xml: <clinical_study>
    <!-- This xml conforms to an XML Schema at:
      https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
    <required_header>
      <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
      <link_text>Link to the current ClinicalTrials.gov record.</link_text>
      <url>https://clinicaltrials.gov/ct2/show/NCT05985122</url>
    </required_header>
    <id_info>
      <org_study_id>COMPRare-IRFMN</org_study_id>
      <nct_id>NCT05985122</nct_id>
    </id_info>
    <brief_title>New Analytic Tools for aHUS and C3G Diagnosis</brief_title>
    <acronym>COMPRare</acronym>
    <official_title>Towards the Most Accurate Diagnosis and Monitoring of Complement-mediated Rare Kidney Diseases</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Mario Negri Institute for Pharmacological Research</agency>
        <agency_class>Other</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Mario Negri Institute for Pharmacological Research</source>
    <oversight_info>
      <has_dmc>No</has_dmc>
      <is_fda_regulated_drug>No</is_fda_regulated_drug>
      <is_fda_regulated_device>No</is_fda_regulated_device>
    </oversight_info>
    <brief_summary>
      <textblock>
        This protocol is part of a larger project, COMPRare (COMPlement-mediated Rare kidney&#xD;
        diseases), which has been financed on behalf of the EJP RD (European Joint Programme on Rare&#xD;
        Diseases) program of EU and is leaded by a scientific consortium from 7 European countries.&#xD;
  &#xD;
        The partners (P) of the consortium are:&#xD;
  &#xD;
        P1. Radboudumc Amalia Children's Hospital (The Netherlands) P2. Semmelweis University&#xD;
        (Hungary) P3. Cordeliers Research Center (France) P4. Max Delbruck Center for Molecular&#xD;
        Medicine (Germany) P5. Istituto di Ricerche Farmacologiche Mario Negri (Italy) P6. Lund&#xD;
        University (Sweden) P7. Lille University (France)&#xD;
  &#xD;
        The general aim of the project is to define new diagnostic tools for complement activation in&#xD;
        order to improve patients stratification and follow-up, thereby affecting time and choice of&#xD;
        treatment in patients with aHUS and C3G.&#xD;
  &#xD;
        Particularly, the specific objectives of the COMPRare are:&#xD;
  &#xD;
          -  To develop new standardized analytic assays thereby identifying specific complement&#xD;
             prognostic biomarkers for early diagnosis, classification, improved monitoring and&#xD;
             treatment of patients with aHUS and C3G;&#xD;
  &#xD;
          -  To in-depth characterize patients' complement abnormalities in blood, in patient-derived&#xD;
             cells and in kidney biopsies;&#xD;
  &#xD;
          -  To identify strategies to classify VUS/LPV&#xD;
  &#xD;
          -  To find new pathophysiological pathways involved in aHUS and C3G for further improving&#xD;
             disease diagnosis, monitoring and treatment.&#xD;
  &#xD;
        The results of these studies will form the basis of personalized treatment with existing and&#xD;
        upcoming complement inhibitory drugs for these rare complement-mediated kidney diseases.&#xD;
      </textblock>
    </brief_summary>
    <detailed_description>
      <textblock>
        Atypical Hemolytic Uremic Syndrome (aHUS) and C3 Glomerulopathies (C3G) are ultra-rare&#xD;
        conditions in which an uncontrolled complement activation results in renal inflammation with&#xD;
        thrombotic microangiopathy (TMA) in aHUS and extensive deposition of complement fragments in&#xD;
        the kidneys in C3G. In both conditions, these alterations lead to acute and chronic kidney&#xD;
        damage and, ultimately, end-stage renal failure. The prevalence of genetic and acquired&#xD;
        complement abnormalities varies between the two diseases, but covers mostly the same&#xD;
        complement proteins. Blocking the complement system is the treatment of choice in aHUS and&#xD;
