/or clinic/hospital setting; - Incidence of all AEs during study participation, including but not limited to AESIs (adverse events of special interest) and AEs leading to permanent treatment discontinuations; - Incidence of severe hypersensitivity reactions, anaphylactic reactions, and severe immune mediated reactions; - Incidence of AEs with permanent treatment discontinuation as outcome, ; - Incidence of ADA (antidrug antibody)/NAb (neutralizing antibodies) over time
• Data analysis plan:
  The primary analysis approach is descriptive, and the same approach will apply to both the primary and secondary endpoints. No hypothesis testing is planned. ; Stratification by prior exposure category (ie, avalglucosidase alfa use prior to enrollment, prior use of other ERTs [enzyme replacement therapy]), region (Europe versus rest of the world) and infusion setting (clinic/hospital versus home) will also be performed, given adequate representations in each stratum.