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aHUS Observational Long Term Follow-Up (LTFU)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Alexion Pharmaceuticals Identifier:
First received: January 18, 2012
Last updated: July 25, 2016
Last verified: July 2016
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.

Atypical Hemolytic Uremic Syndrome

Study Type: Observational [Patient Registry]
Study Design: Observational Model: Case-Only
Target Follow-Up Duration: 5 Years
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

Resource links provided by NLM:

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • 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.

Secondary Outcome Measures:
  • Duration of response, change over time of value for platelets, LDH, eGFR, Hgb, TMA intervention [ Time Frame: 5 Years ]
    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.

Enrollment: 94
Study Start Date: March 2012
Estimated Study Completion Date: January 2017
Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure)

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male and female aHUS, including minors, who previously participated in eculizumab clinical trials.

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01522170

  Show 56 Study Locations
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Responsible Party: Alexion Pharmaceuticals Identifier: NCT01522170     History of Changes
Other Study ID Numbers: C11-003 
Study First Received: January 18, 2012
Last Updated: July 25, 2016

Keywords provided by Alexion Pharmaceuticals:
Atypical Hemolytic Uremic Syndrome

Additional relevant MeSH terms:
Atypical Hemolytic Uremic Syndrome
Hemolytic-Uremic Syndrome
Pathologic Processes
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Hematologic Diseases
Thrombotic Microangiopathies
Blood Platelet Disorders processed this record on February 17, 2017