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Not Yet RecruitingNCT05017402

Higher Dose of Alglucosidase Alpha for Pompe Disease

Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study

Status
Not Yet Recruiting
Phase
Study type
Observational
Enrollment
36 (estimated)
Sponsor
Taipei Veterans General Hospital, Taiwan · Other Government
Sex
All
Age
0 Years – 60 Years
Healthy volunteers
Not accepted

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.

Conditions

Interventions

TypeNameDescription
DRUGAlglucosidase AlfaStandard dose: 20-25 mg/kg every other week; Higher dose: \>25 mg/kg every other week.

Timeline

Start date
2021-09-01
Primary completion
2023-12-31
Completion
2026-12-31
First posted
2021-08-23
Last updated
2021-08-23

Source: ClinicalTrials.gov record NCT05017402. Inclusion in this directory is not an endorsement.