Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring (EspacECU)

NCT ID: NCT04859608

Last Updated: 2025-03-25

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

PHASE3

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-10-20

Study Completion Date

2023-12-28

Brief Summary

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Eculizumab is an anti-C5 monoclonal antibody approved for rare diseases including atypical haemolytic-uraemic syndrome. The maintenance phase dosing regimen is identical for all adult patients but several studies have shown a high interindividual kinetics variability. A tailored administration of eculizumab based on therapeutic drug monitoring will be compared with real-life administration in adults suffering from an atypical haemolytic uraemic syndrome. The objective is to improve efficiency of eculizumab administration.

Detailed Description

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Eculizumab is a very expensive drug (450 000 euros per year per patient). According to the usual administration scheme, the eculizumab is administrated by intravenous infusion, every 14 or 21 days in hospital. Studies have shown the possibility of extending the administration intervals in patients with a high concentration of eculizumab.

A personalized spacing of eculizumab infusions, based on a therapeutic drug monitoring, is expected to improve the patient's quality of life and lead to substantial savings.

For these reasons, the objective of this study is to explore the efficiency of a personalized spacing of eculizumab infusions compared to the usual administration scheme (without personalization) from the French health Insurance perspective. The efficiency will be assessed by estimating the incremental cost per QALY (Quality-Adjusted Life Year) gained at 18 months (duration of follow-up in the clinical trial) and at 5 years thanks to a Markov model.

The total duration of the study is 24 months (6 months of enrolment and 18 months of follow-up).

Conditions

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Hemolytic-Uremic Syndrome, Atypical

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Randomized (2:1) controlled study, open-label, with Markov modeling.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Therapeutic drug monitoring

Tailored dosing schedule for eculizumab based on therapeutic drug monitoring

Group Type EXPERIMENTAL

Spacing of Eculizumab infusions

Intervention Type DRUG

Personalized spacing of eculizumab infusions using a pharmacokinetic population model to estimate eculizumab concentration ( a one-week spacing of eculizumab infusion will be decide if predicted eculizumab concentration in the event of spacing is \> 150 mg/L). Spacing of infusion can be decided every 3 months.

Control

Initial eculizumab schedule is continued (real-life arm). No eculizumab dosages are performed in this arm.

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Spacing of Eculizumab infusions

Personalized spacing of eculizumab infusions using a pharmacokinetic population model to estimate eculizumab concentration ( a one-week spacing of eculizumab infusion will be decide if predicted eculizumab concentration in the event of spacing is \> 150 mg/L). Spacing of infusion can be decided every 3 months.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Adults with an atypical Hemolytic and Uremic Syndrome defined by at least 2 of the following parameters:

* Thrombopenia (platelet count \< 150 G/L)
* Mechanical lytic anemia (Hb \< 10 g/L, Lactate dehydrogenase \> upper limit of normal, undetectable haptoglobin, presence of schistocytes on blood smear)
* Acute renal failure
2. Eculizumab treatment

* Without project of withdrawal in the next 18 months
* Started since at least 6 months
* Administrated with an unchanged administration schedule since at least 3 months (initial episode or relapse)
3. Atypical Hemolytic and Uremic Syndrome in remission
4. Patients who give informed consent.

Exclusion Criteria

1. Patients on dialysis.
2. Women treated starting or planning a pregnancy.
3. Patients suffering from a typical or secondary microangiopathic hemolytic anemia (drugs, malignancies, autoimmune disease...)
4. Patients under protection of a judicial authority
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Centre Hospitalier Universitaire, Amiens

OTHER

Sponsor Role collaborator

University Hospital, Angers

OTHER_GOV

Sponsor Role collaborator

Centre Hospitalier Universitaire de Besancon

OTHER

Sponsor Role collaborator

University Hospital, Caen

OTHER

Sponsor Role collaborator

Centre Hospitalier of Chartres

OTHER

Sponsor Role collaborator

Nantes University Hospital

OTHER

Sponsor Role collaborator

University of Nancy

OTHER

Sponsor Role collaborator

Poitiers University Hospital

OTHER

Sponsor Role collaborator

Rennes University Hospital

OTHER

Sponsor Role collaborator

University Hospital, Rouen

OTHER

Sponsor Role collaborator

University Hospital, Strasbourg, France

OTHER

Sponsor Role collaborator

Centre Hospitalier Universitaire de Nice

OTHER

Sponsor Role collaborator

Hôpital Necker-Enfants Malades

OTHER

Sponsor Role collaborator

Tenon Hospital, Paris

OTHER

Sponsor Role collaborator

University Hospital, Lille

OTHER

Sponsor Role collaborator

Reims University hospital

OTHER

Sponsor Role collaborator

University Hospital, Clermont-Ferrand

OTHER

Sponsor Role collaborator

Assistance Publique Hopitaux De Marseille

OTHER

Sponsor Role collaborator

Hospices Civils de Lyon

OTHER

Sponsor Role collaborator

University Hospital, Tours

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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CHU Amiens Picardie

Amiens, , France

Site Status

CHU d'Angers

Angers, , France

Site Status

Hôpital Tenon

APHP Tenon, , France

Site Status

CHU Besançon

Besançon, , France

Site Status

Hôpital de la Côte de Nacre

Caen, , France

Site Status

Hôpital Louis Pasteur

Chartres, , France

Site Status

Hôpital Gabriel Montpied

Clermont-Ferrand, , France

Site Status

Hospices Civils de Lyon

Lyon, , France

Site Status

Hopital de la conception

Marseille, , France

Site Status

Hôpitaux de Brabois

Nancy, , France

Site Status

Hotel Dieu

Nantes, , France

Site Status

Hopital Necker

Necker, , France

Site Status

Hopital Pasteur 2

Nice, , France

Site Status

Hôpital de la Milétrie

Poitiers, , France

Site Status

Hôpital Maison Blanche

Reims, , France

Site Status

Hôpital Pontchaillou

Rennes, , France

Site Status

Hôpital de Bois-Guillaume

Rouen, , France

Site Status

Nouvel Hôpital Civil

Strasbourg, , France

Site Status

Countries

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France

Other Identifiers

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DR200088-EspacECU

Identifier Type: -

Identifier Source: org_study_id

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