        may have considerable potential in C3G, as evident from the increasing number of phase 3&#xD;
        trials with complement inhibitory drugs. Timely and accurate diagnosis is still challenging&#xD;
        for clinicians, both for aHUS, to differentiate it from other forms of TMA, and for C3G, to&#xD;
        distinguish it from other forms of glomerulonephritis (GN). In C3G, kidney biopsy is the&#xD;
        diagnostic gold standard but it is not sufficient. Therefore, for both aHUS and C3G, there is&#xD;
        an unmet need for accurate testing of genetic and acquired complement abnormalities.&#xD;
        Moreover, interlaboratory complement-assays, in particular the tests for the C3 Nephritic&#xD;
        Factors (C3NeFs; heterogeneous autoantibodies that stabilize the C3 convertase), vary&#xD;
        considerably and, consequently, their standardization is a great necessity. Furthermore, the&#xD;
        interpretation of genetic results in aHUS and C3G is not always conclusive.&#xD;
  &#xD;
        Variants of Unknown Significance (VUS)/Likely Pathogenic Variant (LPV) in complement genes&#xD;
        are often found in aHUS and C3G and require further characterization. A rapid and functional&#xD;
        VUS/LPV determination and interpretation could help to better understand the disease&#xD;
        pathophysiology and might influence diagnosis and treatment options.&#xD;
  &#xD;
        Currently, aHUS patients are effectively treated with the complement C5 inhibiting monoclonal&#xD;
        antibodies Eculizumab and Ravulizumab. However, the duration and optimal dose of treatment&#xD;
        are debated, particularly in view of the high costs of the drug. Even if recent studies&#xD;
        showed that restrictive use of eculizumab is feasible, there is a clear need for biomarkers&#xD;
        identification and protocols for monitoring and predicting disease activity in aHUS and C3G&#xD;
        patients.&#xD;
  &#xD;
        In summary, new analytic tools for further defining the disease profile in blood, (patients)&#xD;
        cells and kidney biopsies are needed.&#xD;
      </textblock>
    </detailed_description>
    <overall_status>Not yet recruiting</overall_status>
    <start_date type="Anticipated">October 2023</start_date>
    <completion_date type="Anticipated">October 2026</completion_date>
    <primary_completion_date type="Anticipated">October 2026</primary_completion_date>
    <phase>N/A</phase>
    <study_type>Interventional</study_type>
    <has_expanded_access>No</has_expanded_access>
    <study_design_info>
      <allocation>Non-Randomized</allocation>
      <intervention_model>Parallel Assignment</intervention_model>
      <intervention_model_description>The samples (healthy volunteers and HUS or MPGN patients) for the study will be identified between those of patients and healthy controls who provided consent to store their samples in the certified biobank of &quot;Centro Daccò&quot; (Centro di Risorse Biologiche Mario Negri - Biobanca Malattie Rare e Renali) for two previousely approved studies.</intervention_model_description>
      <primary_purpose>Diagnostic</primary_purpose>
      <masking>None (Open Label)</masking>
    </study_design_info>
    <primary_outcome>
      <measure>Determination of assay sensitivity and specificity</measure>
      <time_frame>At day 0</time_frame>
      <description>We estimate that 38 VUS/LPV carrying pedigrees should be enough to demonstrate the sensitivity and specificity of the assay for classifying VUS/LPV.</description>
    </primary_outcome>
    <number_of_arms>2</number_of_arms>
    <enrollment type="Anticipated">180</enrollment>
    <condition>Hemolytic-Uremic Syndrome</condition>
    <condition>Membranoproliferative Glomerulonephritis</condition>
    <condition>Healthy</condition>
    <arm_group>
      <arm_group_label>Patients</arm_group_label>
      <arm_group_type>Experimental</arm_group_type>
