Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (EVIDENCE)
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| ClinicalTrials.gov Identifier: NCT02614898 |
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Recruitment Status :
Terminated
(Terminated for administrative reasons before enrollment was complete.)
First Posted : November 25, 2015
Results First Posted : January 2, 2020
Last Update Posted : January 2, 2020
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| Condition or disease | Intervention/treatment |
|---|---|
| Atypical Hemolytic Uremic Syndrome | Other: Eculizumab |
| Study Type : | Observational |
| Actual Enrollment : | 67 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment |
| Actual Study Start Date : | November 4, 2015 |
| Actual Primary Completion Date : | October 5, 2017 |
| Actual Study Completion Date : | October 5, 2017 |
| Group/Cohort | Intervention/treatment |
|---|---|
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Eculizumab
The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician.
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Other: Eculizumab
This study was an observational study. Therefore, the study drug was not provided as part of this study, and the dosing regimen was based solely at the discretion of the treating physician.
Other Name: Soliris® |
- Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment [ Time Frame: Baseline, 24 Months ]
A TMA manifestation was defined as one of following: Hematologic or renal events due to aHUS; extra-renal clinical signs and symptoms of aHUS; tissue (for example, kidney transplant) biopsy demonstrating TMA due to aHUS.
The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
- Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR) [ Time Frame: Baseline, 24 Months ]
Change in estimated eGFR over time using the chronic kidney disease-epidemiology (CKD-EPI) formula.
The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
- Incidence Of Plasma Exchange And Plasma Infusion (PE/PI) [ Time Frame: Baseline, 24 Months ]
The number of occurrences of PE/PI per participant-years was calculated and summarized by treatment status.
The study was terminated with only 20% of the planned enrollment and due to the subsequent loss of funding for the program, the samples collected for outcome measure assessment could not be analyzed to generate summary-level data. All participants enrolled in ECU-aHUS-403 were concurrently enrolled in protocol M11-001, and their data will be included in the analyses for M11-001.
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, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
Inclusion Criteria:
- Currently receiving eculizumab treatment in the M11-001 aHUS Registry
- Two normal platelet counts at least 4 weeks apart
- Two normal lactate dehydrogenase levels at least 4 weeks apart
- Willing, committed, and able to return for all clinic visits and complete all study-related procedures
- Participant or participant's parent/legal guardian must have been willing and able to give written informed consent. Participant (if minor) must have been willing to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)
Exclusion Criteria:
- Any prior eculizumab treatment discontinuation
- On chronic dialysis (defined as ≥3 months on dialysis)
- Currently participating in another complement inhibitor trial
- Life expectancy of <6 months
- Participant or participant's parent/legal guardian was unable to give written informed consent. Participant (if minor) was unable to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02614898
| United States, District of Columbia | |
| Clinical Trial Site | |
| Washington, District of Columbia, United States, 20007 | |
| United States, Georgia | |
| Clinical Trial Site | |
| Atlanta, Georgia, United States, 30322 | |
| United States, Illinois | |
| Clinical Trial Site | |
| Chicago, Illinois, United States, 60611 | |
| United States, Massachusetts | |
| Clinical Trial Site | |
| Boston, Massachusetts, United States, 02115 | |
| United States, New Jersey | |
| Clinical Trial Site | |
| Hackensack, New Jersey, United States, 07601 | |
| United States, North Carolina | |
| Clinical Trial Site | |
| Charlotte, North Carolina, United States, 28203 | |
| United States, Ohio | |
| Clinical Trial Site | |
| Columbus, Ohio, United States, 43210 | |
| United States, Texas | |
| Clinical Trial Site | |
| Houston, Texas, United States, 77030 | |
| Australia | |
| Clinical Trial Site | |
| Adelaide, Australia, 5000 | |
| Clinical Trial Site | |
| Clayton, Australia, 3168 | |
| Clinical Trial Site | |
| Heidelberg, Australia, 3084 | |
| Clinical Trial Site | |
| Kingswood, Australia, 2747 | |
| Clinical Trial Site | |
| Liverpool, Australia, 2170 | |
| Clinical Trial Site | |
| Nedlands, Australia, 6109 | |
| Clinical Trial Site | |
| Parkville, Australia, 3050 | |
| Clinical Trial Site | |
| Parkville, Australia, 3052 | |
| Clinical Trial Site | |
| Perth, Australia, 6008 | |
| Clinical Trial Site | |
| Westmead, Australia, 2145 | |
| Clinical Trial Site | |
| Woolloongabba, Australia, 4102 | |
| Germany | |
| Clinical Trial Site | |
| Hannover, Germany, 30625 | |
| Clinical Trial Site | |
| Hannöver, Germany, 30625 | |
| Clinical Trial Site | |
| Kiel, Germany, 24105 | |
| Clinical Trial Site | |
| Luebeck, Germany, 23538 | |
| United Kingdom | |
| Clinical Trial Site | |
| London, United Kingdom, NW3 2PF | |
| Responsible Party: | Alexion |
| ClinicalTrials.gov Identifier: | NCT02614898 |
| Other Study ID Numbers: |
ECU-aHUS-403 2015-003135-35 ( EudraCT Number ) |
| First Posted: | November 25, 2015 Key Record Dates |
| Results First Posted: | January 2, 2020 |
| Last Update Posted: | January 2, 2020 |
| Last Verified: | December 2019 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Eculizumab Hemolytic-Uremic Syndrome Atypical Hemolytic Uremic Syndrome Syndrome Disease Progression Hemolysis Disease Pathologic Processes Disease Attributes Uremia Kidney Diseases |
Urologic Diseases Anemia, Hemolytic Anemia Hematologic Diseases Thrombotic Microangiopathies Thrombocytopenia Blood Platelet Disorders Complement Inactivating Agents Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |