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

Alexion Pharmaceuticals, Inc.198 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

198

Conditions

Shiga-like Toxin-producing Escherichia Coli

Interventions

Eculizumab (Soliris®)DRUG

Eculizumab 300 mg, 600 mg, 900 mg or 1200 mg will be administered intravenously

Eligibility

Sex: ALLMin age: 2 Months

Medical Language ↔ Plain English

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

Locations (22)

Unnamed facility

Bielefeld, Germany

Unnamed facility

Bielefeld, Germany

Unnamed facility

Bonn, Germany

Outcomes

Primary Outcomes

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

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.

Time frame: 8 weeks

Data from ClinicalTrials.gov

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