Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria

The name “paroxysmal nocturnal hemoglobinuria” (PNH) comes from: Paroxysmal which means “sudden and irregular;” Nocturnal which means “at night;” and Hemoglobinuria which means “hemoglobin in urine;” hemoglobin is the red part of red blood cells, it makes urine look dark. PNH symptoms are sudden, irregular episodes of passing dark colored urine, especially at night or in the early morning, although many people with PNH do not have dark urine. PNH is a rare and serious blood disease that causes red blood cells to break apart. Doctors call this breaking apart “hemolysis.” When red blood cells break apart, the hemoglobin inside is released. Hemoglobin is the red part of red blood cells that carries oxygen around the body.

ALXN2040 (danicopan) is a complement system Factor D inhibitor. Factor D plays a key role in the activation of the alternative complement pathway that signals hemolysis in PNH. This study will determine if danicopan, when used with a C5 inhibitor, improves anemia in patients with PNH.

If you agree to participate in this study, your participation may last up to 18 months (24-week study period and the 52-week long-term extension) and include up to 35 visits (20 to the study center, 15 have the potential to be completed at home using a visiting healthcare service).

During these visits, you will be asked to have physical exams, electrocardiograms (ECGs, tests that measure the electrical activity of your heart), urine sample collections for safety and research testing, and blood sample collections for safety and research purposes. You will also receive an oral thermometer (Day 1) and instruction on how and when to measure your temperature and the importance of taking your body temperature at home. You will be asked to complete quality of life questionnaires at certain timepoints during the study. 

In order to participate you must meet the following criteria:

• Are 18 years of age or older and are diagnosed with paroxysmal nocturnal hemoglobinuria (PNH).
• Have anemia.
• Are receiving an approved C5 inhibitor (such as eculizumab or ravulizumab )for at least 6 months prior to Day 1 in this study at an approved dose (or higher) and with no change in dose or interval for at least 24 weeks preceding Day 1.

You will be excluded from the study if any of the following criteria apply to you:You cannot participate in this study if:

• Have a history of a major organ transplant (such as heart, lung, kidney, liver) or HSCT.
• Have known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants.
• Have known underlying bleeding disorders (such as coagulation factor deficiencies, idiopathic thrombocytopenic purpura.

This is a partial list of eligibility requirements. To inquire about your eligibility, please call the contact number provided. If you wish to inquire via email, please include the title of the study in your message.