Efficacy and safety of AT1001 and ERT in patients with Fabry Disease
Research type
Research Study
Full title
RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001- RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY TREATED WITH ERT
IRAS ID
72451
Contact name
Derralynn Hughes
Sponsor organisation
Amicus Therapeutics
Eudract number
2010-022636-37
Clinicaltrials.gov Identifier
Research summary
This is a phase 3 open label study to evaluate the efficacy and safety of AT1001 and enzyme replacement therapy (ERT) in male and female patients with Fabry disease who are currently receiving ERT and who have AT1001-responsive GLA mutations. Patients will be randomly assigned into one of two treatment groups either to stop ERT treatment and start treatment with AT1001 or to continue on ERT. Each patient's participation will last approximately 21 months, including a screening/baseline period (approximately 2 months), an open-label treatment period (approximately 18 months), and a follow-up period (1 month). After completing the 18 month open-label treatment period, subjects will be offered the opportunity to participate in an optional AT1001 extension period. For subjects who choose to participate in the optional extension period the duration of study participation will last a total of approximately 33 months. This is a multicenter international study with approximately 35 study sites
REC name
London - Fulham Research Ethics Committee
REC reference
11/LO/0101
Date of REC Opinion
15 Apr 2011
REC opinion
Further Information Favourable Opinion