Eval. of Potential Predictors of Disease Progression in Patients With aHUS Including Genetics, Biomarkers and Treatment
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Gastrointestinal |
| Therapuetic Areas: | Gastroenterology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 11/9/2017 |
| Start Date: | November 2015 |
| End Date: | April 2020 |
Evaluation of Potential Predictors of Disease Progression in Patients With aHUS, Including Genetics, Biomarkers and Treatment
This is a prospective, open-label study with no patient randomization. Treatment for atypical
hemolytic uremic syndrome (aHUS) will remain observational and at the discretion of the
treating physician. The purpose of this study is to assess disease manifestations of
complement mediated thrombotic microangiopathy (TMA) and potential clinical predictors of
disease manifestations and progression in patients with aHUS with or without eculizumab
treatment in the clinical setting.
hemolytic uremic syndrome (aHUS) will remain observational and at the discretion of the
treating physician. The purpose of this study is to assess disease manifestations of
complement mediated thrombotic microangiopathy (TMA) and potential clinical predictors of
disease manifestations and progression in patients with aHUS with or without eculizumab
treatment in the clinical setting.
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 LDH levels at least 4 weeks apart
4. Willing, committed, and able to return for ALL clinic visits and complete all
study-related procedures
5. Patient or patient's parent/legal guardian must be willing and able to give written
informed consent. Patient (if minor) must be 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. 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)
We found this trial at
10
sites
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