Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
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|ClinicalTrials.gov Identifier: NCT02194985|
Recruitment Status : Active, not recruiting
First Posted : July 21, 2014
Last Update Posted : June 15, 2018
This is an open-label extension study intended to provide continued treatment with migalastat HCl for subjects with Fabry disease who completed treatment of a previous migalastat HCl monotherapy study.
The study will assess the long-term safety and effectiveness of migalastat HCl in subjects with Fabry disease who completed migalastat HCl treatment in a previous study.
|Condition or disease||Intervention/treatment||Phase|
|Fabry Disease||Drug: migalastat HCl 150 mg||Phase 3|
Expanded Access : An investigational treatment associated with this study is available outside the clinical trial. More info ...
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||76 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease|
|Study Start Date :||October 2014|
|Estimated Primary Completion Date :||October 2019|
|Estimated Study Completion Date :||October 2019|
Experimental: migalastat HCl 150 mg
Migalastat HCl is a capsule provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken or a punch-out reminder is used on days between migalastat HCl.
Drug: migalastat HCl 150 mg
Capsule provided in 14-day supply blister packs. Migalastat HCl is taken every other day by mouth. An inactive reminder capsule is taken or a punch-out reminder is used on the days between migalastat HCl.
Other Name: AT1001
- Incidence of adverse events (AEs) [ Time Frame: up to 5 years ]non-serious AEs, serious AEs., possible suicidality related AEs
- Withdrawal due to AEs [ Time Frame: up to 5 years ]including non-serious and serious AEs
- Vital signs and body weight - change in baseline in vital signs and body weight [ Time Frame: up to 5 years ]blood pressure and heart rate
- Change from baseline in laboratory parameters [ Time Frame: up to 5 years ]hematology, chemistry, and urinalysis
- Change from baseline in electrocardiogram (ECG) [ Time Frame: up to 5 years ]12-lead ECG
- Change from baseline in echocardiography [ Time Frame: up to 5 years ]echocardiography ultrasound
- Estimated glomerular filtration rate (eGFR) [ Time Frame: up to 5 years ]based on the Modification of Diet in Renal Disease (MDRD) equation and CKD-EPI equation
- Measurement of left ventricular mass (LVM) and left ventricular mass index (LVMi) [ Time Frame: up to 5 years ]as measured by echocardiography
- Ejection fraction [ Time Frame: up to 5 years ]as measured by echocardiography
- Fractional shortening [ Time Frame: up to 5 years ]as assessed by echocardiography
- Measurement of LV internal dimension (LVIDd and LVIDs) and wall thickness [ Time Frame: up to 5 years ]as assessed by echocardiography
- Measurement of white blood cell (WBC) α-Gal A activity [ Time Frame: up to 5 years ]enzyme responsible for breaking down GL-3
- Measurement of subject reported Quality of Life [ Time Frame: up to 5 years ]as assessed by the Short Form - 36 Survey (SF-36)
- Measurement of 24-hour urine protein [ Time Frame: up to 5 years ]
- Plasma lyso-Gb3 [ Time Frame: up to 5 years ]biomarker shown to be elevated in Fabry subjects
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): NCT02194985
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|Study Director:||Medical Monitor, Clinical Research, MD||Amicus Therapeutics, Inc.|