Paroxysmal Nocturnal Hemoglobinuria Treatment Study
People with paroxysmal nocturnal hemoglobinuria (PNH) experience sudden, irregular episodes of passing dark colored urine, especially at night or in the early morning. It is a rare and serious blood disease that causes red blood cells to break apart. Many patients with PNH are dependent on blood transfusions.
The only approved treatment for PNH is eculizumab. Eculizumab (Soliris) 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.
ALXN1210 is a longer-acting humanized monoclonal antibody directed against terminal complement protein C5, with potential anti-inflammatory activity. After receiving this complement C5 inhibitor, ALXN1210 binds to terminal complement protein C5. It blocks C5 splitting into pro-inflammatory components and prevents the complement-assisted destruction of red blood cells seen in paroxysmal nocturnal hemoglobinuria.
In order to participate you must meet the following criteria:
- Are 18 years of age or older.
- Have a diagnosis of PNH that is confirmed by high-sensitivity flow cytometry.
- Have one or more of the following PNH-related signs or symptoms within 3 months of screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath, anemia, history of a major adverse vascular event, such as thrombosis.
You will be excluded from the study if any of the following criteria apply to you:
- Have been previously treated with a complement inhibitor drug.
- Have a history of bone marrow transplantation.
- Have a body weight less than 88 pounds (or 40 kilograms).
This is a partial list of inclusion and exclusion criteria.