Therapeutic Orientation Test in Thrombotic Microangiopathy

Not Applicable

Recruiting

• Conditions: Thrombotic Micro-angiopathy
• Interventions: Diagnostic Test: Therapeutic orientation test for TMA

• Registration Number: NCT04777435
• Lead Sponsor: University Hospital, Montpellier

Brief Summary

Haemolytic and Uraemic Syndrome (HUS) is a serious disease requiring rapid diagnosis and management. The atypical HUS diagnosis has been greatly improved by anti-CS antibody (Eculizumab) wich block alternative complement pathway activation. To rise treatment success, Eculizumab introduction should be as early as possible. In some secondary HUS (infection, drugs...) complement is also involved as "second-hit".

To date, there is no tool to confirm complement involvement in a HUS at diagnosis stage. This study suggest to evaluate a therapeutic orientation test, in order to determine the complement implication in HUS diagnosis. The test evaluates the complement deposits on endothelial cell surface in vitro, compared to a normal human serum.

In order to determine the test performance, first the positive or negative results will be compared to the HUS clinical evolution, treated or not by the clinician with Eculizumab. Second, the test results will be compared to the presence of alternative complement pathway regulation abnormalities.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-02-19
• Study First Submitted QC: 2021-02-25
• Study First Posted: 2021-03-02
• Study Start: 2021-04-03
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-25
• Last Update Posted: 2024-01-26
• Primary Completion: 2025-10-03
• Study Completion: 2026-04-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

TMA with:

• mechanic haemolytic anemia, undetectable haptoglobin, LDH>1.5*LNS
• thrombopenia
• acute kidney injury TMA on native kidney or in post-transplantation.

Exclusion Criteria

• DIVC patients
• plasma exchange during 1 month before sample collection
• treatment by Eculizumab before sample collection
• no consent
• not beneficiary of a social security
• pregnancy or breastfeeding
• patient Under guardianship

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patients with Thrombotic micro-angiopathy	Therapeutic orientation test for TMA	-

Primary Outcome Measures

Name	Time	Method
Therapeutic orientation test sensitivity	Through study completion, an average of 3 years.	The proportion of patients testing positive among those receiving a relevant Eculizumab treatment ( TMA resolution with treatment or presence of abnormalities in alternative complement pathway.
Therapeutic orientation test specificity	Through study completion, an average of 3 years.	The proportion of patients testing negative among patients who did not receive Eculizumab treatment.(TMA resolution without treatment or therapeutic failure with Eculizumab).

Secondary Outcome Measures

Name	Time	Method
Untreated test positive patients	Through study completion, an average of 3 years.	The proportion of patients with positive test but untreated by Eculizumab, and who therefore could have benefited from treatment.

Trial Locations

Locations (20)

Site Médipôle Cabestany; 🇫🇷 Cabestany, France

CHU de Bordeaux; 🇫🇷 Bordeaux, France

Centre Nephrocare Castelnau-le-Lez; 🇫🇷 Castelnau-le-Lez, France

CHU de Lille; 🇫🇷 Lille, France

CHU de Grenoble; 🇫🇷 Grenoble, France

CHU de Limoges; 🇫🇷 Limoges, France

APHM-Hôpital de la Conception; 🇫🇷 Marseille, France

Montpellier University Hospital; 🇫🇷 Montpellier, France

CHU de Nantes; 🇫🇷 Nantes, France

HPGN- Narbonne; 🇫🇷 Narbonne, France

CHU de Nice; 🇫🇷 Nice, France

CHU de Nîmes; 🇫🇷 Nîmes, France

Hôpital Paris Necker; 🇫🇷 Paris, France

CHU de Rouen; 🇫🇷 Rouen, France

Hôpital Robert Debré; 🇫🇷 Paris, France

CHU de Poitiers; 🇫🇷 Poitiers, France

CH de Perpignan; 🇫🇷 Perpignan, France

APHP-Hôpital Tenon; 🇫🇷 Paris, France

Hôpitaux Universitaires de Strasbourg; 🇫🇷 Strasbourg, France

CHU de Toulouse; 🇫🇷 Toulouse, France