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Completed

NCT Number: NCT00122304

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

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

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Key information

Conditions

Hemoglobinuria, Paroxysmal Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal North Shore Hospital, Haematology Department, Saint Leonard, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Eculizumab

Drug

Primary outcomes

  1. Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)

  2. Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.

Secondary outcomes

  1. Hemolysis measured by the change of LDH from baseline;

  2. Quality of Life

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study

Important dates

Study start
2004
Study completion
2006
First posted
Jul 22, 2005
Registry last updated
Feb 21, 2007

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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