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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01410916
First received: August 3, 2011
Last updated: April 4, 2013
Last verified: April 2013
  Purpose

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.


Condition Intervention Phase
Shiga-like Toxin-producing Escherichia Coli
Drug: Eculizumab (Soliris®)
Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center Trial of Eculizumab in Patients With Shiga-Toxin Producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

Resource links provided by NLM:


Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • 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 [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    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.


Enrollment: 198
Study Start Date: July 2011
Study Completion Date: June 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Eculizumab (Soliris®)
    Eculizumab 300 mg, 600 mg, 900 mg or 1200 mg will be administered intravenously
  Eligibility

Ages Eligible for Study:   2 Months and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01410916

Locations
Germany
Bielefeld, Germany, 33617
Bielefeld, Germany, 33615
Bonn, Germany, 53127
Bremen, Germany, 28177
Bremerhaven, Germany, 27574
Essen, Germany, 45147
Flensburg, Germany, 24939
Göttingen, Germany, 37075
Hamburg, Germany, 22291
Hamburg, Germany, 20246
Hamburg, Germany, 22359
Hannover, Germany, 30625
Karlsruhe, Germany, 76133
Köln, Germany, 50937
Lubeck, Germany, 23538
Magdeburg, Germany, 39120
Munchen, Germany, 81377
Munich, Germany, 80804
Munster, Germany, 48149
Oldenburg, Germany, 26133
Ulm, Germany, 89081
Wildeshausen, Germany, 27793
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01410916     History of Changes
Other Study ID Numbers: C11-001, 2011-002691-17
Study First Received: August 3, 2011
Last Updated: April 4, 2013
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by Alexion Pharmaceuticals:
STEC-HUS
Shiga-like toxin-producing Escherichia Coli Hemolytic-Uremic Syndrome
uncontrolled complement activation

Additional relevant MeSH terms:
Hemolytic-Uremic Syndrome
Azotemia
Hemolysis
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders
Pathologic Processes
Shiga Toxins
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 18, 2014