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Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00122304
Recruitment Status : Completed
First Posted : July 22, 2005
Last Update Posted : February 21, 2007
Sponsor:
Information provided by:
Alexion Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE July 19, 2005
First Posted Date  ICMJE July 22, 2005
Last Update Posted Date February 21, 2007
Study Start Date  ICMJE December 2004
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: February 20, 2007)
  • Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
  • Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Original Primary Outcome Measures  ICMJE
 (submitted: July 19, 2005)
Acceptable safety (AEs, labs, ECGs, vital signs)
Change History Complete list of historical versions of study NCT00122304 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: February 20, 2007)
  • Hemolysis measured by the change of LDH from baseline;
  • Quality of Life
Original Secondary Outcome Measures  ICMJE
 (submitted: July 19, 2005)
  • Hemolysis
  • Quality of Life
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
Official Title  ICMJE Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
Brief Summary The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemoglobinuria, Paroxysmal
Intervention  ICMJE Drug: eculizumab
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   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
 (submitted: July 19, 2005)
85
Original Enrollment  ICMJE Same as current
Study Completion Date  ICMJE November 2006
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • PNH > 6 months
  • Type III PNH red blood cell (RBC) clone by flow cytometry >10%
  • At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH
  • Lactate dehydrogenase (LDH) >1.5 x upper limit of normal
  • Must avoid conception
  • Willing and able to give written informed consent

Exclusion Criteria:

  • Platelet count of <30,000/mm3
  • Absolute neutrophil count <500/ul
  • Active bacterial infection
  • Hereditary complement deficiency
  • History of bone marrow transplantation
  • Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
  • Pregnant, breast-feeding, or intending to conceive
  • History of meningococcal disease
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   Canada,   France,   Germany,   Ireland,   Italy,   Netherlands,   Spain,   Sweden,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00122304
Other Study ID Numbers  ICMJE SHEPHERD
C04-002
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE Alexion Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Alexion Pharmaceuticals
Verification Date February 2007

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