Atypical Hemolytic Uremic SyndromeLTFUNCT01522170
aHUS Observational Long Term Follow-Up
An observational study of Atypical Hemolytic Uremic Syndrome, run by Alexion Pharmaceuticals, Inc. at 56 sites in 12 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome
- How many people
- 94 participants
- Where
- 56 sitesin 12 countries
- Study dates
- Mar 2012to Jan 2017
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Male and female aHUS, including minors, who previously participated in eculizumab clinical trials.
Who may be able to join
- 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.
Who cannot join
- Not applicable.
Trial locations and contacts
56 sites in 12 countries.
56 sites in 12 countries
France17 sites
Centre Hospitalier Universitaire Pellegrin, Service de Nephrologie Transplantation Dialyse
Bordeaux
CHRU de Caen
Caen
Le Kremlin Bicetre Hospital, Nephrology Unit
Le Kremlin-Bicêtre
CHRU de Lille-Hopital A.Calmette
Lille
Hôpital Edouard Herriot
Lyon
Hopital de la Timone Enfants, Unité de Néphrologie
Marseille
CHU Hotel Dieu
Nantes
Le Centre Hospitalier Universitaire de Nice
Nice
Centre Hospitalier Regional de la Source, Service de nephrologie/hemodialyse
Orléans
Hopital Robert-Debré, Service de Néphrologie Pédiatrique
Paris
Hopital Tenon
Paris
Centre Hospitalier Inter-Communal de Cornouaille
Quimper
CHU-CH Charles Nicolle Pavillon de Pédiatrie
Rouen
Hôpital de Bois Guillaume CHU de Rouen
Rouen
CHU de Saint-Etienne
Saint-Priest-en-Jarez
Nouvel Hopital Civil
Strasbourg
CHRU de Tours
Tours
United States16 sites
Emory Healthcare - Children's Center
Atlanta, Georgia
Boston Children's Hospital
Boston, Massachusetts
Cincinnati Children's Hospital
Cincinnati, Ohio
Arthur James Cancer Hospital
Columbus, Ohio
Driscoll Children's Hospital
Corpus Christi, Texas
Henry Ford Health System
Detroit, Michigan
Dunwoody Pediatrics & Children's Healthcare of Atlanta
Dunwoody, Georgia
Fort Wayne Medical Oncology and Hematology
Fort Wayne, Indiana
Study site
Fort Wayne, Indiana
Hudson Valley Oncology Hematology
Hawthorne, New York
The Methodist Hospital Research Institute
Houston, Texas
Weill Cornell Medical College
New York, New York
Fox Valley Hematology
Oshkosh, Wisconsin
Seattle Children's Hospital
Seattle, Washington
Providence Sacred Heart Medical Center & Children's Hospital
Spokane, Washington
Children's Hospital at Montefiore
The Bronx, New York
Italy5 sites
A.O. Papa Giovanni XXIII
Bergamo
Azienda Ospedaliero-Universitaria Careggi
Florence
Istituto Giannina Gaslini
Genova
Fondazione IRCCS Ca Granda, Ospedale Maggiore Policlinico Maggiore Policlinico
Milan
Ospedale dei Bambini G. di Cristina
Palermo
United Kingdom4 sites
Royal Devon & Exeter NHS Foundation Trust
Exeter
Gartnavel General Hospital
Glasgow
The Newcastle upon Tyne Hospitals NHS
Newcastle upon Tyne
City Hospital, Nottingham University Hospitals, NHS Trust
Nottingham
Germany3 sites
Universitaetsklinikum Aachen Klinik fuer Nieren- und Hochdruckkrankheiten
Aachen
Hannover University Clinic
Hanover
Universitaet Heidelberg
Heidelberg
Austria2 sites
Universitatsklinik fur Innere Medizin Medizinische Universitat Graz
Graz
Medizinische Universitaet Innsbruck
Innsbruck
Canada2 sites
Centre Hospitalier Universitaire (CHU) Sainte-Justine
Montreal
The Hospital for Sick Children
Toronto
Netherlands2 sites
AMC Medical Research B.V.
Amsterdam
Radboud University Nijmegen Medical Centre Nijmegen
Nijmegen
Switzerland1 site
INSELSPITAL Universitaetsklinik fuer Kinderheilkunde Kindernephrologie
Bern
About this study
Official title: 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
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.
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Keywords
- Atypical Hemolytic Uremic Syndrome, aHUS, Eculizumab
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
TMA complication-free survival
Assess the long term efficacy of eculizumab in patients with aHUS who have previously participated in an eculizumab clinical study.
Measured: 5 Years
Secondary outcomes (1)
Duration of response, change over time of value for platelets, LDH, eGFR, Hgb, TMA intervention
Assess the long term safety of eculizumab in patients with aHUS who have previously participated in an eculizumab clinical study by collecting data on serious adverse events and targeted adverse events.
Measured: 5 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jan 2012 (estimated)
- Study startMar 2012
- Primary completionJan 2017
- Study completionJan 2017
Why it stopped: Remaining patients were offered a similar observational study (Alexion M11-001) to allow for robust, high quality data collection in a larger, single database
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Atypical Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 94 participants (actual)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01522170
- Sponsor's protocol ID
- C11-003
- Acronym
- LTFU
- Status last verified
- Feb 2017
- First submitted
- 18 Jan 2012
- Last updated
- 24 Feb 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Menne J, Delmas Y, Fakhouri F, Licht C, Lommele A, Minetti EE, Provot F, Rondeau E, Sheerin NS, Wang J, Weekers LE, Greenbaum LA. Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study. BMC Nephrol. 2019 Apr 10;20(1):125. doi: 10.1186/s12882-019-1314-1.
PubMed 30971227
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01522170
- Acronym / sponsor ID
- LTFU / C11-003
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Keywords
- Atypical Hemolytic Uremic Syndrome, aHUS, Eculizumab
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Remaining patients were offered a similar observational study (Alexion M11-001) to allow for robust, high quality data collection in a larger, single database
- Enrolment
- 94 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Mar 2012 (actual)
- Primary completion
- Jan 2017 (actual)
- Study completion
- Jan 2017 (actual)
- First posted
- 31 Jan 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 5 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Male and female aHUS, including minors, who previously participated in eculizumab clinical trials.
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (1 listed)
- Not applicable.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
TMA complication-free survival
Time frame: 5 Years
Assess the long term efficacy of eculizumab in patients with aHUS who have previously participated in an eculizumab clinical study.
1 secondary outcome
Duration of response, change over time of value for platelets, LDH, eGFR, Hgb, TMA intervention
Time frame: 5 Years
Results and safety
The study was stopped early (reason given: Remaining patients were offered a similar observational study (Alexion M11-001) to allow for robust, high quality data collection in a larger, single database). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2017.
- Derived from this study: PubMed 30971227Menne J, Delmas Y, Fakhouri F, Licht C, Lommele A, Minetti EE, Provot F, Rondeau E, Sheerin NS, Wang J, Weekers LE, Greenbaum LA. Outcomes in patients with atypical hemolytic uremic syndrome treated with eculizumab in a long-term observational study. BMC Nephrol. 2019 Apr 10;20(1):125. doi: 10.1186/s12882-019-1314-1.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
56 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Atypical Hemolytic Uremic Syndrome, run by Alexion Pharmaceuticals, Inc. at 56 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 56 sites in France, United States, Italy, United Kingdom, Germany and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Remaining patients were offered a similar observational study (Alexion M11-001) to allow for robust, high quality data collection in a larger, single database). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
TMA complication-free survival (time frame: 5 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01522170, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2017; AzTrials last refreshed its copy on 29 Oct 2026.