    </arm_group>
    <arm_group>
      <arm_group_label>Healthy controls</arm_group_label>
      <arm_group_type>Active Comparator</arm_group_type>
    </arm_group>
    <intervention>
      <intervention_type>Diagnostic Test</intervention_type>
      <intervention_name>C3NEF assay</intervention_name>
      <description>This assay will consist of a dual test, detecting C3 convertase binding C3NEF autoantibodies and measuring the functional consequence by complement alternative pathway (AP) activity using two distinct ELISA designs: C3NEF detection assay and AP activity assay.</description>
      <arm_group_label>Healthy controls</arm_group_label>
      <arm_group_label>Patients</arm_group_label>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Aim 1&#xD;
  &#xD;
          Inclusion Criteria:&#xD;
  &#xD;
            -  Male and female patients (children and adults) with C3G diagnosis&#xD;
  &#xD;
            -  Biobank written informed consent&#xD;
  &#xD;
          Aim 2 Inclusion criteria&#xD;
  &#xD;
            -  Male and female patients (children and adults) with aHUS diagnosis in acute phase&#xD;
               (before any treatment), or in remission either untreated or undergoing anti-C5&#xD;
               treatment at standard dosing&#xD;
  &#xD;
            -  Written informed consent Exclusion criteria&#xD;
  &#xD;
            -  Stx-associated HUS&#xD;
  &#xD;
            -  TTP (ADAMTS13&lt;10%)&#xD;
  &#xD;
            -  Plasma therapy within 2 weeks from blood sampling&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>N/A</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>Accepts Healthy Volunteers</healthy_volunteers>
    </eligibility>
    <overall_official>
      <last_name>Marina Noris, PhD</last_name>
      <role>Principal Investigator</role>
      <affiliation>Istituto di Ricerche Farmacologiche Mario Negri IRCCS</affiliation>
    </overall_official>
    <overall_contact>
      <last_name>Marina Noris, PhD</last_name>
      <phone>+3903545351</phone>
      <email>marina.noris@marionegri.it</email>
    </overall_contact>
    <overall_contact_backup>
      <last_name>Erica Daina</last_name>
      <phone>+3903545351</phone>
      <email>erica.daina@marionegri.it</email>
    </overall_contact_backup>
    <location>
      <facility>
        <name>Centro di Ricerche Cliniche per le Malattie Rare &quot;Aldo e Cele Daccò&quot;</name>
        <address>
          <city>Ranica</city>
          <state>BG</state>
          <zip>24020</zip>
          <country>Italy</country>
        </address>
      </facility>
      <contact>
        <last_name>Erica Daina</last_name>
        <phone>0039 035 45351</phone>
        <email>erica.daina@marionegri.it</email>
      </contact>
    </location>
    <location_countries>
      <country>Italy</country>
    </location_countries>
    <reference>
      <citation>Noris M, Galbusera M, Gastoldi S, Macor P, Banterla F, Bresin E, Tripodo C, Bettoni S, Donadelli R, Valoti E, Tedesco F, Amore A, Coppo R, Ruggenenti P, Gotti E, Remuzzi G. Dynamics of complement activation in aHUS and how to monitor eculizumab therapy. Blood. 2014 Sep 11;124(11):1715-26. doi: 10.1182/blood-2014-02-558296. Epub 2014 Jul 18.</citation>
      <PMID>25037630</PMID>
    </reference>
    <reference>
      <citation>Iatropoulos P, Daina E, Curreri M, Piras R, Valoti E, Mele C, Bresin E, Gamba S, Alberti M, Breno M, Perna A, Bettoni S, Sabadini E, Murer L, Vivarelli M, Noris M, Remuzzi G; Registry of Membranoproliferative Glomerulonephritis/C3 Glomerulopathy; Nastasi. Cluster Analysis Identifies Distinct Pathogenetic Patterns in C3 Glomerulopathies/Immune Complex-Mediated Membranoproliferative GN. J Am Soc Nephrol. 2018 Jan;29(1):283-294. doi: 10.1681/ASN.2017030258. Epub 2017 Oct 13.</citation>
      <PMID>29030465</PMID>
    </reference>
    <reference>
