MPS VI Clinical Surveillance Program (CSP)

Study type

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

  Disease epidemiology

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational disease registry

Study drug and medical condition

• Name of medicine:
  NAGLAZYME
• Medical condition to be studied:
  Mucopolysaccharidosis VI

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:
  176

Study design details

• Main study objective:
  Characterize & describe the MPS VI population as a whole, Help the MPS VI medical community with development of recommendations for monitoring patients reports and optimize patient care, Evaluate long-term effectiveness and safety of Naglazyme, Determine presence of Naglazyme in the infants of treated mothers, characterize effects of Naglazyme in pts <5 yrs enrolled in CSP (1mg/kg at least 1 year)
• Data analysis plan:
  CSP data will be analyzed as per the program’s statistical analysis plan (SAP) and reported periodically. Physicians may obtain data on their individual patients and aggregate data on patients at their clinic. Longitudinal prospective and retrospective data may be collected. Demographic and baseline characteristics will be summarized. Frequencies will be presented for the categorical variables (eg, sex and race), and descriptive statistics will be presented for continuous variables (eg, height, weight, and age).