Glycogen storage disease type II research hub
5 indexed studies · 2 currently recruiting
- recruitingTherapeutic confirmatory (Phase III)
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)
This study is testing a new medicine, Nexviadyme, for babies with a rare genetic condition called Pompe disease. It aims to see if the medicine helps them breathe better and grow, and if it's safe, especially for those who haven't tried other treatments.
Spain · Netherlands · Germany - recruiting
Pompe Disease Registry Protocol
This registry tracks people with Pompe disease globally. It helps us understand the condition better, how it changes over time, and the effects of treatments. The goal is to improve care for everyone with Pompe disease.
United States · Argentina · Australia - active not recruitingPHASE1, PHASE2
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
This study is testing a new medicine called SPK-3006 for adults with late-onset Pompe disease. It aims to see if it's safe and helps improve the condition. Participants will receive one dose of the new medicine and will be carefully checked.
United States · Canada · Denmark - active not recruitingPHASE3
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
This study is for babies under one year old newly diagnosed with Infantile-Onset Pompe Disease. It's testing a new medicine, avalglucosidase alfa, to see if it's safe and effective in improving their condition, following them for up to four years.
United States · Belgium · China - active not recruiting
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
This study looks at how a treatment called alglucosidase alfa works for babies up to 6 months old with a serious genetic condition called Pompe disease. Researchers want to see if it helps them breathe without a ventilator and how it affects their heart and development.
United States · Belgium · France