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

This study has been completed.

First Received on July 19, 2005. Last Updated on February 20, 2007 History of Changes

Sponsor:	Alexion Pharmaceuticals
Information provided by:	Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT00122304

Purpose

The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH

Condition	Intervention	Phase
Hemoglobinuria, Paroxysmal	Drug: eculizumab	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

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:

Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.

Secondary Outcome Measures:

Estimated Enrollment:	85
Study Start Date:	December 2004
Estimated Study Completion Date:	November 2006

Eligibility

Criteria

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00122304

Sponsors and Collaborators

Alexion Pharmaceuticals

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00122304 History of Changes
Obsolete Identifiers:	NCT00133120
Other Study ID Numbers:	SHEPHERD, C04-002
Study First Received:	July 19, 2005
Last Updated:	February 20, 2007
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations

Signs and Symptoms
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases

ClinicalTrials.gov processed this record on May 21, 2012