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

This study has been completed.
Sponsor:
Information provided by:
Alexion Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00122304
First received: July 19, 2005
Last updated: February 20, 2007
Last verified: February 2007
History of Changes
  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:
  • Hemolysis measured by the change of LDH from baseline;
  • Quality of Life

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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Show 48 Study Locations
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 16, 2013