Study Overview

What is the ATTRACT study?

The ATTRACT Study (AT1001 Therapy Compared to Enzyme Replacement in Fabry Patients with AT1001-responsive Mutations: a Global Clinical Trial) will assess an investigational treatment option for people with a rare genetic disorder known as Fabry disease.

Details about the ATTRACT Study

The ATTRACT Study is a Phase 3 clinical study that will measure the effectiveness and safety of an oral investigational treatment, migalastat HCl (also known as AT1001 or GR181413A), when compared to agalsidase (ERT), for the treatment of individuals with Fabry disease.

  • The study will enroll approximately 50 participants currently receiving agalsidase (ERT) who will be randomized to receive either migalastat HCl or agalsidase (ERT). Approximately 30 participants will stop their agalsidase (ERT) regimen and start with migalastat HCl and 20 participants will remain on their prescribed agalsidase (ERT) regimen.
  • Each participant’s study participation will last approximately 21 months, including a screening/baseline period (2 months), an open-label treatment period (18 months), and a follow-up period (1 month).
  • After completing the 18 month treatment period, participants will have the option to continue in a 12-month treatment extension where all participants will be treated with the investigational treatment, migalastat HCl. This extension phase will include a total of 5 study visits. Additional study extensions may be considered by the sponsor. Kidney biopsies will not be performed as part of this study.
 

Primary Outcome Measure:

The change from baseline in glomerular filtration rate (GFR) as assessed by plasma clearance of iohexol (“iohexol GFR”).

Secondary Outcome Measures:

    • Renal function (assessed by estimated GFR and 24-hour urine protein)
    • Composite clinical outcome (assessed by time to occurrence of renal, cardiac, cerebrovascular events or death)
    • Cardiac function (assessed by echocardiography)
    • Patient reported outcomes (pain and quality of life)