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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01522183




Registration number
NCT01522183
Ethics application status
Date submitted
18/01/2012
Date registered
31/01/2012
Date last updated
18/07/2024

Titles & IDs
Public title
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
Scientific title
An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Secondary ID [1] 0 0
M11-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Atypical Hemolytic-Uremic Syndrome 0 0
Condition category
Condition code
Blood 0 0 0 0
Other blood disorders
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Blood 0 0 0 0
Anaemia
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of patients who experience specified events
Timepoint [1] 0 0
10 years
Primary outcome [2] 0 0
Time to first and subsequent occurrence of specified events.
Timepoint [2] 0 0
5 years

Eligibility
Key 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.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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]).

Study design
Purpose
Duration
Selection
Timing
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/04/2012
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2031
Actual
Sample size
Target
3000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Clinical Trial Site 1 - Westmead
Recruitment hospital [2] 0 0
Clinical Trial Site 2 - Westmead
Recruitment hospital [3] 0 0
Clinical Trial Site - Cairns
Recruitment hospital [4] 0 0
Clinical Trial Site - Herston
Recruitment hospital [5] 0 0
Clinical Trial Site - Woolloongabba
Recruitment hospital [6] 0 0
Clinical Trial Site - Adelaide
Recruitment hospital [7] 0 0
Clinical Trial Site - Heidelberg
Recruitment hospital [8] 0 0
Clinical Trial Site - Parkville
Recruitment hospital [9] 0 0
Clinical Trial Site - Murdoch
Recruitment hospital [10] 0 0
Clinical Trial Site - Nedlands
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4870 - Cairns
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
3084 - Heidelberg
Recruitment postcode(s) [7] 0 0
3050 - Parkville
Recruitment postcode(s) [8] 0 0
3052 - Parkville
Recruitment postcode(s) [9] 0 0
6961 - Murdoch
Recruitment postcode(s) [10] 0 0
6109 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
District of Columbia
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
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Georgia
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United States of America
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Illinois
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United States of America
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Massachusetts
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United States of America
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Missouri
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United States of America
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North Carolina
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United States of America
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Ohio
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Belgium
State/province [9] 0 0
Brussels
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Belgium
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Edegem
Country [11] 0 0
Belgium
State/province [11] 0 0
Gent
Country [12] 0 0
Belgium
State/province [12] 0 0
Leuven
Country [13] 0 0
Belgium
State/province [13] 0 0
Liège
Country [14] 0 0
Belgium
State/province [14] 0 0
Woluwe-Saint-Lambert
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec
Country [17] 0 0
Denmark
State/province [17] 0 0
Odense
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France
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Amiens
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France
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Angers
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France
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Bayonne
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France
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Bordeaux
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France
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Brest
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France
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Bron
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France
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Chambéry
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France
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Clermont-Ferrand
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France
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Dijon
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France
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Grenoble
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France
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Le Kremlin-Bicêtre
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France
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Lille
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France
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Lyon
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France
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Marseille
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France
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Montpellier
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France
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Nancy
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France
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Nantes
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France
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Nice
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France
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Paris
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France
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Perpignan
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France
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Poitiers
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France
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Pontoise
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France
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Rennes
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France
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Rouen
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France
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Strasbourg
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France
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Toulouse
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France
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Tours
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France
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Valenciennes
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Germany
State/province [46] 0 0
Essen
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Germany
State/province [47] 0 0
Hannover
Country [48] 0 0
Germany
State/province [48] 0 0
Heidelberg
Country [49] 0 0
Germany
State/province [49] 0 0
Luebeck
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Germany
State/province [50] 0 0
Münster
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Germany
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Tübingen
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Israel
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Be'er Sheva
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Italy
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Bari
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Italy
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Firenze
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Italy
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Genova
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Italy
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Milano
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Italy
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Roma
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Italy
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Torino
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Korea, Republic of
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Chungcheongnam-do
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Korea, Republic of
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Dongnam-ro
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Korea, Republic of
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Gyeonggi-do
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Korea, Republic of
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Hyeonchung-ro
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Korea, Republic of
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Jebong Ro
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Korea, Republic of
State/province [64] 0 0
Changwon
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Korea, Republic of
State/province [65] 0 0
Seoul
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Poland
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Lódz
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Russian Federation
State/province [67] 0 0
Moscow
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Russian Federation
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Saint Petersburg
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Spain
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Barcelona
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Spain
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Pamplona
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Spain
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Sevilla
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Taiwan
State/province [72] 0 0
Kaohsiung
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Taiwan
State/province [73] 0 0
New Taipei City
Country [74] 0 0
Taiwan
State/province [74] 0 0
Taichung
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Taiwan
State/province [75] 0 0
Taipei
Country [76] 0 0
United Kingdom
State/province [76] 0 0
Exeter
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United Kingdom
State/province [77] 0 0
London
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United Kingdom
State/province [78] 0 0
Manchester
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United Kingdom
State/province [79] 0 0
Newcastle
Country [80] 0 0
United Kingdom
State/province [80] 0 0
Southampton
Country [81] 0 0
United Kingdom
State/province [81] 0 0
Wales

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Syneos Health
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Post-marketing safety data on patients treated and untreated with eculizumab or ravulizumab.
Trial website
https://clinicaltrials.gov/study/NCT01522183
Trial related presentations / publications
Halimi JM, Al-Dakkak I, Anokhina K, Ardissino G, Licht C, Lim WH, Massart A, Schaefer F, Walle JV, Rondeau E. Clinical characteristics and outcomes of a patient population with atypical hemolytic uremic syndrome and malignant hypertension: analysis from the Global aHUS registry. J Nephrol. 2023 Apr;36(3):817-828. doi: 10.1007/s40620-022-01465-z. Epub 2022 Sep 24.
Woodward L, Johnson S, Walle JV, Beck J, Gasteyger C, Licht C, Ariceta G; aHUS Registry SAB. An innovative and collaborative partnership between patients with rare disease and industry-supported registries: the Global aHUS Registry. Orphanet J Rare Dis. 2016 Nov 21;11(1):154. doi: 10.1186/s13023-016-0537-5.
Licht C, Ardissino G, Ariceta G, Cohen D, Cole JA, Gasteyger C, Greenbaum LA, Johnson S, Ogawa M, Schaefer F, Vande Walle J, Fremeaux-Bacchi V. The global aHUS registry: methodology and initial patient characteristics. BMC Nephrol. 2015 Dec 10;16:207. doi: 10.1186/s12882-015-0195-1.
Belingheri M, Possenti I, Tel F, Paglialonga F, Testa S, Salardi S, Ardissino G. Cryptic activity of atypical hemolytic uremic syndrome and eculizumab treatment. Pediatrics. 2014 Jun;133(6):e1769-71. doi: 10.1542/peds.2013-2921. Epub 2014 May 19.
Ardissino G, Possenti I, Tel F, Testa S, Paglialonga F. Time to change the definition of hemolytic uremic syndrome. Eur J Intern Med. 2014 Feb;25(2):e29. doi: 10.1016/j.ejim.2013.12.002. Epub 2013 Dec 21. No abstract available.
Public notes

Contacts
Principal investigator
Name 0 0
Nuria Saval
Address 0 0
Alexion Pharmaceuticals, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Alexion Pharmaceuticals, Inc. (Sponsor)
Address 0 0
Country 0 0
Phone 0 0
1-855-752-2356
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01522183