Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  Disease Monitoring Program

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This DMP is a global, prospective, multicenter, longitudinal protocol focused on clinical presentation, heterogeneity, and disease progression. This is not a randomized study and both treated and untreated patients will be enrolled.

Study drug and medical condition

• Medical condition to be studied:
  Mucopolysaccharidosis VII

Population studied

• Age groups:
  • Adolescents (12 to < 18 years)
  • Children (2 to < 12 years)
  • Infants and toddlers (28 days – 23 months)
  • Preterm newborn infants (0 – 27 days)
  • Term newborn infants (0 – 27 days)
  • 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:
  50

Study design details

• Main study objective:
  The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
• Outcomes:
  1. Clinical Course of MPS VII Disease 2. Long-term Effectiveness of Vestronidase Alfa 3. Long-term Safety of Vestronidase Alfa
• Data analysis plan:
  The Full Analysis Set (FAS) will include all enrolled patients. All analyses will be done based on the FAS. With the small sample size and the heterogeneity among patients with MPS VII, no formal statistical testing is planned. Descriptive summary statistics will be generated depending on the sample size.