Clinical Research Directory

New Analytic Tools for aHUS and C3G Diagnosis

Sponsor: Mario Negri Institute for Pharmacological Research

Summary

This protocol is part of a larger project, COMPRare (COMPlement-mediated Rare kidney diseases), which has been financed on behalf of the EJP RD (European Joint Programme on Rare Diseases) program of EU and is leaded by a scientific consortium from 7 European countries. The partners (P) of the consortium are: P1. Radboudumc Amalia Children's Hospital (The Netherlands) P2. Semmelweis University (Hungary) P3. Cordeliers Research Center (France) P4. Max Delbruck Center for Molecular Medicine (Germany) P5. Istituto di Ricerche Farmacologiche Mario Negri (Italy) P6. Lund University (Sweden) P7. Lille University (France) The general aim of the project is to define new diagnostic tools for complement activation in order to improve patients stratification and follow-up, thereby affecting time and choice of treatment in patients with aHUS and C3G. Particularly, the specific objectives of the COMPRare are: * To develop new standardized analytic assays thereby identifying specific complement prognostic biomarkers for early diagnosis, classification, improved monitoring and treatment of patients with aHUS and C3G; * To in-depth characterize patients' complement abnormalities in blood, in patient-derived cells and in kidney biopsies; * To identify strategies to classify VUS/LPV * To find new pathophysiological pathways involved in aHUS and C3G for further improving disease diagnosis, monitoring and treatment. The results of these studies will form the basis of personalized treatment with existing and upcoming complement inhibitory drugs for these rare complement-mediated kidney diseases.

Official title: Towards the Most Accurate Diagnosis and Monitoring of Complement-mediated Rare Kidney Diseases

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