Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

NCT01522183 | Recruiting

• 1 other identifier: M11-001
• Study Type: observational
• Target: 3,000
• Locations: 17 countries
• Sites: 141

Brief Summary

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

Conditions

Atypical Hemolytic-Uremic Syndrome

Keywords

Atypical Hemolytic-Uremic Syndrome; aHUS; Thrombotic Microangiopathy; TMA

Outcome Measures

Primary Outcomes (2)

• Proportion of patients who experience specified events

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

  10 years

• 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 or ravulizumab treatment or other disease management approaches

  5 years

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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)

You may qualify if:

• 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.

You may not qualify if:

• 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 \[IRB/IEC\]).

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead
• Syneos Health: collaborator

Study Sites (141)

Clinical Trial Site

Aurora, Colorado, 80045, United States

RECRUITING

Clinical Trial Site

Washington D.C., District of Columbia, 20007, United States

RECRUITING

Clinical Trial Site

Gainesville, Florida, 32610, United States

RECRUITING

Clinical Trial Site

Atlanta, Georgia, 30322, United States

RECRUITING

Clinical Trial Site

Chicago, Illinois, 60614, United States

WITHDRAWN

Clinical Trial Site

Boston, Massachusetts, 02115, United States

RECRUITING

Clinical Trial Site

St Louis, Missouri, 63110, United States

RECRUITING

Clinical Trial Site

Winston-Salem, North Carolina, 27103, United States

WITHDRAWN

Clinical Trial Site

Columbus, Ohio, 43210, United States

ACTIVE NOT RECRUITING

Clinical Trial Site 1

Westmead, New South Wales, 2145, Australia

RECRUITING

Clinical Trial Site 2

Westmead, New South Wales, 2145, Australia

RECRUITING

Clinical Trial Site

Cairns, Queensland, 4870, Australia

RECRUITING

Clinical Trial Site

Herston, Queensland, 4029, Australia

RECRUITING

Clinical Trial Site

Woolloongabba, Queensland, 4102, Australia

RECRUITING

Clinical Trial Site

Adelaide, South Australia, 5000, Australia

RECRUITING

Clinical Trial Site

Heidelberg, Victoria, 3084, Australia

RECRUITING

Clinical Trial Site

Parkville, Victoria, 3050, Australia

RECRUITING

Clinical Trial Site

Parkville, Victoria, 3052, Australia

RECRUITING

Clinical Trial Site

Murdoch, Western Australia, 6961, Australia

RECRUITING

Clinical Trial Site

Nedlands, Western Australia, 6109, Australia

ACTIVE NOT RECRUITING

Clinical Trial Site

Brussels, 1020, Belgium

ACTIVE NOT RECRUITING

Clinical Trial Site

Brussels, 1070, Belgium

RECRUITING

Clinical Trial Site

Brussels, 1090, Belgium

RECRUITING

Clinical Trial Site (Adult)

Edegem, 2650, Belgium

RECRUITING

Clinical Trial Site (Pediatric)

Edegem, 2650, Belgium

RECRUITING

Clinical Trial Site (pediatric)

Ghent, 9000, Belgium

RECRUITING

Clinical Trial Site (Adult)

Leuven, 3000, Belgium

RECRUITING

Clinical Trial Site (pediatric)

Leuven, 3000, Belgium

RECRUITING

Clinical Trial Site (Adult)

Liège, 4000, Belgium

RECRUITING

Clinical Trial Site (pediatric)

Liège, 4000, Belgium

RECRUITING

Clinical Trial Site (adult)

Woluwe-Saint-Lambert, 1200, Belgium

RECRUITING

Clinical Trial Site (Pediatric)

