Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

This study has been completed.

Sponsor:

Information provided by:

Alexion Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT00122330

First received: July 18, 2005

Last updated: November 30, 2006

Last verified: November 2006

History of Changes

Tracking Information
First Received Date ^ICMJE	July 18, 2005
Last Updated Date	November 30, 2006
Start Date ^ICMJE	October 2004
Primary Completion Date	Not Provided
Current Primary Outcome Measures ^ICMJE	Not Provided
Original Primary Outcome Measures ^ICMJE	Not Provided
Change History	Complete list of historical versions of study NCT00122330 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	Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Official Title ^ICMJE	Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
Brief Summary	The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
Detailed Description	Not Provided
Study Type ^ICMJE	Interventional
Study Phase	Phase 3
Study Design ^ICMJE	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double-Blind Primary Purpose: Educational/Counseling/Training
Condition ^ICMJE	Hemoglobinuria, Paroxysmal
Intervention ^ICMJE	Drug: eculizumab
Study Arm (s)	Not Provided
Publications *	Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. Hill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. Epub 2010 Mar 8.
* 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	Completed
Enrollment ^ICMJE	75
Completion Date	January 2006
Primary Completion Date	Not Provided
Eligibility Criteria ^ICMJE	Inclusion Criteria: Must have required at least 4 transfusions in the past 12 months PNH type III red blood cell (RBC) clone by flow cytometry of >10% Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal Platelet count > 100,000/mm3 Patient taking erythropoietin must be on a stable dose for at least 26 weeks Patient taking immunosuppressants must be on a stable dose for at least 26 weeks Patient taking corticosteroids must be on a stable dose for at least 4 weeks Patient taking coumadin must be at a stable INR for at least 4 weeks Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks Willing and able to give written informed consent Must avoid conception Exclusion Criteria: Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl Absolute neutrophil count <500/ul Active bacterial infection Hereditary complement deficiency Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days Pregnant, breast-feeding, or intending to conceive History of meningococcal disease History of bone marrow transplantation
Gender	Both
Ages	18 Years and older
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States, Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Sweden, Switzerland, United Kingdom

Administrative Information
NCT Number ^ICMJE	NCT00122330
Other Study ID Numbers ^ICMJE	TRIUMPH
Has Data Monitoring Committee	Not Provided
Responsible Party	Not Provided
Study Sponsor ^ICMJE	Alexion Pharmaceuticals
Collaborators ^ICMJE	Not Provided
Investigators ^ICMJE	Not Provided
Information Provided By	Alexion Pharmaceuticals
Verification Date	November 2006
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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