German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.
|ClinicalTrials.gov Identifier: NCT03135197|
Recruitment Status : Active, not recruiting
First Posted : May 1, 2017
Last Update Posted : August 31, 2018
|Condition or disease|
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.
|Study Type :||Observational|
|Estimated Enrollment :||60 participants|
|Official Title:||German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl.|
|Actual Study Start Date :||June 8, 2017|
|Estimated Primary Completion Date :||May 2019|
|Estimated Study Completion Date :||September 2019|
Migalastat administered according to SmPC
- LVMI [ Time Frame: two years ]Primary endpoint of the observational study is the change in left ventricular mass index (LVMI) over two years.
- GFR [ Time Frame: 24 months ]Change in GFR over 24 months
- Cerebral ischemia or stroke. [ Time Frame: 24 months ]Incidence of transient/manifest cerebral ischemia or stroke over 24 months.
- Neuropathic Pain (GCPS) [ Time Frame: 24 months ]Change in severity of neuropathic pain measured by Graded Chronic Pain Scale (GCPS)
- Neuropathic Pain (NPSI) [ Time Frame: 24 months ]Change in severity of neuropathic pain measured by Neuropathic Pain Symptom Inventory (NPSI) Score (items are quantified on a (0-10) numerical scale).
- Fabry Disease Severity (MSSI) [ Time Frame: 24 months ]Change in disease severity measured by Mainz Severity Score Index (MSSI)
- Fabry Disease Severity (DS3) [ Time Frame: 24 months ]Change in disease severity measured by the Disease Severity Scoring System (DS3)
- Lyso-Gb3 [ Time Frame: 24 months ]Change in Lyso-Gb3
- White Matter Lesion load [ Time Frame: 24 months ]Change of White Matter Lesion load (quantified by WML volumetry [ml]).
- Cerebral microbleeds/hemorrhagic lesions. [ Time Frame: 24 months ]Stabilization of cerebral microbleeds/hemorrhagic lesions.
- Gastrointestinal symptoms [ Time Frame: 24 months ]Change in gastrointestinal symptoms (gastrointestinal symptoms rating scale, GSRS).
- Quality of life (SF-36) [ Time Frame: 24 months ]Change in quality of life (Short Form (SF-36) Health Survey: 36-item, patient-reported survey of patient health).
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): NCT03135197
|Universtiy Hospital Münster|
|Münster, Germany, 48149|
|Principal Investigator:||Eva Brand, Prof.||University Hospital Muenster|