Fabry Disease MedDRA version: 14.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: migalastat hydrochloride
Product Code: AT1001
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: migalastat hydrochloride
Sponsors
Amicus Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Subject completed the main treatment period of another Phase 2 trial of AT1001 in Fabry disease 2.Women of childbearing potential must have a negative result on their pregnancy test 3.Male and female subjects agree to use reliable methods of contraception during study treatment and for 4 weeks after study treatment termination 4.Subject is willing and able to provide written informed consent Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject had a major protocol violation in the preceding AT1001 trial and was discontinued. 2.Subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis 3.Subject is treated or has been treated with another investigational drug (except AT1001) within 30 days of study start 4.Subject has been treated with Fabrazyme (agalsidase beta), Replagal (agalsidase alfa), Glyset (miglitol) or Zavesca(miglustat) within 2 weeks prior to enrollment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety and tolerability of oral AT1001 in patients with Fabry disease;Secondary Objective: To gain information about the pharmacodynamics and pharmacokinetics of orally administered AT1001 in patients with Fabry disease;Timepoint(s) of evaluation of this end point: End of study visit; Primary end point(s): •Treatment emergent marked laboratory abnormalities up to end of study (EOS) visit •Treatment emergent adverse events up to 48 hours after study medication discontinuation •Serious adverse events up to 28 days after study medication discontinuation •Change in concomitant treatments | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Levels of a-Gal A in leukocytes Levels of GL-3 in plasma and Levels of AT1001 in plasma ;Timepoint(s) of evaluation of this end point: Monitored throughout study | — |
Countries
Australia, Brazil, United Kingdom, United States
Contacts
Public ContactPatient Advocacy
Amicus Therapeutics
Outcome results
None listed