Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
Completed · Phase 3
Conditions studied: Paroxysmal Hemoglobinuria, Nocturnal
In brief
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.
Key facts
- Study ID
- NCT00130000
- Run by
- National Heart, Lung, and Blood Institute (NHLBI)
- People needed
- 87
- Starts
- 2005-08-09
- Expected to finish
- 2007-06-25
- Last updated by the study team
- 2017-07-02
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- Any patient meeting any of the following criteria will be excluded from the study:
- Platelet count of less than 30,000/mm3
- Absolute Neutrophil count less than or equal to 500/uL
- Presence or suspicion of active bacterial infection, in the opinion of the Investigator, at Visit 2 or recurrent bacterial infections
- Known or suspected 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 prior to screening
- Pregnant, breast-feeding, or intending to conceive during the course of the study,including the Safety Follow-up Visits
- 8 .History of meningococcal disease
- Patients who are not vaccinated against N. meningitidis at least 14 days prior to Visit 2
- Any condition that, in the opinion of the Investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.
Where it is running
- National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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