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Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Sponsor
Alexion Pharmaceuticals (Industry)
Overall Status
Recruiting
CT.gov ID
NCT01522183
Collaborator
Syneos Health (Other)
3,000
Enrollment
131
Locations
164
Anticipated Duration (Months)
22.9
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    The study will capture post-marketing safety data on patients treated with eculizumab or ravulizumab. Additionally, the study will collect information on the progression of disease in all patients.

    Study Design

    Study Type:
    Observational [Patient Registry]
    Anticipated Enrollment :
    3000 participants
    Observational Model:
    Other
    Time Perspective:
    Other
    Official Title:
    An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
    Actual Study Start Date :
    Apr 30, 2012
    Anticipated Primary Completion Date :
    Dec 31, 2023
    Anticipated Study Completion Date :
    Dec 31, 2025

    Outcome Measures

    Primary Outcome Measures

    1. Proportion of patients who experience specified events [10 years]

      To collect and evaluate safety and effectiveness data specific to the use of eculizumab or ravulizumab in aHUS patients.

    2. Time to first and subsequent occurrence of specified events. [5 years]

      To assess the long term manifestations of thrombotic microangiopathy (TMA) complications of aHUS as well as other clinical outcomes, including morbidity and mortality in aHUS patients, receiving eculizumab or ravulizumab treatment or other disease management approaches

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    N/A and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Male or female patients of any age, including minors, who have been diagnosed with aHUS

    • Patients with or without an identified complement pathogenic variant or anti-complement factor antibody

    • Able to give written informed consent. Patient or patient's parent/legal guardian must be willing and able to given written informed consent and the patient (if minor) must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].

    • ADAMTS13 > 5%, if performed.

    Exclusion Criteria:

    • Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin producing Escherichia coli (STEC).

    • Unable to give written informed consent. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees [IRB/IEC]).

