paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0 Level: PT Classification code 10034042
Conditions
Sponsors
ALEXION PHARMACEUTICALS INCORPORATED
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Individuals at least 18 years of age 2. Patients must have required at least 4 episodes of transfusions in the 12 months prior to Visit 1 for anemia or anemia-related symptoms 3. Presence of a GPI deficient red blood cell clone (type III cells) by flow cytometry equal to or greater than 10% 4. Patients taking erythropoeitin must be on a stable dose for 26 weeks prior to Visit 1 and the dose must be expected to remain stable during the observation period and the treatment phase 5. Patients taking immunosuppressants must be on a stable dose for at least 26 weeks prior to visit 1 and the dose must be expected to rermain stable during the Observation Period and the Treatment Phase 6. Other criteria specified in the Protocol Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients whose mean hemoglobin levels prior to transfusion over the previous 12 months is greater than 10.5 gm/dL 2. Absolute neutrophil count < 500 per microlitre 3. Presence or suspicion of active bacterial infection in the opinion of the investigator, at Visit 3 or recurrent bacterial infections 4. Known or suspected hereditary complement deficiency 5. Participation in any other drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to Visit 1 6. Pregnant, breast-feeding or intending to conceive during the course of the study, including the post-treatment phase 7. Any of the criteria stated in the Protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic paroxysmal hemoglobinuria (PNH) ;Secondary Objective: ;Primary end point(s): Co-primary endpoints for this study are: hemoglobin stablization and the number of PRBC units transfused during the treatment phase of the study to 26 weeks. | — |
Countries
Ireland, Italy, Sweden
Outcome results
None listed