This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This is an open-label multicenter study of Eculizumab administered intravenously (IV) to approximately 85 patients with hemolytic paroxysmal nocturnal hemoglobinuria (PNH). Patients who meet the entry criteria will receive 600 mg of Eculizumab IV once a week for 4 doses, followed by 900 mg Eculizumab IV 1 week later for 1 dose, then 900 mg Eculizumab IV every 2 weeks for approximately 24 doses. Each patient who completes the study will receive approximately 29 IV infusions. The estimated duration of the study, given a 12-week enrollment period, 2-week screening period, 52-week Treatment Period, and 8-week post-treatment period is approximately 74 weeks. Patients who prematurely discontinue study medication will nevertheless require 4 follow-up visits at 1, 2, 4, and 8 weeks post last dose of study medication and, a follow-up contact 12 weeks after their last dose of study medication. In rare circumstances related to holidays, travel or weather issues, or the patient's medical needs, a patient may be allowed to vary the visit interval by 2 days. If a patient has shortened visit intervals, additional visits may be required in order to meet the 52-week Treatment Period. Sampling for pharmacokinetics (PK) and pharmacodynamics (PD) (% hemolysis) will be done for the purpose of studying the dose-effect relationship. Covariates of interest will be explored.
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