NCT06575829View on ClinicalTrials.gov

Treatment Frequency Reduction in Pompe Disease

PHASE4Not yet recruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

October 1, 2024

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2027

Conditions
Pompe Disease (Late-onset)GAA DeficiencyGlycogen Storage Disease Type IIAcid Maltase Deficiency
Interventions
DRUG

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.

Sponsors
All Listed Sponsors
lead

Erasmus Medical Center

OTHER