Evaluation of Potential Predictors of Disease Progression in Participants With Atypical Hemolytic Uremic Syndrome (aHUS) Including Genetics, Biomarkers, and Treatment (EVIDENCE)

Alexion Pharmaceuticals

Status: Terminated

Conditions: Atypical Hemolytic Uremic Syndrome

Treatments: Other: Eculizumab

Study type: Observational

Funder types: Industry

Identifiers

NCT02614898

ECU-aHUS-403

2015-003135-35 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 67 patients

Sex: All

Volunteers: No Healthy Volunteers

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)

Trial design

67 participants in 1 patient group

Eculizumab

Description:

The targeted population consists of pediatric and adult participants with aHUS who received eculizumab at the discretion of the treating physician.

Treatment:

Other: Eculizumab