Clinical trial NCT01522183: Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
Official title: An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With Atypical Hemolytic-Uremic Syndrome (aHUS Registry)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Atypical Hemolytic-Uremic Syndrome
Keywords: Atypical Hemolytic-Uremic Syndrome
aHUS
Thrombotic Microangiopathy
TMA
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Syneos Health
First posted: 2012-01-31
Enrollment: 3000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male or female patients of any age, including minors, who have been diagnosed with aHUS; clinical diagnosis of aHUS, patients with or without an identified complement regulatory factor genetic abnormality or anti-complement factor antibody, ADAMTS13 \> 5% (if performed)

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

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.

Eligibility criteria: 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 \[IRB/IEC\]).

Primary outcomes:
measure: Proportion of patients who experience specified events
timeFrame: 10 years
measure: Time to first and subsequent occurrence of specified events.
timeFrame: 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Aurora
country: United States
facility: Clinical Trial Site
state: Colorado
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Gainesville
country: United States
facility: Clinical Trial Site
state: Florida
city: Atlanta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Saint Louis
country: United States
facility: Clinical Trial Site
state: Missouri
city: Winston-Salem
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Westmead
country: Australia
facility: Clinical Trial Site 1
state: New South Wales
city: Westmead
country: Australia
facility: Clinical Trial Site 2
state: New South Wales
city: Cairns
country: Australia
facility: Clinical Trial Site
state: Queensland
city: Herston
country: Australia
facility: Clinical Trial Site
state: Queensland
city: Woolloongabba
country: Australia
facility: Clinical Trial Site
state: Queensland
city: Adelaide
country: Australia
facility: Clinical Trial Site
state: South Australia
city: Heidelberg
country: Australia
facility: Clinical Trial Site
state: Victoria
city: Parkville
country: Australia
facility: Clinical Trial Site
state: Victoria
city: Parkville
country: Australia
facility: Clinical Trial Site
state: Victoria
city: Murdoch
country: Australia
facility: Clinical Trial Site
state: Western Australia
city: Nedlands
country: Australia
facility: Clinical Trial Site
state: Western Australia
city: Brussels
country: Belgium
facility: Clinical Trial Site
city: Brussels
country: Belgium
facility: Clinical Trial Site
city: Brussels
country: Belgium
facility: Clinical Trial Site
city: Edegem
country: Belgium
facility: Clinical Trial Site (Adult)
city: Edegem
country: Belgium
facility: Clinical Trial Site (Pediatric)
city: Gent
country: Belgium
facility: Clinical Trial Site (pediatric)
city: Leuven
country: Belgium
facility: Clinical Trial Site (Adult)
city: Leuven
country: Belgium
facility: Clinical Trial Site (pediatric)
city: Liège
country: Belgium
facility: Clinical Trial Site (Adult)
city: Liège
country: Belgium
facility: Clinical Trial Site (pediatric)
city: Woluwe-Saint-Lambert
country: Belgium
facility: Clinical Trial Site (adult)
city: Woluwe-Saint-Lambert
country: Belgium
facility: Clinical Trial Site (Pediatric)
city: London
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Toronto
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Montréal
country: Canada
facility: Clinical Trial Site
state: Quebec
city: Québec
country: Canada
facility: Clinical Trial Site
state: Quebec
city: Odense
country: Denmark
facility: Clinical Trial Site
city: Amiens
country: France
facility: Clinical Trial Site 1
city: Amiens
country: France
facility: Clinical Trial Site 2
city: Angers
country: France
facility: Clinical Trial Site
city: Bayonne
country: France
facility: Clinical Trial Site
city: Bordeaux
country: France
facility: Clinical Trial Site
city: Brest
country: France
facility: Clinical Trial Site
city: Bron
country: France
facility: Clinical Trial Site
city: Caen
country: France
facility: Clinical Trial Site
city: Chambéry
country: France
facility: Clinical Trial Site
city: Clermont-Ferrand
country: France
facility: Clinical Trial Site
city: Dijon
country: France
facility: Clinical Trial Site
city: Grenoble
country: France
facility: Clinical Trial Site
city: Le Kremlin-Bicêtre
country: France
facility: Clinical Trial Site
city: Lille
country: France
facility: Clinical Trial Site
city: Lille
country: France
facility: Clinical Trial Site
city: Lyon
country: France
facility: Clinical Trial Site
city: Marseille
country: France
facility: Clinical Trial Site
city: Montpellier
country: France
facility: Clinical Trial Site
city: Nancy
country: France
facility: Clinical Trial Site 1
city: Nancy
country: France
facility: Clinical Trial Site
city: Nantes
country: France
facility: Clinical Trial Site
city: Nantes
country: France