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Georgetown University Hospital Washington District of Columbia United States 20007
    2 University of Florida, Division of Nephrology Gainesville Florida United States 32610
    3 Emory Children's Center Emory University Atlanta Georgia United States 30322
    4 Children's Memorial Hospital Chicago Illinois United States 60614
    5 Brigham and Women's Hospital Boston Massachusetts United States 02115
    6 Washington University School of Medicine Saint Louis Missouri United States 63110
    7 Brookview Hills Research Associates LLC Winston-Salem North Carolina United States 27103
    8 Arthur James Cancer Hospital Columbus Ohio United States 43210
    9 Nepean Hospital Penrith New South Wales Australia
    10 The Children's Hospital at Westmead Westmead New South Wales Australia
    11 Westmead Hospital Westmead New South Wales Australia
    12 Cairns Base Hospital Cairns Queensland Australia
    13 Royal Brisbane & Women's Hospital Herston Queensland Australia
    14 Princess Alexandra Hospital Woolloongabba Queensland Australia
    15 Royal Adelaide Hospital Adelaide South Australia Australia
    16 Austin Health Heidelberg Victoria Australia
    17 The Royal Children's Hospital Parkville Victoria Australia
    18 The Royal Melbourne Hospital Parkville Victoria Australia
    19 Fiona Stanley Hospital Murdoch Western Australia Australia
    20 Sir Charles Gairdner Hospital Nedlands Western Australia Australia
    21 Hopital Erasme Brussels Belgium
    22 Hopital Universitaire des Enfants Reine Brussels Belgium
    23 UZ Brussels Brussels Belgium
    24 UZA (Adult) Edegem Belgium
    25 UZA (Pediatric) Edegem Belgium
    26 UZ Gent (pediatric) Gent Belgium
    27 University Hospital Leuven (Adult) Leuven Belgium
    28 University Hospital Leuven (pediatric) Leuven Belgium
    29 CHU de Liege - Clinique de l'Esperance (pediatric) Liège Belgium
    30 CHU de Liege - Sart Tilman Liège Belgium
    31 Cliniques Universitaires Saint-Luc (adult) Woluwe-Saint-Lambert Belgium
    32 Cliniques Universitaires Saint-Luc (Pediatric) Woluwe-Saint-Lambert Belgium
    33 London Health Science Center London Ontario Canada
    34 The Hospital for Sick Children Toronto Ontario Canada
    35 CHU Ste Justine Montréal Quebec Canada
    36 CHU de Quebec Hotel-Dieu de Quebec Québec Quebec Canada
    37 Odense University Hospital Odense Denmark
    38 CH Amiens - Hopital Nord Amiens France
    39 Hopital Sud Amiens France
    40 CHU Angers Angers France
    41 CHG Bayonne Bayonne France
    42 CHU de Bordeaux Pellegrin Bordeaux France
    43 CHRU Brest Brest France
    44 Hopital Femme Mere Enfant Bron France
    45 CHRU de Caen Hopital Clemenceau Service de Nephrologie Caen France
    46 Centre Hospitialier Regional de Chambery Chambéry France
    47 CHU Clermont Ferrand Clermont-Ferrand France
    48 CHU Dijon Dijon France
    49 CHU Grenoble Grenoble France
    50 Le Kremlin Bicetre Hospital Le Kremlin-Bicêtre France
    51 CHRU de Lille Hopital A Calmette Lille France
    52 CHRU de Lille Hopital Jeanne de Flandre Lille France
    53 Hopital Edouard Herriot (CHU Lyon) Lyon France
    54 Hopital de la Timone Enfants Marseille France
    55 CHU de Montpellier Montpellier France
    56 Hopital Brabois Nancy France
    57 Hopital de Nancy Nancy France
    58 CHU de Nantes-Hopital Hotel Dieu Nantes France
    59 Hopital Enfants et Adolescents Nantes France
    60 Le Centre Hospitialier Universitarie de Nice Hopital Pasteur Nice France
    61 HEGP - Hopital Europeen Georges Pompidou Paris France
    62 Hopital Necker, Enfants Malades Service de Nephrologie Pediatrique Paris France
    63 Hopital Necker Paris France
    64 Hopital Robert Debre Service de Nephrologie Pediatrique Paris France
    65 Hopital Tenon Paris France
    66 CH Perpignan Perpignan France
    67 Hopital Poitiers Poitiers France
    68 CHU de Pontoise Pontoise France
    69 CHU de Rennes Ponchaillou Rennes France
    70 CHU Rennes, Hopital Sud Rennes France
    71 CHU-CH Charles Nicolle Rouen France
    72 Hopital de Bois Guillaume CHU de Rouen Rouen France
    73 Hopital Civil de Strasbourg Strasbourg France
    74 Nouvel Hopital Civil Strasbourg France
    75 CHU Rangueil Toulouse France
    76 Hopital des Enfants Toulouse France
    77 CHFU de Tours Tours France
    78 CH Valenciennes Valenciennes France
    79 Universitaetsklinikum Essen Essen Germany
