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Treatment Frequency Reduction in Pompe Disease
Sponsor: Erasmus Medical Center
Summary
The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg/kg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.
Official title: Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg/kg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)
Key Details
Gender
All
Age Range
50 Years - Any
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2024-10-01
Completion Date
2027-12-31
Last Updated
2024-08-28
Healthy Volunteers
No
Conditions
Interventions
Algucosidase alfa 20 mg/kg once every 4 weeks instead of once every 2 weeks
The interval of ERT with alglucosidase alfa will be extended from once every 2 weeks to once every 4 weeks. The dose of 20 mg/kg per infusion remains the same.