None listed
Conditions
Interventions
None listed
Sponsors
Sanofi-Aventis Korea
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Describe the inclusion criteria other than gender and age in detail. • The patient with Pompe disease who has been prescribed Nexviazyme® according to the approved indication of the product at the study institution. • The patient who consents to participate in the study by signing and dating the informed consent form (ICF) voluntarily by the patient or the patient's parent(s) or legal representative.
Exclusion criteria
Exclusion criteria: The patient who has contraindications to Nexviazyme® in the product information.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| SAEs/SADRs;Unexpected AEs/ADRs that are not reflected on the precaution in use;ADRs that are already known;Non-serious ADRs;Other safety related information | — |
Secondary
| Measure | Time frame |
|---|---|
| overall improvement of the subject;Forced Vital Capacity (FVC) % predicted;Maximum Inspiratory Pressure (MIP) and Maximum Expiratory Pressure (MEP) % predicted;6-Minute Walk Test (6MWT) distance (m) walked;lower extremity muscle strength (Hand-Held Dynamometer [HHD]);upper extremity muscle strength (HHD);motor function (Quick Motor Function Test [QMFT], Gross Motor Function Measure-88 [GMFM-88] or Gait, Stairs, Gower’s, and Chair Maneuver [GSGC]);health-related quality of life (19 years and older: Short Form-12 [SF-12] or EuroQoL in 5 dimensions [EQ-5D-5L]; younger than 19 years: Pediatric Quality of Life Inventory [PedsQL]);Patient-Reported Outcome [PRO] (Pompe Disease Symptom Scale/Pompe Disease Impact Scale [PDSS/PDIS] or Patient Global Impression of Change [PGIC] | — |
Countries
Korea, Republic of
Contacts
Public ContactHa Young Shin
Yonsei University Health System, Severance Hospital
Outcome results
None listed