An Observational, Multi-Center, Multi-National, Long Term Follow-Up Study of Atypical Hemolytic Uremic Syndrome (aHUS) Patients Treated With Eculizumab in a Prior Clinical Study

aHUS Observational Long Term Follow-Up


Lead sponsor: Alexion Pharmaceuticals

Source Alexion Pharmaceuticals
Brief Summary

There is growing but limited information on the long term clinical status of aHUS patients who have previously received or are continuing to receive treatment with eculizumab. This study is designed to collect clinical data that will provide insight into the long-term outcomes of patients with aHUS.

Overall Status Terminated
Start Date March 2012
Completion Date January 2017
Primary Completion Date January 2017
Study Type Observational [Patient Registry]
Primary Outcome
Measure Time Frame
TMA complication-free survival 5 Years
Secondary Outcome
Measure Time Frame
Duration of response, change over time of value for platelets, LDH, eGFR, Hgb, TMA intervention 5 Years
Enrollment 94

Sampling method: Non-Probability Sample


Inclusion Criteria:

- aHUS patients who participated in any one of the aHUS-eculizumab clinical studies.

- aHUS patients or legal representative who are able and willing to given written informed consent for their study information to be collected and retained in a database.

Exclusion Criteria:

- Not applicable.

Gender: All

Minimum age: N/A

Maximum age: N/A

Healthy volunteers: No

Emory Healthcare - Children's Center | Atlanta, Georgia, United States
Dunwoody Pediatrics & Children's Healthcare of Atlanta | Dunwoody, Georgia, United States
Fort Wayne Medical Oncology and Hematology | Fort Wayne, Indiana, United States
| Fort Wayne, Indiana, United States
Boston Children's Hospital | Boston, Massachusetts, United States
Henry Ford Health System | Detroit, Michigan, United States
Children's Hospital at Montefiore | Bronx, New York, United States
Hudson Valley Oncology Hematology | Hawthorne, New York, United States
Weill Cornell Medical College | New York, New York, United States
Cincinnati Children's Hospital | Cincinnati, Ohio, United States
Arthur James Cancer Hospital | Columbus, Ohio, United States
Driscoll Children's Hospital | Corpus Christi, Texas, United States
The Methodist Hospital Research Institute | Houston, Texas, United States
Seattle Children's Hospital | Seattle, Washington, United States
Providence Sacred Heart Medical Center & Children's Hospital | Spokane, Washington, United States
Fox Valley Hematology | Osh Kosh, Wisconsin, United States
Royal Adelaide Hospital | North Tce. Adelaide, Australia
Universitatsklinik fur Innere Medizin Medizinische Universitat Graz | Graz, Austria
Medizinische Universitaet Innsbruck | Innsbruck, Austria
UZ Gent Dienst nefrologie | Gent, Belgium
Chu Sart Tilman | Liège, Belgium
Centre Hospitalier Universitaire (CHU) Sainte-Justine | Montreal, Canada
The Hospital for Sick Children | Toronto, Canada
Centre Hospitalier Universitaire Pellegrin, Service de Nephrologie Transplantation Dialyse | Bordeaux Cedex, France
CHRU de Caen | Caen, France
Le Kremlin Bicetre Hospital, Nephrology Unit | Le Kremlin Bicetre, France
CHRU de Lille-Hopital A.Calmette | Lille, France
Hôpital Edouard Herriot | Lyon, France
Hopital de la Timone Enfants, Unité de Néphrologie | Marseille, France
CHU Hotel Dieu | Nantes, France
Le Centre Hospitalier Universitaire de Nice | Nice, France
Centre Hospitalier Regional de la Source, Service de nephrologie/hemodialyse | Orleans, France
Hopital Robert-Debré, Service de Néphrologie Pédiatrique | Paris, France
Hopital Tenon | Paris, France
Centre Hospitalier Inter-Communal de Cornouaille | Quimper, France
Hôpital de Bois Guillaume CHU de Rouen | Rouen Cedex, France
CHU-CH Charles Nicolle Pavillon de Pédiatrie | Rouen, France
CHU de Saint-Etienne | Saint Priest En Jarez, France
Nouvel Hopital Civil | Strasbourg, France
CHRU de Tours | Tours, France
Universitaetsklinikum Aachen Klinik fuer Nieren- und Hochdruckkrankheiten | Aachen, Germany
Hannover University Clinic | Hannover, Germany
Universitaet Heidelberg | Heidelberg, Germany
A.O. Papa Giovanni XXIII | Bergamo, Italy
Azienda Ospedaliero-Universitaria Careggi | Firenze, Italy
Istituto Giannina Gaslini | Genova, Italy
Fondazione IRCCS Ca Granda, Ospedale Maggiore Policlinico Maggiore Policlinico | Milano, Italy
Ospedale dei Bambini G. di Cristina | Palermo, Italy
AMC Medical Research B.V. | Amsterdam, Netherlands
Radboud University Nijmegen Medical Centre Nijmegen | Nijmegen, Netherlands
Nephrology Clinic, Karolinska University Hospital | Stockholm, Sweden
INSELSPITAL Universitaetsklinik fuer Kinderheilkunde Kindernephrologie | Bern, Switzerland
Royal Devon & Exeter NHS Foundation Trust | Exeter, United Kingdom
Gartnavel General Hospital | Glasgow, United Kingdom
The Newcastle upon Tyne Hospitals NHS | Newcastle upon Tyne, United Kingdom
City Hospital, Nottingham University Hospitals, NHS Trust | Nottingham, United Kingdom
Location Countries











United Kingdom

United States

Verification Date

February 2017

Responsible Party

Responsible party type: Sponsor

Has Expanded Access No
Condition Browse
Acronym LTFU
Study Design Info

Observational model: Case-Only

Time perspective: Other