A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease

Brief description of study

The purpose of this study is to find out if an investigational new drug called ATB200 and AT2221 can help people with Pompe disease and if it is safe. This study will also look at the way the body absorbs, distributes, and gets rid of this combination medication in Japanese subjects. Subjects with Pompe disease (like yourself) are born with a genetic mutation. This genetic mutation causes your body to have lower-than normal levels of an enzyme, called acid alpha-glucosidase (GAA). The GAA enzyme helps the body break down glycogen, a type of stored sugar. For example, muscle cells cannot function normally if glycogen cannot break down. This is what causes Pompe disease symptoms. ATB200 and AT2221 are investigational drugs. An “investigational drug” is a drug that is still being tested and not approved for sale. ATB200 has not been approved by the United States (US) Food and Drug Administration (FDA) in the United States or in any other country. The active ingredient in AT2221 (miglustat) is the same ingredient that is found in Zavesca®. It is approved for use by the US FDA, European Medicines Agency (EMA), and regulatory agencies in other countries. It is used for people with Gaucher disease but its use in Pompe disease is investigational.


Clinical Study Identifier: s19-01680
ClinicalTrials.gov Identifier: NCT04138277
Principal Investigator: Heather Ann Lau.


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