Woluwe-Saint-Lambert, 1200, Belgium

RECRUITING

Clinical Trial Site

London, Ontario, N6A 5W9, Canada

RECRUITING

Clinical Trial Site

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Clinical Trial Site

Montreal, Quebec, H3T 1C5, Canada

RECRUITING

Clinical Trial Site

Québec, Quebec, 44035, Canada

RECRUITING

Clinical Trial Site

Odense, 5000, Denmark

RECRUITING

Clinical Trial Site 1

Amiens, 80054, France

RECRUITING

Clinical Trial Site 2

Amiens, 80054, France

RECRUITING

Clinical Trial Site

Angers, 49933, France

RECRUITING

Clinical Trial Site

Bayonne, 64109, France

RECRUITING

Clinical Trial Site

Bordeaux, 33076, France

ACTIVE NOT RECRUITING

Clinical Trial Site

Brest, 29609, France

RECRUITING

Clinical Trial Site

Bron, 69677, France

RECRUITING

Clinical Trial Site

Caen, 30001, France

RECRUITING

Clinical Trial Site

Chambéry, 73011, France

RECRUITING

Clinical Trial Site

Clermont-Ferrand, 63000, France

RECRUITING

Clinical Trial Site

Dijon, 21080, France

RECRUITING

Clinical Trial Site

Grenoble, 38043, France

RECRUITING

Clinical Trial Site

Le Kremlin-Bicêtre, 94270, France

RECRUITING

Clinical Trial Site

Lille, 59037, France

RECRUITING

Clinical Trial Site

Lille, France

WITHDRAWN

Clinical Trial Site

Lyon, France

RECRUITING

Clinical Trial Site

Marseille, France

RECRUITING

Clinical Trial Site

Montpellier, France

RECRUITING

Clinical Trial Site 1

Nancy, 54500, France

RECRUITING

Clinical Trial Site

Nancy, 54500, France

RECRUITING

Clinical Trial Site

Nantes, 44 093, France

RECRUITING

Clinical Trial Site 1

Nantes, 44000, France

RECRUITING

Clinical Trial Site

Nice, 51069, France

RECRUITING

Clinical Trial Site 2

Paris, 75015, France

RECRUITING

Clinical Trial Site 4

Paris, 75015, France

RECRUITING

Clinical Trial Site 1

Paris, 75019, France

RECRUITING

Clinical Trial Site 5

Paris, 75743, France

RECRUITING

Clinical Trial Site 3

Paris, 75970, France

RECRUITING

Clinical Trial Site

Perpignan, France

RECRUITING

Clinical Trial Site

Poitiers, France

RECRUITING

Clinical Trial Site

Pontoise, France

RECRUITING

Clinical Trial Site

Rennes, 35203, France

RECRUITING

Clinical Trial Site

Rennes, France

RECRUITING

Clinical Trial Site 1

Rouen, France

RECRUITING

Clinical Trial Site 2

Rouen, France

RECRUITING

Clinical Trial Site 1

Strasbourg, France

RECRUITING

Clinical Trial Site 2

Strasbourg, France

RECRUITING

Clinical Trial Site

Toulouse, 31059, France

RECRUITING

Clinical Trial Site

Toulouse, 70034, France

RECRUITING

Clinical Trial Site

Tours, France

RECRUITING

Clinical Trial Site

Valenciennes, 50479, France

RECRUITING

Clinical Trial Site

Essen, 45147, Germany

RECRUITING

Clinical Trial Site 1

Hanover, 30625, Germany

RECRUITING

Clinical Trial Site 2

Hanover, 30625, Germany

RECRUITING

Clinical Trial Site

Heidelberg, 69120, Germany

RECRUITING

Clinical Trial Site

Lübeck, 23538, Germany

ACTIVE NOT RECRUITING

Clinical Trial Site

Münster, 48149, Germany

RECRUITING

Clinical Trial Site

Tübingen, 72076, Germany

WITHDRAWN

Clinical Trial Site

Beersheba, 84101, Israel

RECRUITING

Clinical Trial Site 1

Bari, 70124, Italy

RECRUITING

Clinical Trial Site 2

Bari, 70124, Italy

RECRUITING

Clinical Trial Site

Florence, 50134, Italy

RECRUITING

Clinical Trial Site

Genova, 16147, Italy

RECRUITING

Clinical Trial Site 1

Milan, 20122, Italy

RECRUITING

Clinical Trial Site 2

Milan, 20122, Italy

RECRUITING

Clinical Trial Site

Milan, 20163, Italy

RECRUITING

Clinical Trial Site

Roma, 00165, Italy

RECRUITING

Clinical Trial Site

Torino, Italy

WITHDRAWN

Clinical Trial Site

Lodz, 92-213, Poland

RECRUITING

Clinical Trial Site

Lodz, 93-338, Poland

RECRUITING

Clinical Trial Site 1

Moscow, Russia

COMPLETED

Clinical Trial Site 2

Moscow, Russia

COMPLETED

Clinical Trial Site

Saint Petersburg, Russia

COMPLETED

Clinical Trial Site

Dongan, Chungcheongnam-do, 31151, South Korea

RECRUITING

Clinical Trial Site

Seoul, Dongnam-ro, 05278, South Korea

RECRUITING

Clinical Trial Site

