Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS (STOPECU)
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|ClinicalTrials.gov Identifier: NCT02574403|
Recruitment Status : Active, not recruiting
First Posted : October 12, 2015
Last Update Posted : November 24, 2017
|Condition or disease||Intervention/treatment||Phase|
|Atypical Hemolytic Uremic Syndrome||Drug: eculizumab||Phase 4|
A visit (physical examination; blood pressure measurement) will be performed every month for 3 months, and every 3 months for 21 months.
Blood (serum creatinine, platelet count, hemoglobin, LDH, haptoglobin) and urine (proteinuria/creatininuria ratio and microscopic hematuria) tests will be performed every 2 weeks from inclusion to M6 and subsequently every month starting M7 Urine dipstick (for albuminuria and microscopic hematuria) will be performed by the patients at home at least twice a week.
Markers of complement activation and biomarkers of endothelial cells activation and immune cells activation will be assessed at baseline, M1, M3, M6, M9, M12, M18 and M24.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multicentric, Prospective Open-label Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With Atypical Hemolytic Uremic Syndrome (aHUS)|
|Study Start Date :||November 2015|
|Estimated Primary Completion Date :||November 2019|
|Estimated Study Completion Date :||November 2019|
|Experimental: without eculizumab||
- The incidence of aHUS relapse during 2 years of follow-up after eculizumab discontinuation [ Time Frame: 24 months ]
aHUS relapse will be defined by the coexistence of at least two of the following:
- thrombocytopenia (platelet count < 150 G/L),
- mechanical hemolytical anaemia (Hb < 10 g/dl, LDH > upper limit of normal, undetectable haptoglobin, presence of schizocytes on blood smear),
- acute kidney injury (serum creatinine and/or proteinuria/creatininuria > upper limit of normal for age or an increase > 15% compared to baseline levels ),
- features of thrombotic microangiopathy (glomerular and/or arteriolar thrombi, doubles contours, endothelial cells detachment) in a kidney biopsy, if performed.
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): NCT02574403
|CH métropole Savoie|
|Ch Le Mans|
|Le Mans, France|
|CH Metz Thionville|
|Hôpital Européen Georges Pompidou|
|CH Alpes Léman|
|Study Director:||Fadi FAKHOURI, Pr||Nantes CHU|