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

Tracking Information
First Received Date ^ICMJE	August 31, 2010
Last Updated Date	June 11, 2012
Start Date ^ICMJE	September 2010
Estimated Primary Completion Date	July 2012 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: August 31, 2010)	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 ]
Original Primary Outcome Measures ^ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01193348 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE	Not Provided
Original Secondary Outcome Measures ^ICMJE	Not Provided
Current Other Outcome Measures ^ICMJE	Not Provided
Original Other Outcome Measures ^ICMJE	Not Provided

Descriptive Information
Brief Title ^ICMJE	An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
Official Title ^ICMJE	An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome
Brief Summary	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.
Detailed Description	Not Provided
Study Type ^ICMJE	Interventional
Study Phase	Phase 2
Study Design ^ICMJE	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Condition ^ICMJE	Atypical Hemolytic-Uremic Syndrome
Intervention ^ICMJE	Drug: Eculizumab Fixed dosing is based on body weight cohorts. Adjustment of dose to accommodate patient growth is possible.
Study Arm (s)	Not Provided
Publications *	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. Epub 2011 Aug 30.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Active, not recruiting
Estimated Enrollment ^ICMJE	15
Completion Date	Not Provided
Estimated Primary Completion Date	July 2012 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	Inclusion: 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)]. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease. Patients from 1 month up to 18 years of age and body weight ≥ 5 kg. Patients exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine. Exclusion: Plasma therapy for > 5 weeks prior to enrollment. Chronic dialysis. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
Gender	Both
Ages	1 Month to 18 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States, Canada

Administrative Information
NCT Number ^ICMJE	NCT01193348
Other Study ID Numbers ^ICMJE	C10-003
Has Data Monitoring Committee	Not Provided
Responsible Party	Alexion Pharmaceuticals
Study Sponsor ^ICMJE	Alexion Pharmaceuticals
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	Alexion Pharmaceuticals
Verification Date	August 2011
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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