Current Research Studies
An open-label ascending dose cohort study to assess the safety, pharmacokinetics, and preliminary efficacy of neoGAA (GZ402666) in patients with infantile-onset Pompe disease treated with alglucosidase alfa who demonstrate clinical decline or sub-optimal response
ACT14132: Mini-Comet
What is the goal of the study?
This is a drug study to evaluate the safety of neoGAA in patients with infantile-onset Pompe Disease previously treated with alglucosidase alfa. Secondary objectives include characterizing the pharmacokinetic profile and efficacy of neoGAA in comparison to alglucosidase alfa and to determine the effect of neoGAA of functional endurance, respiratory function, health-related quality of life, pain, developmental disability, and hearing in patients with IOPD. This study utilizes ascending dose cohorts who receive repeated intravenous infusions of neoGAA. Patients involved have demonstrated clinical decline or sub-optimal response to treatment with alglucosase alfa.
Who can participate in the study?
Please contact the study team listed below to learn more.