facility: Clinical Trial Site 1
city: Nice
country: France
facility: Clinical Trial Site
city: Paris
country: France
facility: Clinical Trial Site 2
city: Paris
country: France
facility: Clinical Trial Site 4
city: Paris
country: France
facility: Clinical Trial Site 1
city: Paris
country: France
facility: Clinical Trial Site 5
city: Paris
country: France
facility: Clinical Trial Site 3
city: Perpignan
country: France
facility: Clinical Trial Site
city: Poitiers
country: France
facility: Clinical Trial Site
city: Pontoise
country: France
facility: Clinical Trial Site
city: Rennes
country: France
facility: Clinical Trial Site
city: Rennes
country: France
facility: Clinical Trial Site
city: Rouen
country: France
facility: Clinical Trial Site 1
city: Rouen
country: France
facility: Clinical Trial Site 2
city: Strasbourg
country: France
facility: Clinical Trial Site 1
city: Strasbourg
country: France
facility: Clinical Trial Site 2
city: Toulouse
country: France
facility: Clinical Trial Site
city: Toulouse
country: France
facility: Clinical Trial Site
city: Tours
country: France
facility: Clinical Trial Site
city: Valenciennes
country: France
facility: Clinical Trial Site
city: Essen
country: Germany
facility: Clinical Trial Site
city: Hannover
country: Germany
facility: Clinical Trial Site 1
city: Hannover
country: Germany
facility: Clinical Trial Site 2
city: Heidelberg
country: Germany
facility: Clinical Trial Site
city: Luebeck
country: Germany
facility: Clinical Trial Site
city: Münster
country: Germany
facility: Clinical Trial Site
city: Tübingen
country: Germany
facility: Clinical Trial Site
city: Be'er Sheva
country: Israel
facility: Clinical Trial Site
city: Bari
country: Italy
facility: Clinical Trial Site 1
city: Bari
country: Italy
facility: Clinical Trial Site 2
city: Firenze
country: Italy
facility: Clinical Trial Site
city: Genova
country: Italy
facility: Clinical Trial Site
city: Milano
country: Italy
facility: Clinical Trial Site 1
city: Milano
country: Italy
facility: Clinical Trial Site 2
city: Milano
country: Italy
facility: Clinical Trial Site
city: Roma
country: Italy
facility: Clinical Trial Site
city: Torino
country: Italy
facility: Clinical Trial Site
city: Dongan
country: Korea, Republic of
facility: Clinical Trial Site
state: Chungcheongnam-do
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
state: Dongnam-ro
city: Anyang-si
country: Korea, Republic of
facility: Clinical Trial Site
state: Gyeonggi-do
city: Goyang-si
country: Korea, Republic of
facility: Clinical Trial Site
state: Gyeonggi-do
city: Daegu
country: Korea, Republic of
facility: Clinical Trial Site
state: Hyeonchung-ro
city: Gwangju
country: Korea, Republic of
facility: Clinical Trial Site
state: Jebong Ro
city: Daegu
country: Korea, Republic of
facility: Clinical Trial Site
city: Gyeonggi-do
country: Korea, Republic of
facility: Clinical Trial Site 1
city: Gyeonggi-do
country: Korea, Republic of
facility: Clinical Trial Site 2
city: Jeju-do
country: Korea, Republic of
facility: Clinical Trial Site
city: Jeollabuk-do
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 2
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 3
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 5
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 6
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 7
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 4
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site 1
city: Łódź
country: Poland
facility: Clinical Trial Site
city: Łódź
country: Poland
facility: Clinical Trial Site
city: Moscow
country: Russian Federation
facility: Clinical Trial Site 1
city: Moscow
country: Russian Federation
facility: Clinical Trial Site 2
city: Saint Petersburg
country: Russian Federation
facility: Clinical Trial Site
city: Barcelona
country: Spain
facility: Clinical Trial Site 1
city: Barcelona
country: Spain
facility: Clinical Trial Site 2
city: Pamplona
country: Spain
facility: Clinical Trial Site
city: Sevilla
country: Spain
facility: Clinical Trial Site
city: Kaohsiung
country: Taiwan
facility: Clinical Trial Site
city: New Taipei City
country: Taiwan
facility: Clinical Trial Site
city: Taichung
country: Taiwan
facility: Clinical Trial Site
city: Taoyuan
country: Taiwan
facility: Clinical Trial Site
city: Ankara
country: Turkey
facility: Clinical Trial Site
city: Istanbul
country: Turkey
facility: Clinical Trial Site
city: Kayseri
country: Turkey
facility: Clinical Trial Site
city: Cardiff
country: United Kingdom
facility: Clinical Trial Site
city: Exeter
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
city: London
country: United Kingdom
facility: Clinical Trial Site
city: Manchester
country: United Kingdom
facility: Clinical Trial Site
city: Newcastle
country: United Kingdom
facility: Clinical Trial Site
city: Southampton
country: United Kingdom
facility: Clinical Trial Site
city: Wales
country: United Kingdom
facility: Clinical Trial Site
Study officials:
affiliation: Alexion Pharmaceuticals, Inc.
name: Nuria Saval
role: STUDY_DIRECTOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.