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Study Assessing an Algorithm-based Strategy of Eculizumab Discontinuation in Children and Adults With aHUS

NCT ID: NCT02574403

Last Updated: 2019-12-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

58 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-11-30

Study Completion Date

2019-12-03

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Atypical Hemolytic Uremic Syndrome

Keywords

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atypical hemolytic uremic syndrome (aHUS) eculizumab discontinuation

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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without eculizumab

Group Type EXPERIMENTAL

eculizumab

Intervention Type DRUG

eculizumab discontinuation

Interventions

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eculizumab

eculizumab discontinuation

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. 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 )
2. Patients not requiring dialysis.
3. Adults: HUS remission and normal or stabilized renal function under eculizumab treatment since at least 6 months (3 months in patients with MCP mutations)
4. 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.

Exclusion Criteria

1. Patients on dialysis.
2. Women treated with eculizumab starting or planning a pregnancy. Pregnancy including the post-partum period is high-risk periods for the occurrence of aHUS.
3. Patients who did not give informed consent.
4. Patients under protection of a judicial authority

Patients can be enrolled in the study within ten weeks after Eculizumab stop.
Minimum Eligible Age

3 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Nantes University Hospital

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Fadi FAKHOURI, Pr

Role: STUDY_DIRECTOR

Nantes CHU

Locations

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CHU Montpellier

Montpellier, , France

Site Status

CHU Nantes

Nantes, , France

Site Status

CHU Nice

Nice, , France

Site Status

BICHAT

Paris, , France

Site Status

Hopital Necker

Paris, , France

Site Status

Hopital Tenon

Paris, , France

Site Status

Hôpital Européen Georges Pompidou

Paris, , France

Site Status

CHU Rouen

Rouen, , France

Site Status

CH Alpes Léman

Sallanches, , France

Site Status

CHU Strasbourg

Strasbourg, , France

Site Status

Hopital FOCH

Suresnes, , France

Site Status

CHU toulouse

Toulouse, , France

Site Status

CHU Amiens

Amiens, , France

Site Status

CHU Bordeaux

Bordeaux, , France

Site Status

CHU Caen

Caen, , France

Site Status

CH métropole Savoie

Chambéry, , France

Site Status

CH Dijon

Dijon, , France

Site Status

Ch Le Mans

Le Mans, , France

Site Status

CHRU Lille

Lille, , France

Site Status

CHU lyon

Lyon, , France

Site Status

AP-HM

Marseille, , France

Site Status

CH Metz Thionville

Metz, , France

Site Status

Countries

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France

References

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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.

Reference Type DERIVED
PMID: 33270832 (View on PubMed)

Other Identifiers

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RC15_0061

Identifier Type: -

Identifier Source: org_study_id