AT1001-013 Study Overview

What is the AT1001-013 Co-Administration Study?

The AT1001-013 Co-Administration Study will assess an investigational treatment for people with a rare genetic disorder known as Fabry disease.

Study 013 is a Phase 2A clinical trial designed to evaluate the safety and effects of co-administering the oral investigational treatment, migalastat HCl (also known as AT1001 or GR181413A), with intravenous agalsidase (ERT). The study will enroll up to 24 participants. Blood, urine, and skin samples will be collected as part of this study.

The first 12 participants to enroll in the study will be part of STAGE 1. The next 12 participants will be part of STAGE 2. STAGE 1 has 3 periods and STAGE 2 has 2 periods:

STAGE 1:

  • Period 1: Agalsidase (ERT) alone as an intravenous infusion
  • Period 2: Oral dose of migalastat HCl, 2 hours before initiation of an
    intravenous infusion of agalsidase (ERT)
  • Period 3: Oral dose of migalastat HCl alone

STAGE 2:

  • Period 1: Agalsidase (ERT) alone as an intravenous infusion
  • Period 2: Oral dose of migalastat HCl, 2 hours before initiation of an intravenous infusion of agalsidase (ERT)

For this study all mutations are eligible. Patients who participate in this study would be eligible to be screened in future, longer-term agalsidase (ERT) plus migalastat HCl co-administration studies.

Primary Outcome Measures:

  • Migalastat HCl plasma pharmacokinetic parameter values (drug levels in the blood) after administration of a single oral dose of migalastat HCl alone and co-administered with agalsidase (ERT)
  • Agalsidase plasma pharmacokinetic parameter values by measurement of α-Gal A enzyme levels and protein levels after agalsidase infusion alone and co-administered with migalastat HCl
  • Safety labs and evaluations, adverse events
 

Secondary Outcome Measure:

  • Distribution of agalsidase (ERT) to skin after dosing with agalsidase (ERT) alone and agalsidase (ERT) co-administered with migalastat HCl