Safety and Efficacy Study of Eculizumab in Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

Phase 2

Completed

• Conditions: Shiga-like Toxin-producing Escherichia Coli

• Registration Number: NCT01410916
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

This protocol is designed to collect safety and efficacy data on patients that have been or will be treated with eculizumab for STEC-HUS, in the context of the 2011 STEC-HUS epidemic in Germany.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-07
• Study First Submitted: 2011-08-03
• Study First Submitted QC: 2011-08-04
• Study First Posted: 2011-08-05
• Primary Completion: 2012-01
• Study Completion: 2012-06
• Status Verified: 2013-04
• Last Update Submitted: 2013-04-04
• Last Update Posted: 2013-04-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 198

Inclusion Criteria

1. Patient must be willing and able to give written informed consent/Assent.
2. Adults, adolescents, or pediatric (≥2 months and ≥5kg) patients
3. Patient has been diagnosed with Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

Exclusion Criteria

1. Known complement regulatory mutation or family history of complement regulatory mutation
2. Unresolved systemic meningococcal disease
3. 3. Hypersensitivity to eculizumab, to murine proteins or to one of the excipients

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Improvement in Systemic TMA & Vital Organ Involvement at 8 weeks of treatment defined as either complete or partial responder based on hematologic normalization/improvement & clinically important improvement in Vital Organs: Brain, Kidney, and Thrombosis	8 weeks	Analysis of primary endpoint will occur after all patients have reached 8 weeks of treatment. The response rate will be summarized as patients who are either a complete or partial responder.