Trial Profile

Pompe Disease Registry

Status: Recruiting

Phase of Trial: Phase IV

Latest Information Update: 23 May 2025

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At a glance

Drugs Alglucosidase alfa (Primary)
Indications Glycogen storage disease type II
Focus Therapeutic Use
Sponsors Sanofi Genzyme

Most Recent Events

18 Apr 2024 Results(As of August 4, 2023, n=145 )characterizing the demographic and clinical characteristics of infantile-onset (IOPD) and late-onset Pompe disease (LOPD) patients who switched from alglucosidase alfa (ALG) to avalglucosidase alfa (AVA), presented at the 76th Annual Meeting of the American Academy of Neurology 2024
22 Sep 2023 Planned End Date changed from 1 Sep 2021 to 31 Jan 2034.
22 Sep 2023 Planned primary completion date changed from 1 Sep 2021 to 31 Jan 2034.

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