      <citation>Donadelli R, Pulieri P, Piras R, Iatropoulos P, Valoti E, Benigni A, Remuzzi G, Noris M. Unraveling the Molecular Mechanisms Underlying Complement Dysregulation by Nephritic Factors in C3G and IC-MPGN. Front Immunol. 2018 Oct 15;9:2329. doi: 10.3389/fimmu.2018.02329. eCollection 2018.</citation>
      <PMID>30487789</PMID>
    </reference>
    <reference>
      <citation>Galbusera M, Noris M, Gastoldi S, Bresin E, Mele C, Breno M, Cuccarolo P, Alberti M, Valoti E, Piras R, Donadelli R, Vivarelli M, Murer L, Pecoraro C, Ferrari E, Perna A, Benigni A, Portalupi V, Remuzzi G. An Ex Vivo Test of Complement Activation on Endothelium for Individualized Eculizumab Therapy in Hemolytic Uremic Syndrome. Am J Kidney Dis. 2019 Jul;74(1):56-72. doi: 10.1053/j.ajkd.2018.11.012. Epub 2019 Mar 7.</citation>
      <PMID>30851964</PMID>
    </reference>
    <reference>
      <citation>Piras R, Breno M, Valoti E, Alberti M, Iatropoulos P, Mele C, Bresin E, Donadelli R, Cuccarolo P, Smith RJH, Benigni A, Remuzzi G, Noris M. CFH and CFHR Copy Number Variations in C3 Glomerulopathy and Immune Complex-Mediated Membranoproliferative Glomerulonephritis. Front Genet. 2021 Jun 11;12:670727. doi: 10.3389/fgene.2021.670727. eCollection 2021.</citation>
      <PMID>34211499</PMID>
    </reference>
    <reference>
      <citation>Aiello S, Gastoldi S, Galbusera M, Ruggenenti P, Portalupi V, Rota S, Rubis N, Liguori L, Conti S, Tironi M, Gamba S, Santarsiero D, Benigni A, Remuzzi G, Noris M. C5a and C5aR1 are key drivers of microvascular platelet aggregation in clinical entities spanning from aHUS to COVID-19. Blood Adv. 2022 Jan 8;6(3):866-881. doi: 10.1182/bloodadvances.2021005246. Erratum In: Blood Adv. 2022 May 10;6(9):2812.</citation>
      <PMID>34852172</PMID>
    </reference>
    <reference>
      <citation>Piras R, Valoti E, Alberti M, Bresin E, Mele C, Breno M, Liguori L, Donadelli R, Rigoldi M, Benigni A, Remuzzi G, Noris M. CFH and CFHR structural variants in atypical Hemolytic Uremic Syndrome: Prevalence, genomic characterization and impact on outcome. Front Immunol. 2023 Jan 30;13:1011580. doi: 10.3389/fimmu.2022.1011580. eCollection 2022.</citation>
      <PMID>36793547</PMID>
    </reference>
    <reference>
      <citation>Gastoldi S, Aiello S, Galbusera M, Breno M, Alberti M, Bresin E, Mele C, Piras R, Liguori L, Santarsiero D, Benigni A, Remuzzi G, Noris M. An ex vivo test to investigate genetic factors conferring susceptibility to atypical haemolytic uremic syndrome. Front Immunol. 2023 Feb 9;14:1112257. doi: 10.3389/fimmu.2023.1112257. eCollection 2023.</citation>
      <PMID>36845135</PMID>
    </reference>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 2, 2023</study_first_submitted>
    <study_first_submitted_qc>August 2, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 14, 2023</study_first_posted>
    <last_update_submitted>August 9, 2023</last_update_submitted>
    <last_update_submitted_qc>August 9, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 14, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>Complement-mediated kidney diseases</keyword>
    <keyword>Blood and tissue biomarkers</keyword>
    <keyword>Assay standardization</keyword>
    <keyword>Glycocalyx</keyword>
    <keyword>Characterization of variants of unknown significance</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Glomerulonephritis</mesh_term>
      <mesh_term>Hemolytic-Uremic Syndrome</mesh_term>
      <mesh_term>Glomerulonephritis, Membranoproliferative</mesh_term>
    </condition_browse>
    <patient_data>
      <sharing_ipd>No</sharing_ipd>
    </patient_data>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: download date: ClinicalTrials.gov processed this data on September 20, 2023
  link text: Link to the current ClinicalTrials.gov record.
  url: https://clinicaltrials.gov/ct2/show/NCT05985122
  org study id: COMPRare-IRFMN
  nct id: NCT05985122
  lead sponsor: 
  has dmc: No
  is fda regulated drug: No
  is fda regulated device: No
  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.
