Hemoglobinuria, Paroxysmal
Conditions
Brief summary
The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH
Interventions
DRUGeculizumab
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* PNH \> 6 months * Type III PNH red blood cell (RBC) clone by flow cytometry \>10% * At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH * Lactate dehydrogenase (LDH) \>1.5 x upper limit of normal * Must avoid conception * Willing and able to give written informed consent
Exclusion criteria
* Platelet count of \<30,000/mm3 * Absolute neutrophil count \<500/ul * Active bacterial infection * 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 * Pregnant, breast-feeding, or intending to conceive * History of meningococcal disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs) | — |
| Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve. | — |
Secondary
| Measure | Time frame |
|---|---|
| Hemolysis measured by the change of LDH from baseline; | — |
| Quality of Life | — |
Countries
Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States
Outcome results
None listed