To determine the efficacy of an investigational coadministration regimen, AT-GAA2, by assessing pharmacokinetic parameters of the ATB200 protein and AT2221 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 separate long-term extension study.
You may be able to join the ZIP study if you are a male or female participant (Enzyme replacement therapy (ERT)-naïve [have never received a dose of alglucosidase alfa] or ERT-experienced [have received at least 1 dose of alglucosidase alfa]), diagnosed with late-onset Pompe disease who are aged 12 to < 18 years at screening. Volunteers must have a diagnosis of LOPD based on either a deficiency of acid alpha-glucosidase (GAA) enzyme or GAA genotyping. Other criteria may apply.
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 PK, safety, efficacy, and PD of an investigational treament, ATB200/AT2221, in pediatric participants aged 12 to < 18 years with LOPD.3
Amicus Therapeutics is pursuing the devlopment of an investigational coadministration regimen, ATB200/AT2221, for Pompe Disease. ATB200 is an investigational proprietary ERT administered by intravenous (IV) infusion, and AT2221 is an investigational pharmacological chaperone 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. Enzyme replacement therapy (ERT) using recombinant human GAA (rhGAA) is the only approved treatment available for Pompe disease.