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

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2011 by Alexion Pharmaceuticals.
Recruitment status was  Active, not recruiting
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

August 31, 2010
June 11, 2012
September 2010
July 2012   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01193348 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome

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.

Not Provided
Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Atypical Hemolytic-Uremic Syndrome
Drug: Eculizumab
Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Not Provided
Tschumi S, Gugger M, Bucher BS, Riedl M, Simonetti GD. Eculizumab in atypical hemolytic uremic syndrome: long-term clinical course and histological findings. Pediatr Nephrol. 2011 Nov;26(11):2085-8. doi: 10.1007/s00467-011-1989-4. Epub 2011 Aug 30.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
15
Not Provided
July 2012   (final data collection date for primary outcome measure)

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.
Both
1 Month to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Canada
 
NCT01193348
C10-003
Not Provided
Alexion Pharmaceuticals
Alexion Pharmaceuticals
Not Provided
Not Provided
Alexion Pharmaceuticals
August 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP