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 : Completed
First Posted : October 12, 2015
Last Update Posted : December 6, 2019
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|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)|
|Actual Enrollment :||58 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)|
|Actual Study Start Date :||November 2015|
|Actual Primary Completion Date :||December 3, 2019|
|Actual Study Completion Date :||December 3, 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.
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|Ages Eligible for Study:||3 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Children and adults under eculizumab treatment for aHUS (initial episode or relapse) defined by 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 )
- Patients not requiring dialysis.
- Adults: HUS remission and normal or stabilized renal function under eculizumab treatment since at least 6 months (3 months in patients with MCP mutations)
- Children: age > 3 years at eculizumab withdrawal; HUS remission and normal renal function under eculizumab treatment since at least 3 months in children with isolated MCP mutation, at least 6 months in children with complement mutation other than MCP.
- Patients on dialysis.
- Women treated with eculizumab starting or planning a pregnancy. Pregnancy including the post-partum period is high-risk periods for the occurrence of aHUS.
- Patients who did not give informed consent.
- Patients under protection of a judicial authority
Patients can be enrolled in the study within ten weeks after Eculizumab stop.
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
|Study Director:||Fadi FAKHOURI, Pr||Nantes CHU|
|Responsible Party:||Nantes University Hospital|
|Other Study ID Numbers:||
|First Posted:||October 12, 2015 Key Record Dates|
|Last Update Posted:||December 6, 2019|
|Last Verified:||December 2019|
atypical hemolytic uremic syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome
Blood Platelet Disorders
Complement Inactivating Agents
Physiological Effects of Drugs