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 type:
  Non-interventional study

Study drug and medical condition

• Name of medicine, other:
  Nexviazyme

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)
• 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 and event rate of participants with IARs Incidence rate and event rate of IARs Cumulative incidence of IARs leading to treatment discontinuation Incidence rate and event rate of medication errors Cumulative incidence of medication errors leading to AE/SAE/treatment discontinuation, Incidence of all AEs during study participation, including but not limited to AESIs 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/NAb 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), region (Europe versus rest of the world) and infusion setting (clinic/hospital versus home) will also be performed, given adequate representations in each stratum.