Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
Status:
Completed
Trial end date:
2007-06-25
Target enrollment:
Participant gender:
Summary
This study will evaluate the safety and effectiveness of the experimental drug eculizumab for
treating paroxysmal nocturnal hemoglobinuria (PNH), a disorder that can cause premature
destruction of red blood cells. PNH may result in anemia requiring blood transfusions.
Patients may be at high risk of life-threatening blood clots in their veins and may have
urine discoloration, stomach pain, difficulty swallowing, tiredness, and poor quality of
life. Men may have problems getting or maintaining an erection. Eculizumab is a monoclonal
antibody that may improve the survival of red blood cells in patients with PNH.
Patients 18 years of age and older who have been diagnosed with PNH for more than 6 months,
who have active disease, and who require blood transfusions may be eligible for this study.
Each candidate is screened with a physical examination, electrocardiogram, blood and urine
tests, and a questionnaire for information on how PNH affects the patient physically,
socially, emotionally, and functionally.
Participants receive infusions of eculizumab through a needle in a vein once a week for five
doses and then every two weeks for another 24 doses. All patients are vaccinated against
Neisseria meningitides, a bacteria that can cause symptoms, possibly including
life-threatening meningitis, in susceptible people, including people who take eculizumab.
At every treatment visit, patients update their health status, transfusion record, and
medication use; review their laboratory results from the preceding visit; have vital signs
measured; and provide a blood sample for laboratory tests. At selected visits, they also
provide a urine sample, have a repeat electrocardiogram, and complete a questionnaire. At the
final treatment visit, participants have a complete physical examination, in addition to the
routine procedures.