    80 Medizinische Hochschule Hannover Adult Nephrology Hannover Germany
    81 Medizinische Hochschule Hannover Pediatric Nephrology Hannover Germany
    82 Universitaet Heidelberg Heidelberg Germany
    83 Universtaetskilinkum Schlkeswig-Holstein Luebeck Germany
    84 Universitaetskilinikum Muenster Münster Germany
    85 Universitaetsklinikum Tuebingen Tübingen Germany
    86 Soroka University Medical Center Be'er Sheva Israel
    87 Policlinico Consorziale de Bari Bari Italy
    88 U O Nefrologia e Dialisi Pediatrica Ospedale Giovanni XXIII Bari Italy
    89 Azienda Ospedaliero - Universitaria Careggi Firenze Italy
    90 Istituto Giannina Gaslini Genova Italy
    91 Fondazione IRCCS Ca Granda, Ospedale Maggiore Policlinico Milano Italy
    92 Pad Croff Nefrologia Ospedale Maggiore Policlinico Milano Italy
    93 S C Nefrologia - A O Ospedale Niguarda-Ca Granda Milano Italy
    94 Dipartmento di Nefrologia e Urologia Ospedale Pediatrico Bambin Gesu Roma Italy
    95 Ospediale Pediatrico Regina Margherita Torino Italy
    96 Soon ChunHyang University Hospital Cheonan Dongan Chungcheongnam-do Korea, Republic of 31538
    97 Kyung Hee University Hospital at Gangdong Seoul Dongnam-ro Korea, Republic of 02447
    98 Hallym University Sacred Heart Hospital Anyang-si Gyeonggi-do Korea, Republic of 14068
    99 Inje University IlsanPaik Hospital Goyang-si Gyeonggi-do Korea, Republic of 411-706
    100 Yeungnam University Hospital Daegu Hyeonchung-ro Korea, Republic of 42415
    101 Chonnam National University Hospital Gwangju Jebong Ro Korea, Republic of 61469
    102 CHA Bundang Medical Center, CHA University Gyeonggi-do Korea, Republic of
    103 The Catholic University of Korea, Uijeongnu St Mary's Hospital Gyeonggi-do Korea, Republic of
    104 Pusan National University Yangsan Hospital Gyeongsang Korea, Republic of
    105 Asan Medical Center Seoul Korea, Republic of
    106 Esha Womans University Medical Center Seoul Korea, Republic of
    107 Korea University Anam Hospital Seoul Korea, Republic of
    108 Korea University Guro Hospital Seoul Korea, Republic of
    109 Samsung Medical Center Seoul Korea, Republic of
    110 Seoul National University Hospital Seoul Korea, Republic of
    111 Severance Hospital Yonsei University Health System Seoul Korea, Republic of
    112 NZOZ - TRI Medica Łódź Poland
    113 Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Klinkzny Łódź Poland
    114 Children's City Clinical Hospital of Saint Vladimir Moscow Russian Federation
    115 City Clinical Hospital #52 Moscow Russian Federation
    116 Saint Petersburg Medical University Saint Petersburg Russian Federation
    117 H. Valle Hebron Barcelona Spain
    118 Complejo Hospitalario de Navarra Pamplona Spain
    119 Hospital Virgen del Rocio Sevilla Spain
    120 Veteran's General Hospital - Kaohsiung (VGH-KS) Kaohsiung Taiwan
    121 Chang Cung Memorial Hospital - LK (CGMH-LK) New Taipei City Taiwan
    122 China Medical University Hospital (CMUH) Taichung Taiwan
    123 National Taiwan University Hospital (NTUH) Taipei Taiwan
    124 Royal Devon & Exeter NHS Foundation Trust Exeter United Kingdom
    125 Great Ormond Street Hospital London United Kingdom
    126 Royal Free Hospital London United Kingdom
    127 University College London Hospitals NHS Foundation Trust London United Kingdom
    128 Royal Manchester Children's Hospital Manchester United Kingdom
    129 Newcastle Upon Tyne Hospitals NHS Foundation Trust Newcastle United Kingdom
    130 Southampton General Hospital Southampton United Kingdom
    131 Morriston Hospital Wales United Kingdom

    Sponsors and Collaborators

    • Alexion Pharmaceuticals
    • Syneos Health

    Investigators

    • Study Director: Katerina Anokhina, MD, Alexion Pharmaceuticals

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Alexion Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT01522183
    Other Study ID Numbers:
    • M11-001
    First Posted:
    Jan 31, 2012
    Last Update Posted:
    Mar 16, 2022
    Last Verified:
    Mar 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Keywords provided by Alexion Pharmaceuticals
    Atypical Hemolytic-Uremic Syndrome
    aHUS
    Thrombotic Microangiopathy
    TMA
    Additional relevant MeSH terms:
    Azotemia
    Hemolytic-Uremic Syndrome
    Atypical Hemolytic Uremic Syndrome
    Syndrome
    Hemolysis

    Study Results

    No Results Posted as of Mar 16, 2022