To Characterize the Safety and Tolerability of Eculizumab in Two Japanese aHUS Patients

This study has been completed.
Information provided by (Responsible Party):
Alexion Pharmaceuticals Identifier:
First received: December 4, 2012
Last updated: December 21, 2012
Last verified: February 2012
This protocol is intended to formally collect data on the treatment of aHUS with eculizumab in Japanese patients.


Study Type: Observational
Study Design: Time Perspective: Retrospective
Official Title: A Research Study to Describe the Safety and Efficacy of Eculizumab in Japanese Patients With Atypical Hemolytic Uremic Syndrome (aHUS). An Assessment of Two Case Studies

Resource links provided by NLM:

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Change in platelet count from baseline [ Time Frame: 26 weeks ] [ Designated as safety issue: Yes ]

Enrollment: 2
Study Start Date: May 2012
Study Completion Date: July 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The two identified Japanese patients with aHUS for which eculizumab treatment was initiated in 2011

Inclusion Criteria:

  1. Patient and/or legal guardian must be willing and able to give written informed consent and id the patient is not an adult and able to read and write, the patient is willing to give written informed assent
  2. Eculizumab treatment initiated as personal importation for aHUS in 201

Exclusion Criteria:

  Contacts and Locations
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Please refer to this study by its identifier: NCT01755429

Shinshu University School of Medicine
Matsumoto, Japan
Tokyo Medical and Dental University
Tokyo, Japan
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

Responsible Party: Alexion Pharmaceuticals Identifier: NCT01755429     History of Changes
Other Study ID Numbers: C11-004J 
Study First Received: December 4, 2012
Last Updated: December 21, 2012
Health Authority: Japan: Pharmaceuticals and Medical Devices Agency

Additional relevant MeSH terms:
Atypical Hemolytic Uremic Syndrome
Hemolytic-Uremic Syndrome
Anemia, Hemolytic
Blood Platelet Disorders
Hematologic Diseases
Kidney Diseases
Pathologic Processes
Thrombotic Microangiopathies
Urologic Diseases processed this record on May 26, 2016