Text Size

An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome (aHUS)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01193348
First received: August 31, 2010
Last updated: June 11, 2012
Last verified: August 2011
History of Changes
  Purpose

The primary purpose is to assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.


Condition Intervention Phase
Atypical Hemolytic-Uremic Syndrome
 Drug: Eculizumab
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

Resource links provided by NLM:

Drug Information available for: Eculizumab
U.S. FDA Resources

Further study details as provided by Alexion Pharmaceuticals:

Primary Outcome Measures:
  • Study endpoints will be assessed at every dosing visit on an ongoing basis during and at the end of the Treatment Period. An evaluation of these parameters will also occur at every dosing visit during and at the end of the extension treatment period. [ Time Frame: 6 month enrollment, 6 to 24 (or more) months treatment and extension period respectively. ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: September 2010
Estimated Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Eculizumab
    Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
  Eligibility

Ages Eligible for Study:   1 Month to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion:

  1. Patient's parent/legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].
  2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease.
  3. Patients from 1 month up to 18 years of age and body weight ≥ 5 kg.
  4. Patients exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine.

Exclusion:

  1. Plasma therapy for > 5 weeks prior to enrollment.
  2. Chronic dialysis.
  3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01193348

Locations
United States, California
Los Angeles, California, United States, 90095
San Diego, California, United States, 92123
United States, Colorado
Aurora, Colorado, United States, 80045
United States, Georgia
Atlanta, Georgia, United States, 30322
United States, Iowa
Iowa City, Iowa, United States, 52242
United States, New Jersey
Hackensack, New Jersey, United States, 07601
United States, New York
Bronx, New York, United States, 10467
United States, North Carolina
Charlotte, North Carolina, United States, 28203
United States, Ohio
Cincinnati, Ohio, United States, 45229
United States, Texas
Corpus Christi, Texas, United States, 78411
United States, Washington
Spokane, Washington, United States, 99204
Canada, Ontario
Toronto, Ontario, Canada, M5G1X8
Sponsors and Collaborators
Alexion Pharmaceuticals
  More Information

No publications provided by Alexion Pharmaceuticals

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01193348     History of Changes
Other Study ID Numbers: C10-003
Study First Received: August 31, 2010
Last Updated: June 11, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Alexion Pharmaceuticals:
Atypical Hemolytic-Uremic Syndrome

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

ClinicalTrials.gov processed this record on May 19, 2013