Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
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|ClinicalTrials.gov Identifier: NCT00925301|
Recruitment Status : Completed
First Posted : June 22, 2009
Last Update Posted : May 2, 2016
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat hydrochloride Drug: Placebo||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||67 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
|Study Start Date :||August 2009|
|Actual Primary Completion Date :||July 2012|
|Actual Study Completion Date :||January 2014|
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
- kidney GL-3 (assessed histologically in kidney biopsy samples) [ Time Frame: 6 months ]
- urine GL-3 levels [ Time Frame: 6 months ]
- renal function (assessed by iohexol GFR, eGFR, and 24-hour urine protein) [ Time Frame: 6 months ]
- safety and tolerability [ Time Frame: 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00925301
Show 38 Study Locations
|Study Director:||Medical Monitor, Clinical Research||Amicus Therapeutics|