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

NCT ID: NCT02614898

Last Updated: 2020-01-02

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Total Enrollment

67 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-11-04

Study Completion Date

2017-10-05

Brief Summary

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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.

Detailed Description

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Conditions

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Atypical Hemolytic Uremic Syndrome

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

Eculizumab

Intervention Type OTHER

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.

Interventions

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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.

Intervention Type OTHER

Other Intervention Names

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Soliris®

Eligibility Criteria

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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)
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Trial Site

Washington D.C., District of Columbia, United States

Site Status

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Boston, Massachusetts, United States

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Hackensack, New Jersey, United States

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Charlotte, North Carolina, United States

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Columbus, Ohio, United States

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Houston, Texas, United States

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Adelaide, , Australia

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Clayton, , Australia

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Heidelberg, , Australia

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Kingswood, , Australia

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Liverpool, , Australia

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Nedlands, , Australia

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Parkville, , Australia

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Parkville, , Australia

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Perth, , Australia

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Westmead, , Australia

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Woolloongabba, , Australia

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Hannover, , Germany

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Hanover, , Germany

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Kiel, , Germany

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Lübeck, , Germany

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London, , United Kingdom

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Countries

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United States Australia Germany United Kingdom

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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2015-003135-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ECU-aHUS-403

Identifier Type: -

Identifier Source: org_study_id

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