A Prospective Safety Sub-Registry to Assess Anaphylaxis and Severe Allergic Reactions, and Severe Cutaneous and Systemic Immune-Mediated Reactions with Alglucosidase Alfa Treatment (Pompe Safety Sub-Registry)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

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

Study drug and medical condition

• Name of medicine:
  MYOZYME
• Name of medicine, other:
  Lumizyme
• Medical condition to be studied:
  Glycogen storage disease type II

Population studied

• Short description of the study population:
  At least 100 patients enrolled in the Pompe Registry at selected sites around the world who meet the inclusion criteria for this Safety Sub-Registry are eligible to participate in the Safety SubRegistry. This includes patients with onset of clinical signs/symptoms at ≤12 months of age (infantile-onset Pompe disease), as well as those with symptom onset at >12 months of age (lateonset Pompe disease). No single participating site is allowed to enroll more than 20% of the total Safety Sub-Registry patient population. Patients currently treated with alglucosidase alfa and treatment-naïve patients who initiate treatment at time of enrollment in the Safety Sub-Registry are targeted for enrollment at each site. ; INCLUSION CRITERIA; Patients must meet all of the following criteria to be eligible for inclusion in this Safety SubRegistry:; • be enrolled in the Pompe Registry;; • provide a signed Patient Information and Authorization form;; • have a confirmed diagnosis of Pompe disease (confirmation of diagnosis is defined as documented GAA enzyme deficiency from any tissue source and/or documentation of 2 GAA gene mutations);; • be naïve to and plan to be treated with alglucosidase alfa at or prior to enrollment, or are being treated with alglucosidase alfa. ; EXCLUSION CRITERIA ; Patients will be excluded if they have received an investigational drug (excluding alglucosidase alfa) within 30 days prior to signing a Safety Sub-Registry Patient Information and Authorization form, or if they are taking or plan to take any investigational product while enrolled in the Safety Sub-Registry.
• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 years)
    • Children (2 to < 12 years)
    • Infants and toddlers (28 days – 23 months)
    • Neonate

      • Term newborn infants (0 – 27 days)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with Glycogen storage disease type II/Pompe disease
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The objectives of this Safety Sub-Registry are to collect uniform and meaningful data on patients with Pompe disease who experience anaphylaxis, severe allergic reactions, and/or signals of severe cutaneous and/or systemic immune-mediated reactions following treatment with alglucosidase alfa.
• Outcomes:
  •the symptoms, severity, outcome, and occurrence of those adverse events (AEs, anaphylaxis, severe allergic reactions, and signals of severe cutaneous and systemic immune mediated reactions), •the effect of antibody responses and cross reacting immunologic material (CRIM) status (in patients with age at symptom onset less than or equal to 12 months only) on the occurrence of such AEs.
• Data analysis plan:
  Genzyme Registry staff will perform the statistical analysis of the data derived from the Registry, using the SAS® statistical software.