Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Pharmacodynamics of AT1001 in Patients With Fabry Disease and AT1001-Responsive GLA Mutations|
- kidney GL-3 (assessed histologically in kidney biopsy samples) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- urine GL-3 levels [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- renal function (assessed by iohexol GFR, eGFR, and 24-hour urine protein) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- safety and tolerability [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||August 2009|
|Study Completion Date:||January 2014|
|Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Experimental: Galafold (AT1001) Oral Capsule
Migalastat HCl 150 mg capsule is taken every other day for 6 months and optional 6 month treatment extension
Drug: migalastat hydrochloride
oral capsule every other day
Placebo Comparator: Placebo Oral Capsule
Placebo capsule is taken every other day for 6 months.
oral capsule every other day
This double-blind, randomized, placebo-controlled study will be conducted in 60 patients at approximately 40 sites worldwide. The study will consist of two stages and an open-label treatment extension phase:
Stage 1 includes a screening period of up to 2 months followed by a 6-month treatment period which will involve 4 visits to the clinic. Patients will be randomized in equal proportions to receive either AT1001 or placebo.
After completing the 6-month double-blind phase, all patients will enter Stage 2 of the study and receive AT1001 in an open-label manner. Stage 2 treatment will last for 6 months and will involve 4 visits to the clinic.
Subjects who complete both Stage 1 and Stage 2 of the study as scheduled may be offered the opportunity to participate in an open-label treatment extension phase with AT1001. The open-label treatment extension phase will last 12 months and will involve 2 visits to the clinic. A follow-up visit will be undertaken 1 month following completion or discontinuation from the open-label treatment extension. Subjects completing the 12 month open-label treatment extension and providing consent to enter a separate long term extension will not be required to complete this follow-up visit.
Study assessments will include clinical laboratory tests, 12-lead ECG, kidney biopsy, kidney function testing, echocardiography, and patient reported outcomes.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00925301
Show 38 Study Locations
|Study Director:||Medical Monitor, Clinical Research||Amicus Therapeutics|