Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
Status:
Completed
Trial end date:
2006-02-01
Target enrollment:
Participant gender:
Summary
This study will examine the safety and effectiveness of the experimental drug eculizumab in
treating patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare disorder of red
blood cells that leads to premature destruction of the cells and resulting anemia. Patients
may be at high risk of blood clots and may develop bone marrow failure or aplastic anemia,
with low white blood cell and platelet counts. Eculizumab is a monoclonal antibody that may
help improve the survival of red blood cells.
Patients 18 years of age and older with PNH who require blood transfusions for anemia and
have received at least four transfusions in the 12 months preceding evaluation for this study
may be eligible to enroll. Candidates are screened with a medical history, physical
examination, and check of vital signs.
Participants have an electrocardiogram (EKG) and blood and urine tests, and are vaccinated
against Neisseria meningitides, a common bacteria that can cause a disabling or fatal type of
meningitis. They then enter an observation phase of the study, with monthly visits during
which they complete a questionnaire; update their health status, transfusion record, and
medication use; have their vital signs checked and PNH symptoms evaluated; have blood and
urine tests; and receive a transfusion, if necessary. These visits continue for up to 3
months until patients receive a "qualifying" transfusion; that is, a transfusion given as a
consequence of a certain hemoglobin level with symptoms or a different level without
symptoms.
Patients are then randomly assigned to receive either eculizumab or a placebo (salt solution
with no active ingredient). Both study medications are given intravenously (through a vein)
over 30 minutes once a week for five doses and then once every 2 weeks for another 11 doses.
At each treatment visit (study weeks 0-24), patients update their health status, transfusion
records, and medication use; have their vital signs checked; and provide a blood sample. At
various visits, they also complete a questionnaire, provide a urine sample and have an EKG.
At the last treatment visit (week 26 or the final visit for patients who end their
participation before visit 18) patients have a complete physical examination in addition to
the procedures listed above.