This study will assess an investigational treatment for patients diagnosed with a rare genetic disorder known as Fabry disease.

The AT1001-013 study is evaluating the safety and effects of co-administering the investigational pharmacological chaperone migalastat HCl (AT1001 or GR181413A) and the enzyme replacement therapy (ERT) agalsidase in subjects with Fabry disease.

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Clinical Trial Overview
The AT1001-013 Co-Administration Study is a Phase 2A clinical trial designed to evaluate the safety and effects of migalastat HCl on agalsidase (ERT), in subjects with Fabry disease, when migalastat HCl is orally administered prior to the agalsidase (ERT) infusion. The duration of study participation for each study subject is approximately 2 to 4.5 months.

 Participant Criteria
The AT1001-013 Co-Administration Study is open to males with Fabry disease, 18-65 years of age, who are on a stable agalsidase (ERT) dose for at least 1 month. U.S. participants must be on Fabrazyme*, whereas ex-U.S. participants may be on either Replagal*or Fabrazyme*.

 Study Locations
The AT1001-013 Co-Administration Study is being conducted at sites in countries around the world, including sites in Europe, Canada, and the United States. In the United States, the study will be conducted in various states from coast-to-coast. Additional locations are continually being added. Check the website or contact us for more details.