Treatment Study for Patients with Paroxysmal Nocturnal Hemoglobinuria
PNH is a rare and serious blood disease that causes red blood cells to break apart. This breaking apart is called hemolysis. This disease can affect people of any age, but it is diagnosed most often in people in their 30s and 40s. It may be associated with aplastic anemia, myelodysplastic syndrome, or acute myelogenous leukemia.
The only approved treatment for PNH is eculizumab. Eculizumab is a type of monoclonal antibody. It is used to prevent red blood cells from being destroyed in patients with PNH. Eculizumab binds to an immune system protein call C5. This helps keep red blood cells from breaking down and helps keep blood clots from forming.
RA101495 is a synthetic peptide with a complex life cycle which binds to a unique site on the C5 protein to inhibit the division into C5a and C5b, thereby preventing red blood cell lysis by inhibiting the production and assembly of the membrane attack complex (MAC). It is being developed as an alternative to the intravenously-administered monoclonal antibody therapy for PNH. This is a Phase II study using RA101495 for patients whose disease has had an inadequate response to treatment with eculizumab. RA101495 is administered by the patient to themselves, which may improve dosing convenience and prevent a patient from having to return to the hospital. Everyone who takes part in the study will receive RA101495.
In order to participate you must meet the following criteria:
- Are 18 years or older and have a diagnosis of PNH by flow cytometry.
- Have had an inadequate response to eculizumab.
You will be excluded from the study if any of the following criteria apply to you:
- Have a platelet count less than 30,000/μL or an absolute neutrophil count (ANC) less than 500 cells/μL at screening.
- Have a history of meningococcal disease.
- Have a current systemic infection or suspicion of active bacterial infection.
This is a partial list of inclusion and exclusion criteria.