Fabry Disease Clinical Trial
Official title:
German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
The objective of the study is to document long term data on treatment with Migalastat under "real world" conditions. The selection of patients is based on the SmPC/Fachinformation. The study duration/patient will be 2 years.
Phase 3 data should be confirmed in this study with long-term data. - LVMI is expected to remain stable or to be ameliorated over an average of 24 months treatment duration. The LVMI reduction observed in patients followed up to 24 months is expected to be significantly reduced with a mean change of -6.6 g/m2 (-11.0, -2.1, 95% CI). - eGFR [CKD-EPI] is expected to remain stable over an average of 24 months treatment duration. The long-term effect of Migalastat on eGFR is expected to be comparable to the decline over time in healthy adults. The annualized rate of change over this period is expected to be ≤1 mL/min/1.73 m2 in females and ≤3 mL/min/1.73 m2 in males. - Significant reduction is expected in plasma lyso-Gb3 concentration at month 6, month 12 and month 24 following treatment with Migalastat. - ERT-naïve patients treated with Migalastat are expected to show an improvement of GI symptoms (diarrhea) over 24 months. - No progression of White Matter Lesions (WML) during treatment duration is expected. - No higher frequency of stroke/transient cerebral ischemia during treatment duration is expected. - Severity of neuropathic pain is expected to remain stable or to improve during treatment duration. - Dosing/amount of symptomatic medications of neuropathic symptoms is expected to decrease during treatment duration. ;
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