Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS
STOPECU
Multicentric, Prospective Open-label Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With Atypical Hemolytic Uremic Syndrome (aHUS)
1 other identifier
interventional
58
1 country
22
Brief Summary
Atypical hemolytic syndrome (aHUS) is a severe renal disease affecting children and adults. It is characterized by the occlusion of intrarenal vessels due to the presence of platelet/fibrin thrombi, and leads to end-stage renal disease in up to 2/3 of patients. The discovery of complement alternative pathway as a major risk factor for aHUS has led to the design of a disease-specific treatment, the anti-C5 monoclonal antibody, eculizumab. Complement inhibition using eculizumab has clearly improved the renal outcome of aHUS patients with a dramatic decrease in the risk of end-stage renal disease. However, the optimal duration of eculizumab therapy is still debated. The present study aims to assess the feasibility and safety of the discontinuation of eculizumab treatment in children and adults with aHUS.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started Nov 2015
Longer than P75 for phase_4
22 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 5, 2015
CompletedFirst Posted
Study publicly available on registry
October 12, 2015
CompletedStudy Start
First participant enrolled
November 1, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 3, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
December 3, 2019
CompletedDecember 6, 2019
December 1, 2019
4.1 years
October 5, 2015
December 5, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The incidence of aHUS relapse during 2 years of follow-up after eculizumab discontinuation
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.
24 months
Study Arms (1)
without eculizumab
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (22)
CHU Amiens
Amiens, France
CHU Bordeaux
Bordeaux, France
CHU Caen
Caen, France
CH métropole Savoie
Chambéry, France
CH Dijon
Dijon, France
Ch Le Mans
Le Mans, France
CHRU Lille
Lille, France
CHU lyon
Lyon, France
AP-HM
Marseille, France
CH Metz Thionville
Metz, France
CHU Montpellier
Montpellier, France
CHU Nantes
Nantes, France
CHU Nice
Nice, France
BICHAT
Paris, France
Hopital Necker
Paris, France
Hopital Tenon
Paris, France
Hôpital Européen Georges Pompidou
Paris, France
CHU Rouen
Rouen, France
CH Alpes Léman
Sallanches, France
CHU Strasbourg
Strasbourg, France
Hopital FOCH
Suresnes, France
CHU toulouse
Toulouse, France
Related Publications (1)
Fakhouri F, Fila M, Hummel A, Ribes D, Sellier-Leclerc AL, Ville S, Pouteil-Noble C, Coindre JP, Le Quintrec M, Rondeau E, Boyer O, Provot F, Djeddi D, Hanf W, Delmas Y, Louillet F, Lahoche A, Favre G, Chatelet V, Launay EA, Presne C, Zaloszyc A, Caillard S, Bally S, Raimbourg Q, Tricot L, Mousson C, Le Thuaut A, Loirat C, Fremeaux-Bacchi V. Eculizumab discontinuation in children and adults with atypical hemolytic-uremic syndrome: a prospective multicenter study. Blood. 2021 May 6;137(18):2438-2449. doi: 10.1182/blood.2020009280.
PMID: 33270832DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Fadi FAKHOURI, Pr
Nantes CHU
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 5, 2015
First Posted
October 12, 2015
Study Start
November 1, 2015
Primary Completion
December 3, 2019
Study Completion
December 3, 2019
Last Updated
December 6, 2019
Record last verified: 2019-12