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

Shiga-like Toxin-producing Escherichia Coli Clinical Trial

— STEC-HUS  

Official title:

An Open-Label, Multi-Center Trial of Eculizumab in Patients With Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01410916
• Other study ID #: C11-001
• Secondary ID: 2011-002691-17
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: August 3, 2011
• Last updated: April 4, 2013
• Start date: July 2011
• Est. completion date: June 2012

Study information

• Verified date: April 2013
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Paul-Ehrlich-Institut
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 198
• Est. completion date: June 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 2 Months and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemolytic-Uremic Syndrome
• Shiga-like Toxin-producing Escherichia Coli

Intervention

Drug:
• Eculizumab (Soliris®)
Eculizumab 300 mg, 600 mg, 900 mg or 1200 mg will be administered intravenously

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	8 weeks	No