  Atypical Hemolytic Uremic Syndrome (aHUS) and C3 Glomerulopathies (C3G) are ultra-rare
        conditions in which an uncontrolled complement activation results in renal inflammation with
        thrombotic microangiopathy (TMA) in aHUS and extensive deposition of complement fragments in
        the kidneys in C3G. In both conditions, these alterations lead to acute and chronic kidney
        damage and, ultimately, end-stage renal failure. The prevalence of genetic and acquired
        complement abnormalities varies between the two diseases, but covers mostly the same
        complement proteins. Blocking the complement system is the treatment of choice in aHUS and
        may have considerable potential in C3G, as evident from the increasing number of phase 3
        trials with complement inhibitory drugs. Timely and accurate diagnosis is still challenging
        for clinicians, both for aHUS, to differentiate it from other forms of TMA, and for C3G, to
        distinguish it from other forms of glomerulonephritis (GN). In C3G, kidney biopsy is the
        diagnostic gold standard but it is not sufficient. Therefore, for both aHUS and C3G, there is
        an unmet need for accurate testing of genetic and acquired complement abnormalities.
        Moreover, interlaboratory complement-assays, in particular the tests for the C3 Nephritic
        Factors (C3NeFs; heterogeneous autoantibodies that stabilize the C3 convertase), vary
        considerably and, consequently, their standardization is a great necessity. Furthermore, the
        interpretation of genetic results in aHUS and C3G is not always conclusive.
  
        Variants of Unknown Significance (VUS)/Likely Pathogenic Variant (LPV) in complement genes
        are often found in aHUS and C3G and require further characterization. A rapid and functional
        VUS/LPV determination and interpretation could help to better understand the disease
        pathophysiology and might influence diagnosis and treatment options.
  
        Currently, aHUS patients are effectively treated with the complement C5 inhibiting monoclonal
        antibodies Eculizumab and Ravulizumab. However, the duration and optimal dose of treatment
        are debated, particularly in view of the high costs of the drug. Even if recent studies
        showed that restrictive use of eculizumab is feasible, there is a clear need for biomarkers
        identification and protocols for monitoring and predicting disease activity in aHUS and C3G
        patients.
  
        In summary, new analytic tools for further defining the disease profile in blood, (patients)
        cells and kidney biopsies are needed.
  allocation: Non-Randomized
  intervention model: Parallel Assignment
  intervention model description: The samples (healthy volunteers and HUS or MPGN patients) for the study will be identified between those of patients and healthy controls who provided consent to store their samples in the certified biobank of "Centro Daccò" (Centro di Risorse Biologiche Mario Negri - Biobanca Malattie Rare e Renali) for two previousely approved studies.
  primary purpose: Diagnostic
  masking: None (Open Label)
  measure: Determination of assay sensitivity and specificity
  time frame: At day 0
  description: We estimate that 38 VUS/LPV carrying pedigrees should be enough to demonstrate the sensitivity and specificity of the assay for classifying VUS/LPV.
  arm group label: Patients
  arm group type: Experimental
  arm group label: Healthy controls
  arm group type: Active Comparator
  intervention type: Diagnostic Test
  intervention name: C3NEF assay
  description: This assay will consist of a dual test, detecting C3 convertase binding C3NEF autoantibodies and measuring the functional consequence by complement alternative pathway (AP) activity using two distinct ELISA designs: C3NEF detection assay and AP activity assay.
  arm group label: Healthy controls
  arm group label: Patients
  criteria: 
  gender: All
  minimum age: N/A
  maximum age: N/A
  healthy volunteers: Accepts Healthy Volunteers
  last name: Marina Noris, PhD
  role: Principal Investigator
  affiliation: Istituto di Ricerche Farmacologiche Mario Negri IRCCS
  last name: Marina Noris, PhD
  phone: +3903545351
  email: marina.noris@marionegri.it
  last name: Erica Daina
  phone: +3903545351
  email: erica.daina@marionegri.it
  facility: 
  contact: 
  country: Italy
  citation: Noris M, Galbusera M, Gastoldi S, Macor P, Banterla F, Bresin E, Tripodo C, Bettoni S, Donadelli R, Valoti E, Tedesco F, Amore A, Coppo R, Ruggenenti P, Gotti E, Remuzzi G. Dynamics of complement activation in aHUS and how to monitor eculizumab therapy. Blood. 2014 Sep 11;124(11):1715-26. doi: 10.1182/blood-2014-02-558296. Epub 2014 Jul 18.
