Morquio A Registry Study (MARS)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prescription event monitoring

Study drug and medical condition

• Medicinal product name:
  VIMIZIM
• Study drug International non-proprietary name (INN) or common name:
  ELOSULFASE ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AB12) elosulfase alfa

  elosulfase alfa
• Medical condition to be studied:
  Mucopolysaccharidosis IV

Population studied

• Short description of the study population:
  All patients with a confirmed diagnosis of MPS IVA disease may be eligible to participate in this Registry. Patients are not required to receive Vimizim to be eligible to participate in this Registry.
• 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:
  419

Study design details

• Study design:
  This is a voluntary multicentre, multinational, observational Registry in patients diagnosed with MPS IVA.
• Main study objective:
  1. To characterize and describe the MPS IVA population as a whole, including the heterogeneity, progression, and natural history of MPS IVA. ; 2. To evaluate the long-term effectiveness and safety of Vimizim, including but not limited to the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status.
• Data analysis plan:
  Efficacy analysis will include all patients in the Efficacy Population, and will be conducted annually and over the duration of MARS. All efficacy variables will be summarized descriptively for baseline and post-baseline. When applicable for the specific efficacy variable, the change from the baseline to post-baseline (post–baseline at annual or semi-annual timepoints, depending on the specific efficacy variable), and/or its percent change will be summarized descriptively. The analyses of safety will include all patients in the Safety Population. Safety data, including vital signs, findings from physical examinations, concomitant medications, and other safety assessments, will be summarized descriptively. Incidence rate calculations will be completed. Where applicable, descriptive statistics will include the number of patients and mean, median, standard deviation, minimum, and maximum values for continuous variables and count and percent for categorical variables.