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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT01522183
Ethics application status
Date submitted
18/01/2012
Date registered
31/01/2012
Date last updated
3/05/2019

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
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
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

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 - To collect and evaluate safety and effectiveness data specific to the use of eculizumab in aHUS patients.
Timepoint [1] 0 0
10 years
Primary outcome [2] 0 0
Time to first and subsequent occurrence of specified events. - 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 treatment or other disease management approaches
Timepoint [2] 0 0
10 years

Eligibility
Key inclusion criteria
- Male or female patients of any age, including minors, who have been diagnosed with
aHUS

- 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
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Hemolytic Uremic Syndrome (HUS) only due to Shiga Toxin

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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Nepean Hospital - Penrith
Recruitment hospital [2] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [3] 0 0
Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Cairns Base Hospital - Cairns
Recruitment hospital [5] 0 0
Royal Brisbane & Women's Hospital - Herston
Recruitment hospital [6] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [7] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [8] 0 0
Austin Health - Heidelberg
Recruitment hospital [9] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [10] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [11] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment hospital [12] 0 0
Sir Charles Gairdner Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
- Penrith
Recruitment postcode(s) [2] 0 0
- Westmead
Recruitment postcode(s) [3] 0 0
- Cairns
Recruitment postcode(s) [4] 0 0
- Herston
Recruitment postcode(s) [5] 0 0
- Woolloongabba
Recruitment postcode(s) [6] 0 0
- Adelaide
Recruitment postcode(s) [7] 0 0
- Heidelberg
Recruitment postcode(s) [8] 0 0
- Parkville
Recruitment postcode(s) [9] 0 0
- Murdoch
Recruitment postcode(s) [10] 0 0
- 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
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
Belgium
State/province [9] 0 0
Brussels
Country [10] 0 0
Belgium
State/province [10] 0 0
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
Country [18] 0 0
France
State/province [18] 0 0
Amiens
Country [19] 0 0
France
State/province [19] 0 0
Angers
Country [20] 0 0
France
State/province [20] 0 0
Bayonne
Country [21] 0 0
France
State/province [21] 0 0
Bordeaux
Country [22] 0 0
France
State/province [22] 0 0
Brest
Country [23] 0 0
France
State/province [23] 0 0
Bron
Country [24] 0 0
France
State/province [24] 0 0
Caen
Country [25] 0 0
France
State/province [25] 0 0
Chambéry
Country [26] 0 0
France
State/province [26] 0 0
Clermont-Ferrand
Country [27] 0 0
France
State/province [27] 0 0
Dijon
Country [28] 0 0
France
State/province [28] 0 0
Grenoble
Country [29] 0 0
France
State/province [29] 0 0
Le Kremlin-Bicêtre
Country [30] 0 0
France
State/province [30] 0 0
Lille
Country [31] 0 0
France
State/province [31] 0 0
Lyon
Country [32] 0 0
France
State/province [32] 0 0
Marseille
Country [33] 0 0
France
State/province [33] 0 0
Montpellier
Country [34] 0 0
France
State/province [34] 0 0
Nancy
Country [35] 0 0
France
State/province [35] 0 0
Nantes
Country [36] 0 0
France
State/province [36] 0 0
Nice
Country [37] 0 0
France
State/province [37] 0 0
Paris
Country [38] 0 0
France
State/province [38] 0 0
Perpignan
Country [39] 0 0
France
State/province [39] 0 0
Poitiers
Country [40] 0 0
France
State/province [40] 0 0
Pontoise
Country [41] 0 0
France
State/province [41] 0 0
Rennes
Country [42] 0 0
France
State/province [42] 0 0
Rouen
Country [43] 0 0
France
State/province [43] 0 0
Strasbourg
Country [44] 0 0
France
State/province [44] 0 0
Toulouse
Country [45] 0 0
France
State/province [45] 0 0
Tours
Country [46] 0 0
France
State/province [46] 0 0
Valenciennes
Country [47] 0 0
Germany
State/province [47] 0 0
Essen
Country [48] 0 0
Germany
State/province [48] 0 0
Hannover
Country [49] 0 0
Germany
State/province [49] 0 0
Heidelberg
Country [50] 0 0
Germany
State/province [50] 0 0
Luebeck
Country [51] 0 0
Germany
State/province [51] 0 0
Münster
Country [52] 0 0
Germany
State/province [52] 0 0
Tübingen
Country [53] 0 0
Israel
State/province [53] 0 0
Be'er Sheva
Country [54] 0 0
Italy
State/province [54] 0 0
Bari
Country [55] 0 0
Italy
State/province [55] 0 0
Firenze
Country [56] 0 0
Italy
State/province [56] 0 0
Genova
Country [57] 0 0
Italy
State/province [57] 0 0
Milano
Country [58] 0 0
Italy
State/province [58] 0 0
Roma
Country [59] 0 0
Italy
State/province [59] 0 0
Torino
Country [60] 0 0
Korea, Republic of
State/province [60] 0 0
Gyeonggi-do
Country [61] 0 0
Korea, Republic of
State/province [61] 0 0
Gyeongsang
Country [62] 0 0
Korea, Republic of
State/province [62] 0 0
Seoul
Country [63] 0 0
Poland
State/province [63] 0 0
Lódz
Country [64] 0 0
Russian Federation
State/province [64] 0 0
Ekaterinburg
Country [65] 0 0
Russian Federation
State/province [65] 0 0
Khabarovsk
Country [66] 0 0
Russian Federation
State/province [66] 0 0
Moscow
Country [67] 0 0
Russian Federation
State/province [67] 0 0
Saint Petersburg
Country [68] 0 0
Spain
State/province [68] 0 0
Barcelona
Country [69] 0 0
Spain
State/province [69] 0 0
Pamplona
Country [70] 0 0
Spain
State/province [70] 0 0
Sevilla
Country [71] 0 0
Taiwan
State/province [71] 0 0
Kaohsiung
Country [72] 0 0
Taiwan
State/province [72] 0 0
New Taipei City
Country [73] 0 0
Taiwan
State/province [73] 0 0
Taichung
Country [74] 0 0
Taiwan
State/province [74] 0 0
Taipei
Country [75] 0 0
United Kingdom
State/province [75] 0 0
Exeter
Country [76] 0 0
United Kingdom
State/province [76] 0 0
London
Country [77] 0 0
United Kingdom
State/province [77] 0 0
Manchester
Country [78] 0 0
United Kingdom
State/province [78] 0 0
Newcastle
Country [79] 0 0
United Kingdom
State/province [79] 0 0
Southampton
Country [80] 0 0
United Kingdom
State/province [80] 0 0
Wales

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals
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.
Trial website
https://clinicaltrials.gov/show/NCT01522183
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Benjamin Miller, MD
Address 0 0
Alexion Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Caroline Collupy
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
ahus-registry@syneoshealth.com
Contact person for scientific queries

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