Atypical Hemolytic Uremic Syndrome Clinical Trial
— EVIDENCEOfficial title:
Evidence - Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
Verified date | December 2019 |
Source | Alexion Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
This was a prospective, open-label study with no participant randomization. Treatment for aHUS was observational and at the discretion of the treating physician. The purpose of this study was to assess disease manifestations of complement-mediated thrombotic microangiopathy (TMA) and evaluate potential clinical predictors of disease manifestations and progression in participants with aHUS with or without eculizumab treatment in the clinical setting.
Status | Terminated |
Enrollment | 67 |
Est. completion date | October 5, 2017 |
Est. primary completion date | October 5, 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility |
Inclusion Criteria: 1. Currently receiving eculizumab treatment in the M11-001 aHUS Registry 2. Two normal platelet counts at least 4 weeks apart 3. Two normal lactate dehydrogenase levels at least 4 weeks apart 4. Willing, committed, and able to return for all clinic visits and complete all study-related procedures 5. 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: 1. Any prior eculizumab treatment discontinuation 2. On chronic dialysis (defined as =3 months on dialysis) 3. Currently participating in another complement inhibitor trial 4. Life expectancy of <6 months 5. 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) |
Country | Name | City | State |
---|---|---|---|
Australia | Clinical Trial Site | Adelaide | |
Australia | Clinical Trial Site | Clayton | |
Australia | Clinical Trial Site | Heidelberg | |
Australia | Clinical Trial Site | Kingswood | |
Australia | Clinical Trial Site | Liverpool | |
Australia | Clinical Trial Site | Nedlands | |
Australia | Clinical Trial Site | Parkville | |
Australia | Clinical Trial Site | Parkville | |
Australia | Clinical Trial Site | Perth | |
Australia | Clinical Trial Site | Westmead | |
Australia | Clinical Trial Site | Woolloongabba | |
Germany | Clinical Trial Site | Hannover | |
Germany | Clinical Trial Site | Hannöver | |
Germany | Clinical Trial Site | Kiel | |
Germany | Clinical Trial Site | Luebeck | |
United Kingdom | Clinical Trial Site | London | |
United States | Clinical Trial Site | Atlanta | Georgia |
United States | Clinical Trial Site | Boston | Massachusetts |
United States | Clinical Trial Site | Charlotte | North Carolina |
United States | Clinical Trial Site | Chicago | Illinois |
United States | Clinical Trial Site | Columbus | Ohio |
United States | Clinical Trial Site | Hackensack | New Jersey |
United States | Clinical Trial Site | Houston | Texas |
United States | Clinical Trial Site | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals |
United States, Australia, Germany, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Rate Of Thrombotic Microangiopathy (TMA) Manifestations During Eculizumab Treatment Compared To Off-Treatment | 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. |
Baseline, 24 Months | |
Secondary | Change From Baseline To 24 Months In Estimated Glomerular Filtration Rate (eGFR) | 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. |
Baseline, 24 Months | |
Secondary | Incidence Of Plasma Exchange And Plasma Infusion (PE/PI) | 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. |
Baseline, 24 Months |
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