Anyang-si, Gyeonggi-do, 14068, South Korea

ACTIVE NOT RECRUITING

Clinical Trial Site

Goyang-si, Gyeonggi-do, 411-706, South Korea

WITHDRAWN

Clinical Trial Site

Daegu, Hyeonchung-ro, 42415, South Korea

RECRUITING

Clinical Trial Site

Gwangju, Jebong Ro, 61469, South Korea

RECRUITING

Clinical Trial Site

Daegu, 35015, South Korea

RECRUITING

Clinical Trial Site 1

Gyeonggi-do, 13620, South Korea

ACTIVE NOT RECRUITING

Clinical Trial Site 2

Gyeonggi-do, 46371, South Korea

RECRUITING

Clinical Trial Site

Jeju-do, 63241, South Korea

ACTIVE NOT RECRUITING

Clinical Trial Site

Jeollabuk-do, 54907, South Korea

ACTIVE NOT RECRUITING

Clinical Trial Site 2

Seoul, 02841, South Korea

RECRUITING

Clinical Trial Site 3

Seoul, 03722, South Korea

RECRUITING

Clinical Trial Site 5

Seoul, 05505, South Korea

RECRUITING

Clinical Trial Site 6

Seoul, 06351, South Korea

RECRUITING

Clinical Trial Site 7

Seoul, 07985, South Korea

RECRUITING

Clinical Trial Site 4

Seoul, 08308, South Korea

RECRUITING

Clinical Trial Site 1

Seoul, South Korea

RECRUITING

Clinical Trial Site 1

Barcelona, 09-08035, Spain

RECRUITING

Clinical Trial Site 2

Barcelona, 09-08035, Spain

RECRUITING

Clinical Trial Site

Pamplona, 15-31008, Spain

RECRUITING

Clinical Trial Site

Seville, 01-41013, Spain

WITHDRAWN

Clinical Trial Site

Basel, 4031, Switzerland

TERMINATED

Clinical Trial Site

Bern, 3010, Switzerland

TERMINATED

Clinical Trial Site

Surich, 8091, Switzerland

TERMINATED

Clinical Trial Site

Kaohsiung City, 81362, Taiwan

RECRUITING

Clinical Trial Site

New Taipei City, 10002, Taiwan

RECRUITING

Clinical Trial Site

Taichung, 40447, Taiwan

RECRUITING

Clinical Trial Site

Taoyuan District, 33305, Taiwan

RECRUITING

Clinical Trial Site

Ankara, 06500, Turkey (Türkiye)

RECRUITING

Clinical Trial Site

Istanbul, 34093, Turkey (Türkiye)

RECRUITING

Clinical Trial Site

Kayseri, 38039, Turkey (Türkiye)

RECRUITING

Clinical Trial Site

Cardiff, C14 4XW, United Kingdom

RECRUITING

Clinical Trial Site

Exeter, EX2 5DW, United Kingdom

RECRUITING

Clinical Trial Site

London, NW3 2PF, United Kingdom

RECRUITING

Clinical Trial Site

London, W1T 4EU, United Kingdom

RECRUITING

Clinical Trial Site

London, WC1N 3JH, United Kingdom

RECRUITING

Clinical Trial Site

Manchester, M13 9WL, United Kingdom

RECRUITING

Clinical Trial Site

Newcastle, NE7 7DN, United Kingdom

WITHDRAWN

Clinical Trial Site

Southampton, SO16 6YD, United Kingdom

RECRUITING

Clinical Trial Site

Wales, SA6 6NL, United Kingdom

RECRUITING

Related Publications (6)

• Gaeckler A, Al-Dakkak I, Saval N, Dieperink HH, Eygenraam M, Greenbaum LA, Isbel N, Walle JV. Effectiveness and Safety of Switching to Ravulizumab From Eculizumab in Kidney Transplant Recipients With Atypical Hemolytic Uremic Syndrome: A Global aHUS Registry Analysis. Clin Transplant. 2025 Sep;39(9):e70278. doi: 10.1111/ctr.70278.

  PMID: 40875209 DERIVED

• 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.

  PMID: 36152218 DERIVED

• 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.

  PMID: 27871301 DERIVED

• 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.

  PMID: 26654630 DERIVED

• 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.

  PMID: 24843058 DERIVED

• 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.

  PMID: 24360866 DERIVED

MeSH Terms

Conditions

Atypical Hemolytic Uremic Syndrome; Thrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Hemolytic-Uremic Syndrome; Uremia; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytopenia; Blood Platelet Disorders; Cytopenia

Study Officials

• Nuria Saval

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 18, 2012
• First Posted: January 31, 2012
• Study Start: March 18, 2013
• Primary Completion (Estimated): January 1, 2031
• Study Completion (Estimated): January 1, 2031
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

BE (12); CA (4); IT (9); ES (4); TU (3); CH (3); RU (3); IL (1); KR (18); PL (2); TW (4); DE (7); AU (11); GB (9); FR (41); US (9); DK (1)