  PMID: 25037630
  citation: Iatropoulos P, Daina E, Curreri M, Piras R, Valoti E, Mele C, Bresin E, Gamba S, Alberti M, Breno M, Perna A, Bettoni S, Sabadini E, Murer L, Vivarelli M, Noris M, Remuzzi G; Registry of Membranoproliferative Glomerulonephritis/C3 Glomerulopathy; Nastasi. Cluster Analysis Identifies Distinct Pathogenetic Patterns in C3 Glomerulopathies/Immune Complex-Mediated Membranoproliferative GN. J Am Soc Nephrol. 2018 Jan;29(1):283-294. doi: 10.1681/ASN.2017030258. Epub 2017 Oct 13.
  PMID: 29030465
  citation: Donadelli R, Pulieri P, Piras R, Iatropoulos P, Valoti E, Benigni A, Remuzzi G, Noris M. Unraveling the Molecular Mechanisms Underlying Complement Dysregulation by Nephritic Factors in C3G and IC-MPGN. Front Immunol. 2018 Oct 15;9:2329. doi: 10.3389/fimmu.2018.02329. eCollection 2018.
  PMID: 30487789
  citation: Galbusera M, Noris M, Gastoldi S, Bresin E, Mele C, Breno M, Cuccarolo P, Alberti M, Valoti E, Piras R, Donadelli R, Vivarelli M, Murer L, Pecoraro C, Ferrari E, Perna A, Benigni A, Portalupi V, Remuzzi G. An Ex Vivo Test of Complement Activation on Endothelium for Individualized Eculizumab Therapy in Hemolytic Uremic Syndrome. Am J Kidney Dis. 2019 Jul;74(1):56-72. doi: 10.1053/j.ajkd.2018.11.012. Epub 2019 Mar 7.
  PMID: 30851964
  citation: Piras R, Breno M, Valoti E, Alberti M, Iatropoulos P, Mele C, Bresin E, Donadelli R, Cuccarolo P, Smith RJH, Benigni A, Remuzzi G, Noris M. CFH and CFHR Copy Number Variations in C3 Glomerulopathy and Immune Complex-Mediated Membranoproliferative Glomerulonephritis. Front Genet. 2021 Jun 11;12:670727. doi: 10.3389/fgene.2021.670727. eCollection 2021.
  PMID: 34211499
  citation: Aiello S, Gastoldi S, Galbusera M, Ruggenenti P, Portalupi V, Rota S, Rubis N, Liguori L, Conti S, Tironi M, Gamba S, Santarsiero D, Benigni A, Remuzzi G, Noris M. C5a and C5aR1 are key drivers of microvascular platelet aggregation in clinical entities spanning from aHUS to COVID-19. Blood Adv. 2022 Jan 8;6(3):866-881. doi: 10.1182/bloodadvances.2021005246. Erratum In: Blood Adv. 2022 May 10;6(9):2812.
  PMID: 34852172
  citation: Piras R, Valoti E, Alberti M, Bresin E, Mele C, Breno M, Liguori L, Donadelli R, Rigoldi M, Benigni A, Remuzzi G, Noris M. CFH and CFHR structural variants in atypical Hemolytic Uremic Syndrome: Prevalence, genomic characterization and impact on outcome. Front Immunol. 2023 Jan 30;13:1011580. doi: 10.3389/fimmu.2022.1011580. eCollection 2022.
  PMID: 36793547
  citation: Gastoldi S, Aiello S, Galbusera M, Breno M, Alberti M, Bresin E, Mele C, Piras R, Liguori L, Santarsiero D, Benigni A, Remuzzi G, Noris M. An ex vivo test to investigate genetic factors conferring susceptibility to atypical haemolytic uremic syndrome. Front Immunol. 2023 Feb 9;14:1112257. doi: 10.3389/fimmu.2023.1112257. eCollection 2023.
  PMID: 36845135
  responsible party type: Sponsor
  mesh term: Glomerulonephritis
  mesh term: Hemolytic-Uremic Syndrome
  mesh term: Glomerulonephritis, Membranoproliferative
  sharing ipd: No