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NOT YET RECRUITING
NCT05017402
Higher Dose of Alglucosidase Alpha for Pompe Disease
Sponsor: Taipei Veterans General Hospital, Taiwan
View on ClinicalTrials.gov
Summary
This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.
Official title: Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study
Key Details
Gender
All
Age Range
0 Years - 60 Years
Study Type
OBSERVATIONAL
Enrollment
36
Start Date
2021-09-01
Completion Date
2026-12-31
Last Updated
2021-08-23
Healthy Volunteers
No
Conditions
Interventions
DRUG
Alglucosidase Alfa
Standard dose: 20-25 mg/kg every other week; Higher dose: \>25 mg/kg every other week.