To determine the efficacy1 of an investigational coadministration regimen, AT-GAA2, by assessing pharmacokinetic parameters of the Cipaglucosidase Alfa protein and Miglustat concentrations in plasma along with measuring the number of participants with adverse events. The study will consist of a 30-day screening period, a 12-month treatment period, and a 30-day safety follow-up period, for a total duration of approximately 14 months. Participants who complete the study may have the opportunity to enroll in a long-term extension period.
You may be able to join the ZIP study if you are a male or female who has been diagnosed with late-onset Pompe disease and you are less than 18 years old at screening. The ZIP study is enrolling people who have never taken enzyme replacement therapy (ERT) before (ERT-naïve) and people who have received at least 1 dose of an rhGAA ERT (ERT-experienced).
The ZIP Study is being conducted at sites in the United States and in countries around the world. In the United States, the study will be conducted in various states from coast-to-coast. Additional locations/sites are continually being explored. Check back periodically for updated trial location/site information.
The ZIP study is designed to evaluate the safety, pharmacokinetics, efficacy, pharmacodynamics, and immunogenicity of Cipaglucosidase Alfa/Miglustat in pediatric subjects aged 0 to < 18 years with LOPD.3
Amicus Therapeutics is pursuing the development of an investigational coadministration regimen, Cipaglucosidase Alfa/Miglustat, for Pompe Disease. ATB200 is an investigational proprietary ERT administered by intravenous (IV) infusion, and AT2221 is an investigational enzyme stabilizer administered orally. Pompe disease is an inherited lysosomal storage disorder that results from a deficiency in acid alpha-glucosidase (GAA) and is characterized by progressive accumulation of lysosomal glycogen primarily in cardiac and skeletal muscles.